Praluent® (alirocumab) Approved By FDA, The Drug Helps to Prevent Heart Attack, Stroke and Unstable Angina Requiring Hospitalization

Praluent® (alirocumab) to Prevent Heart Attack, Stroke and Unstable Angina Requiring Hospitalization, approved by FDA

TARRYTOWN, N.Y. and PARIS, April 26, 2019 /PRNewswire/ --

• Praluent is the first PCSK9 inhibitor that has shown a meaningful reduction in death from any cause
• Praluent treatment effect was observed in patients already receiving other lipid-lowering therapies, including maximally-tolerated statins

Regeneron Pharmaceuticals, Inc. (NASDAQ:  REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Praluent^® (alirocumab) to reduce the risk of heart attack, stroke and unstable angina requiring hospitalization in adults with established cardiovascular (CV) disease.

"Heart disease accounts for one quarter of all American deaths each year and many others are at risk for heart attack and stroke due to uncontrolled LDL-C levels," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer, Regeneron. "The Phase 3 ODYSSEY OUTCOMES trial showed that people who received Praluent significantly reduced their risk for serious cardiovascular events. There was also a clinically-meaningful reduction in death from any cause with Praluent treatment. With this approval, and the recent introduction of a lower U.S. Praluent list price, we hope that more patients in need will be able to access Praluent."

High levels of "bad" cholesterol, also known as low-density lipoprotein cholesterol (LDL-C), increase patients' risk for serious CV events such as heart attack or stroke. Adults who experience a heart attack or stroke have an approximately one in three chance to have another CV event. 

"Today's FDA approval marks a significant achievement in the treatment of adults with established cardiovascular disease, who are among those at greatest risk of death or disability caused by serious cardiovascular events," said John Reed, M.D., Ph.D., Global Head of Research & Development, Sanofi. "Praluent has already helped many adults lower their LDL-C levels, and this new indication provides an opportunity to help appropriate patients by reducing the risk of serious, life-threatening cardiovascular events, including heart attacks and stroke."

The FDA approval is based on data from ODYSSEY OUTCOMES, which was published in the New England Journal of Medicine in November 2018, assessing the effect of adding Praluent to maximally-tolerated statins on CV outcomes in 18,924 patients who had an acute coronary syndrome (ACS) within a year of enrolling in the trial. Patients who received Praluent in the trial experienced:

• A 15% reduced risk for major CV events. The primary endpoint included time to first heart attack, stroke, death from coronary heart disease (CHD), or unstable angina requiring hospitalization (HR 0.85; 95% CI, 0.78 to 0.93; p=0.0003).
• A 27% reduced risk of stroke, 14% reduced risk of non-fatal heart attack and 39% reduced risk of unstable angina requiring hospitalization.*
• A 15% reduced risk of death from any cause (also called all-cause mortality; HR 0.85; 95% CI, 0.73 to 0.98; nominal p=0.026) was also observed.*

*Because statistical testing of these endpoints was performed outside of the hierarchy, the results are not considered statistically significant.

Adverse events were similar between the Praluent and placebo groups, except for injection site reactions (Praluent 3.8%, placebo 2.1%). In ODYSSEY OUTCOMES, the adverse events that occurred in >5% of patients included: non-cardiac chest pain (7.0% Praluent, 6.8% placebo), nasopharyngitis (6.0% Praluent, 5.6% placebo) and myalgia (5.6% Praluent, 5.3% placebo).

The FDA also approved Praluent as an adjunct to diet, alone or in combination with other lipid-lowering therapies (e.g., statins, ezetimibe), for the treatment of adults with primary hyperlipidemia (including heterozygous familial hypercholesterolemia) to reduce LDL-C.

Praluent was the first PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor approved by the FDA and is the only PCSK9 inhibitor available in two doses with two levels of efficacy as a single 1 mL injection (75 mg and 150 mg) once every two weeks. It can also be administered as 300 mg once every four weeks (monthly), enabling physicians to tailor treatment based on an individual patient's LDL-C-lowering needs. 

About ODYSSEY OUTCOMES ODYSSEY OUTCOMES, the longest CV outcomes trial of any PCSK9 inhibitor to date, assessed the effect of Praluent on the occurrence of major adverse CV events in patients who had experienced an ACS before enrolling in the trial, and who were already on intensive or maximally-tolerated statin treatment. Patients were randomized to receive Praluent (n=9,462) or a placebo (n=9,462) and were assessed for a median of 2.8 years, with some patients being treated for up to five years. Approximately 90% of patients were on high-intensity statins prior to randomization.

The trial was designed to maintain patients' LDL-C levels between 25-50 mg/dL (0.65-1.29 mmol/L), using two different doses of Praluent (75 mg and 150 mg). Praluent-treated patients started the trial on 75 mg every two weeks and switched to 150 mg every two weeks if their LDL-C levels remained above 50 mg/dL (n=2,615). Some patients who switched to 150 mg switched back to 75 mg if their LDL-C fell below 25 mg/dL (n=805), and patients who experienced two consecutive LDL-C measurements below 15 mg/dL (0.39 mmol/L) while on the 75 mg dose (n=730) stopped active Praluent therapy for the remainder of the trial.

About PraluentPraluent^® (alirocumab) inhibits the binding of PCSK9 to the LDL receptor and thereby increases the number of available LDL receptors on the surface of liver cells to clear LDL, which lowers LDL-C levels in the blood. Praluent was developed by Regeneron and Sanofi under a global collaboration agreement and invented by Regeneron using the company's proprietary VelocImmune^® technology that yields optimized fully-human monoclonal antibodies.

Praluent is approved in more than 60 countries worldwide, including the U.S., European Union (EU), Japan, Canada, Switzerland, Mexico and Brazil. In the U.S., Praluent is approved to reduce the risk of heart attack, stroke and unstable angina requiring hospitalization in adults with established CV disease. Praluent is also approved as an adjunct to diet, alone or in combination with other lipid lowering therapies (e.g., statins, ezetimibe), for the treatment of adults with primary hyperlipidemia (including heterozygous familial hypercholesterolemia) to reduce LDL-C.

Important Safety Information for the U.S. Do not use Praluent if you are allergic to alirocumab or to any of the ingredients in Praluent.

Before you start using Praluent, tell your healthcare provider about all your medical conditions, including allergies, and if you are pregnant or plan to become pregnant or if you are breastfeeding or plan to breastfeed.

Tell your healthcare provider or pharmacist about any prescription and over-the-counter medicines you are taking or plan to take, including natural or herbal remedies.

Praluent can cause serious side effects, including allergic reactions that can be severe and require treatment in a hospital. Call your healthcare provider or go to the nearest hospital emergency room right away if you have any symptoms of an allergic reaction including a severe rash, redness, severe itching, a swollen face, or trouble breathing.

The most common side effects of Praluent include: redness, itching, swelling, or pain/tenderness at the injection site, symptoms of the common cold, and flu or flu-like symptoms. Tell your healthcare provider if you have any side effect that bothers you or that does not go away.

Talk to your doctor about the right way to prepare and give yourself a Praluent injection and follow the "Instructions for Use" that comes with Praluent.

You are encouraged to report negative side effects of prescription drugs to the FDA. 

Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please click here for the full Prescribing Information.

About Regeneron Pharmaceuticals, Inc.Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to seven FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neuromuscular diseases, infectious diseases and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite^® technologies, such as VelocImmune^® which produces optimized fully-human antibodies, and ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.

For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.

About SanofiSanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Sanofi, Empowering Life

Regeneron Contacts:	Sanofi Contacts:
Media Relations Joe Ricculli Tel: +1 (914) 847-0405 joseph.ricculli@regeneron.com Investor Relations Mark Hudson Tel: +1 (914) 847-3482  mark.hudson@regeneron.com	Media Relations Nicolas Kressmann Tel: +1 (732) 532-5318 nicolas.kressmann@sanofi.com Investor Relations George Grofik Tel: +33 (0)1 53 77 45 45 ir@sanofi.com

SOURCE Regeneron Pharmaceuticals, Inc.

FDA Approves The Marketting Of Clearmate, A New Device By Thornhill Research, Inc. To Help Treat Carbon Monoxide Poisoning

FDA allows marketing of new device to help treat carbon monoxide poisoning

For Immediate Release

March 14, 2019

Release

The U.S. Food and Drug Administration today allowed marketing of a new device, ClearMate, intended to be used in an emergency room setting to help treat patients suffering from carbon monoxide poisoning. The device uses a novel method for quickly removing carbon monoxide from the body by increasing a patient’s rate of breathing.

“Carbon monoxide poisoning is a serious issue, affecting thousands of people each year,” said Malvina Eydelman, M.D., director of the Division of Ophthalmic, and Ear, Nose and Throat Devices in the FDA's Center for Devices and Radiological Health. “While the current standard treatment of administering 100 percent oxygen through a mask can be done anywhere, hyperbaric treatment, which is necessary for severe carbon monoxide poisoning, is less accessible because there are only 60 medical centers with hyperbaric units in the entire U.S. Moreover, those medical facilities are seldom in rural areas, so treatment in those areas could be delayed considerably due to transport time. Today’s marketing authorization provides patients with access to a simple, yet lifesaving device that may minimize the delay of getting vital treatment, especially in severe cases of carbon monoxide poisoning.”

Carbon monoxide is a colorless, odorless gas that is extremely poisonous and can kill within minutes. In the U.S. each year, nearly 500 people die while as many as 20,000 visit emergency rooms for unintended exposure to carbon monoxide, primarily from poorly-maintained heating systems, or gas stoves or gas-powered generators used for heat or power during storms. The most common symptoms of carbon monoxide poisoning are headache, dizziness, weakness, upset stomach, vomiting, chest pain and confusion. Carbon monoxide poisoning occurs when carbon monoxide attaches to the hemoglobin in the blood exactly where oxygen is supposed to attach, reducing the amount of oxygen carried to the brain and other tissues.

The standard form of treatment for carbon monoxide poisoning is to have the patient breathe 100 percent oxygen through a mask. In severe cases, a hyperbaric chamber may be used, which delivers oxygen under higher than normal pressure.

ClearMate—a device consisting of a gas mixer, valves, meters, breathing circuits, an oxygen reservoir, a mask and hoses—works by speeding up the elimination of carbon monoxide from the body. It delivers both 100 percent oxygen to the patient, as well as a mixture of oxygen and carbon dioxide, causing the patient to breathe faster. The increased breathing accelerates the rate at which the carbon monoxide leaves the patient’s body, allowing a normal amount of oxygen to attach to hemoglobin and be carried where it is needed throughout the body.

In authorizing marketing of ClearMate, the FDA reviewed data from multiple clinical studies, which tested the effectiveness of the device on 100 patients. The studies demonstrated the device was effective at eliminating carbon monoxide. The combination of oxygen and carbon dioxide in the ClearMate resulted in a faster elimination of carbon monoxide than treatment with 100 percent oxygen alone but was not faster than hyperbaric oxygen therapy.

Patients did not experience any device-related complications in the clinical studies of efficacy or in a separate study of the device’s safety.  

ClearMate was reviewed under the FDA’s De Novo premarket review pathway, a regulatory pathway for low-to-moderate-risk devices of a new type. This action creates a new regulatory classification, which means that subsequent devices of the same type with the same intended use may go through the FDA’s 510(k) premarket process, whereby devices can obtain marketing authorization by demonstrating substantial equivalence to a predicate device.

The FDA granted marketing authorization of ClearMate to Thornhill Research, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

FDA Permits Marketing Miris Human Milk Analyzer Diagnostic Test To Aid Healthcare Professionals

Miris HMA - Human Milk Analyzer from Camilla Sandberg on Vimeo.

The U.S. Food and Drug Administration, FDA, allowed marketing of the Miris Human Milk Analyzer in the USA.,THe Miris Device is a new diagnostic test to aid healthcare professionals to analyze and measure nutrients in breast milk. The measurements include the concentration of fat, carbohydrate, protein, total solids and energy. These results of the analysis will provide healthcare professionals with a new tool to aid in the nutritional management of newborns and young infants at risk for growth failure due to prematurity or other medical conditions.

“Breast milk provides many health benefits to infants, and for many babies it can meet their early nutritional needs. But some infants — including those who may be born preterm or have certain health conditions — may need additional nutrients in order to support their optimal growth. For the first time, doctors have access to a test to help analyze the nutrients in breast milk. While this test is not for everyone, it has the potential to aid parents and healthcare providers, mainly in a hospital setting, in better assessing the nutrient needs of certain babies who are not growing as expected.” said Courtney Lias, Ph.D. director of the Division of Chemistry and Toxicology Devices in the FDA’s Center for Devices and Radiological Health.

FDA Release

Today, the U.S. Food and Drug Administration permitted marketing of the Miris Human Milk Analyzer, a new diagnostic test to aid healthcare professionals in measuring nutrients in breast milk, including the concentration of fat, carbohydrate, protein, total solids and energy. The test provides healthcare professionals with a new tool to aid in the nutritional management of newborns and young infants at risk for growth failure due to prematurity or other medical conditions.

“Breast milk provides many health benefits to infants, and for many babies it can meet their early nutritional needs. But some infants — including those who may be born preterm or have certain health conditions — may need additional nutrients in order to support their optimal growth."said Courtney Lias, Ph.D. director of the Division of Chemistry and Toxicology Devices in the FDA’s Center for Devices and Radiological Health. "For the first time, doctors have access to a test to help analyze the nutrients in breast milk. While this test is not for everyone, it has the potential to aid parents and healthcare providers, mainly in a hospital setting, in better assessing the nutrient needs of certain babies who are not growing as expected.”

Some infants who are born prematurely or with health conditions may have special nutritional needs. Breast milk composition can vary in individuals, and, in certain cases, breast milk may not contain sufficient protein and energy levels for these infants with increased nutrient needs. In those instances, healthcare professionals may want to test the nutrient content of the milk to help guide nutritional care. Knowing the macronutrient content of the breast milk may help the health care team and parents make informed decisions on how to fortify the breast milk based on the individual needs of the infant.

The Miris Human Milk Analyzer uses an infrared spectroscopy system to analyze samples of human milk and provide a quantitative measurement of fat, protein and total carbohydrate content as well as calculations of the total solids and energy content contained in the milk. This is a prescription device intended for use by trained health care personnel at clinical laboratories.

The FDA reviewed data submitted by the sponsor of 112 samples of human milk to analyze the performance of the device. For the study, the sponsor tested the same 112 samples in the machine and compared them to the expected true values obtained by independent methods; both systems provided similar results for each test. The data showed that the Miris Human Milk Analyzer test was effective at determining levels of protein, fat and carbohydrate in the milk. There may be conditions that limit the information available from the Miris Human Milk Analyzer. For example, certain medications that a nursing mother may be taking could interfere with the test’s ability to accurately measure nutrient levels in breast milk.

Health care professionals should carefully evaluate the Miris Human Milk Analyzer test results in conjunction with clinical assessments (such as weight and growth) to inform their discussions with parents in creating a nutritional management plan for an infant or newborn.

The FDA reviewed the Miris Human Milk Analyzer test through the De Novo premarket review pathway, a regulatory pathway for low-to-moderate-risk devices of a new type. Along with this authorization, the FDA is establishing criteria, called special controls, to provide for the accuracy and reliability of tests intended to measure the nutritional content of human milk to aid in the nutritional management of certain infants. These special controls, when met along with general controls, provide a reasonable assurance of safety and effectiveness for tests of this type. This action also creates a new regulatory classification, which means that subsequent devices of the same type, including the same intended use, may go through the FDA’s 510(k) process, whereby devices can obtain marketing authorization by demonstrating substantial equivalence to a predicate device.

The FDA granted marketing authorization of the Miris Human Milk Analyzer test to Miris AB.

Meridian Bioscience, Inc's Alethia CMV Assay Test System Approved By FDA For Herpes Virus, cytomegalovirus (CMV) Detection In Newborns.

CMV-infected pneumocytes with large, intranuclear, basophilic inclusions with haloes at their periphery as well as small, granular cytoplasmic inclusions. Yale Rosen - Flickr

Today the U.S. Food and Drug Administration permitted marketing of a new diagnostic test to aid in detecting a type of herpes virus called cytomegalovirus (CMV) in newborns less than 21 days of age.

“Although most people who become infected with cytomegalovirus face little to no risk of serious illness, the virus has the potential to cause serious illness for people with weak immune systems and in newborn babies, This test for detecting the virus, when used in conjunction with the results of other diagnostic tests, may help health care providers more quickly identify the virus in newborns and determine the best approach for the child.”said Tim Stenzel, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. 

The Alethia CMV Assay Test System is used as an aid in the diagnosis of congenital CMV infection by detecting CMV deoxyribonucleic acid (DNA) from a saliva swab. Test results should be used only in conjunction with the results of other diagnostic tests and clinical information.

The FDA evaluated the analytical and clinical performance of the device. Data from a prospective clinical study showed that 1,472 saliva samples out of 1,475 samples collected from newborns were correctly identified by the device as negative for the presence of CMV DNA. Three samples were incorrectly identified as positive when they were negative. Five collected saliva specimens were correctly identified as positive for the presence of CMV DNA. The FDA also reviewed data from a testing of 34 samples of archived specimens from babies known to be infected with CMV in which all the archived specimens were correctly identified by the device as positive for presence of CMV DNA. The FDA reviewed the Alethia CMV Assay Test System through the de novo premarket review pathway, a regulatory pathway for novel, low-to-moderate-risk devices of a new type. Along with this authorization, the FDA is establishing criteria, called special controls, which determines the requirements for demonstrating accuracy, reliability and effectiveness of tests intended to be used as an aid in the diagnosis of congenital CMV infection. These special controls, when met along with general controls, provide a reasonable assurance of safety and effectiveness for tests of this type.
This action also creates a new regulatory classification, which means that subsequent devices of the same type with the same intended use may go through the FDA’s 510(k) process, whereby devices can obtain marketing authorization by demonstrating substantial equivalence to a predicate device.
The FDA granted marketing authorization of the Alethia CMV Assay Test System to Meridian Bioscience, Inc.

FDA Rejects KRYSTEXXA As A Treatment For Chronic Gout

Savient Pharmaceuticals (SVNT) announced that it has received a complete response letter from the U.S. Food and Drug Administration stating that the FDA cannot at this time approve the Company's application for KRYSTEXXA as a treatment for chronic gout in patients refractory to conventional therapy.
Market News Video

Tags: Approval, Chronic Gout, Savient Pharmaceuticals, KRYSTEXXA

Onglyza (saxagliptin), New Drug To Treat Type 2 Diabetes, Approved By FDA

For Immediate Release: July 31, 2009

Media Inquiries: Karen Riley, 301-796-4674, karen.riley@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA Approves New Drug Treatment for Type 2 Diabetes

The U.S. Food and Drug Administration today approved Onglyza (saxagliptin), a once-daily tablet to treat Type 2 diabetes in adults. The medication is intended to be used with diet and exercise to control high blood sugar levels.

The hormone insulin keeps blood sugar (glucose) levels within a narrow range in people who don’t have diabetes. People with Type 2 diabetes are either resistant to insulin or do not produce enough insulin to maintain normal blood sugar levels.

Onglyza is in a class of drugs known as dipeptidyl peptidase-4 (DPP-4) inhibitors which stimulate the pancreas to make more insulin after eating a meal.

“Keeping blood sugar levels in adequate control is essential to the good health of the 24 million people in the United States with Type 2 diabetes,” said Mary Parks, M.D., director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation and Research. “High blood sugar levels can cause blurry vision and excessive urination and eventually result in such serious conditions as kidney and eye disease.”

The most common side effects observed with Onglyza are upper respiratory tract infection, urinary tract infection, and headache. Other side effects include allergic-like reactions such as rash and hives.

Approval of Onglyza was primarily based on the results of eight clinical trials. The application seeking FDA approval was submitted before December 2008 when the agency recommended that manufacturers of new diabetes drugs carefully design and evaluate their clinical trials for cardiovascular safety. Although Onglyza was not associated with an increased risk for cardiovascular events in patients who were mainly at low risk for these events, the FDA is requiring a postmarket study that will specifically evaluate cardiovascular safety in a higher risk population.

Onglyza is manufactured by Bristol-Myers Squibb Co. of Princeton, N.J., and marketed by Bristol-Myers and AstraZeneca Pharmaceuticals LP, of Wilmington, Del.

Tags: Onglyza, saxagliptin, Type 2 Diabetes, FDA Approval, dipeptidyl peptidase-4, Bristol-Myers Squibb

First Ablation Catheters for the Treatment of Atrial Fibrillation Approved By FDA

The U.S. Food and Drug Administration today approved the first ablation catheters for the treatment of atrial fibrillation (uncoordinated contractions of the upper heart chambers), one of the most common types of arrhythmias—or abnormal heart rhythms--affecting more than two million Americans.

The devices approved today, the NaviStar ThermoCool saline irrigated radio-frequency ablation catheter and the EZ Steer ThermoCool Nav, can be used to create small, strategically placed scars in heart tissue to block irregular electrical waves that cause atrial fibrillation. The FDA previously approved other ablation catheters to treat arrhythmias such as atrial flutter and ventricular tachyarrhythmia, but not atrial fibrillation.

Atrial fibrillation is usually treated with drugs, and in certain severe cases, with open heart surgery. Catheter ablation should be used only after drug treatment has failed to adequately control the symptoms of the condition.

“This approval provides physicians with another option for treating this common and potentially debilitating condition,” said Daniel G. Schultz, M.D., director of the FDA’s Center for Devices and Radiological Health.

While atrial fibrillation is a major risk factor related to stroke, there is no conclusive evidence that links the treatment of symptoms by ablation to a reduction in stroke. Therefore, the FDA agrees with the American College of Cardiology, the American Heart Association and the European Society of Cardiology, which recommend that patients at risk for stroke continue to take blood-thinning medications after ablation procedures for atrial fibrillation.

The FDA based this approval on a clinical study of 167 patients at 19 medical centers in the United States, Brazil, Canada, the Czech Republic, and Italy. Data from the study showed the devices to be effective in eliminating symptomatic recurrence of atrial fibrillation episodes for one year in approximately 63 percent of treated patients versus 17 percent of the patients in the control group.
The catheters were the focus of an FDA Circulatory System Devices advisory panel on Nov. 20, 2008, when they received the panel’s unanimous recommendation for approval.

As a condition of approval, manufacturer BioSense Webster must establish a physician training program and conduct postmarket studies to collect data on these devices’ long-term safety and effectiveness (including incidence of stroke, mortality, cardiac arrest, major bleeding, and pulmonary vein stenosis), and the effect of physicians’ experience in operating the device on procedural safety.
Both catheters are manufactured by BioSense Webster of Diamond Bar, Calif.

Tags: FDA Approval, ablation catheters, atrial fibrillation, NaviStar ThermoCool saline irrigated radio-frequency ablation catheter, EZ Steer ThermoCool Nav, BioSense Webster of Diamond Bar

DNA Test to Measure Hepatitis B Virus Levels Approved By FDA.

The U.S. Food and Drug Administration today approved the first nucleic acid test for hepatitis B virus (HBV) that measures the amount of viral DNA (viral load) in a patient’s blood. Assessing a patient’s viral load provides health care professionals with a highly sensitive method for gauging the progress of antiviral therapy in patients with chronic HBV infections.

The COBAS TaqMan HBV Test extracts and then amplifies sections of viral DNA from human plasma or serum. The viral DNA sections are measured to establish a baseline level before beginning treatment, and then used again during treatment to assess an individual’s response to therapy. (The baseline level of hepatitis B virus should decrease with successful treatment.) The test is used with other clinical findings, such as results from biochemical and serological testing.

“Measuring a patient’s HBV viral load is an important aspect of managing chronic hepatitis B infections,” said Daniel G. Schultz, M.D., director of FDA’s Center for Devices and Radiological Health. “The COBAS TaqMan test gives health care providers a new and sensitive tool for this process.”

HBV is the most serious type of viral hepatitis, infecting about two billion people worldwide each year, according to the World Health Organization. A vaccine for HBV has been available in the United States since 1982. However, according to the Centers for Disease Control and Prevention, about 1.25 million people in the United States have chronic hepatitis B. Another 60,000 become infected each year and some 5,000 die from hepatitis B-related complications.

HBV is spread through sexual exposure, use of infected needles, and transmitted from infected mother to child during birth. Symptoms occur in about 70 percent of patients, and include abdominal pain, jaundice, fatigue, loss of appetite, nausea, and vomiting.

COBAS TaqMan HBV Test is manufactured by Roche Diagnostic Division, Basel, Switzerland.

Tags: FDA Approval, Drug Approvals, nucleic acid test for hepatitis B virus, viral DNA, COBAS TaqMan HBV Test, World Health Organization, Roche Diagnostic Division

First Bone Marrow Stimulator to Treat Immune-Related Low Platelet Counts Approved By FDA.

FOR IMMEDIATE RELEASE August 22, 2008

Media Inquiries: Karen Riley, 301-827-6244 Consumer Inquiries: 888-INFO-FDA

FDA Approves First Bone Marrow Stimulator to Treat Immune-Related Low Platelet Counts

The U.S. Food and Drug Administration today approved Nplate (romiplostim), the first product that directly stimulates the bone marrow to produce needed platelets in patients with a rare blood disorder that can lead to serious bleeding.

The condition, which usually develops in adults, is known as chronic immune thrombocytopenic purpura (ITP), a disease that results in a low number of platelets, the blood components that help with clotting. In patients with chronic ITP, the immune system is believed to destroy platelets and the patient's bone marrow is often unable to compensate for this loss.

"This product is important in that it offers a new approach to the treatment of patients with an uncommon blood disorder who are often very ill," said Janet Woodcock, M.D., director, Center for Drug Evaluation and Research, FDA.

The estimated 140,000 people with chronic ITP are prone to bruising and at risk for life-threatening bleeding. Current medical treatment includes corticosteroids and immunoglobulin. Surgery to remove the spleen, a procedure known as a splenectomy, may help some patients. Nplate is approved only for patients with chronic ITP who do not respond sufficiently to current treatments.

FDA based its approval on two randomized clinical trials of about 125 patients who had received at least one prior ITP treatment. One study enrolled patients who still had their spleen, the other enrolled patients who did not.

During six months of treatment, patients who received Nplate had significantly higher platelet counts and maintained those higher counts compared to those who did not receive the drug. The response to Nplate was higher in those patients who still had their spleen than in those patients who had undergone a splenectomy. In those patients who did not receive Nplate, only one experienced a sustained increase in platelet counts.

Safety concerns with Nplate include fibrous deposits in the bone marrow and the possibility that once Nplate is stopped, platelet counts could drop below what they were before beginning treatment.

Additional risks include blood clots due to excessive increases in platelets and, if Nplate were given to patients with an abnormal blood condition known as myelodysplasia, a risk for a form of blood cancer known as acute leukemia. Myelodysplasia, which is associated with low platelet counts, predisposes some patients to leukemia. In a study of 44 patients who had myelodysplasia and received Nplate, four patients developed leukemia. Further clinical trials in patients with predisposing conditions for leukemia will be needed to determine whether the development of leukemia may relate to the use of Nplate. Nplate is approved only for use among patients with chronic ITP.

A Risk Evaluation and Mitigation Strategy (REMS) has been developed to address the risks of Nplate therapy. Under the Food and Drug Administration Amendments Act of 2007, FDA has determined that a REMS is necessary for the benefits of Nplate to outweigh the risks of the product. The REMS will include a Medication Guide for patients and requires that all prescribers and patients enroll in a special program to track the long term safety of Nplate therapy.

Nplate is manufactured by Amgen, Inc. of Thousand Oaks, Calif.

Tags: FDA Approval, FDA News, Bone Marrow Stimulator, Nplate, romiplostim, corticosteroids, immunoglobulin, splenectomy, myelodysplasia, Amgen, Inc

FDA approves an expanded label for CANCIDAS®, The first and only echinocandin therapy approved in the United States for pediatric patients with fungal infections.

WHITEHOUSE STATION, N.J., Aug. 5, 2008 - Merck & Co., Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved an expanded label for CANCIDAS®, which makes it the first and only echinocandin therapy approved in the United States for the treatment of pediatric patients aged 3 months to 17 years with indicated fungal infections.

In the United States, CANCIDAS is now indicated in adults and pediatric patients (3 months and older) for:

• Empirical therapy for presumed fungal infections in febrile, neutropenic patients
• Treatment of Candidemia and the following Candida infections: intra-abdominal abscesses, peritonitis and pleural space infections. CANCIDAS has not been studied in endocarditis, osteomyelitis and meningitis due to Candida
• Treatment of Esophageal Candidiasis
• Treatment of Invasive Aspergillosis in patients who are refractory to or intolerant of other therapies (i.e., amphotericin B, lipid formulations of amphotericin B, and/or itraconazole). CANCIDAS has not been studied as initial therapy for invasive aspergillosis.

The efficacy and safety of CANCIDAS has not been adequately studied in prospective clinical trials involving neonates and infants under 3 months of age.
"In the pediatric population, safety and tolerability can be especially critical. With the expanded label for CANCIDAS that now includes use in pediatric patients aged 3 months to 17 years, clinicians have a new option – with both a demonstrated efficacy and safety profile – for treating these pediatric patients with indicated fungal infections," said Dr. Theoklis Zaoutis, assistant professor, Pediatrics and Epidemiology, Division of Infectious Diseases, Children's Hospital, Philadelphia, and lead study investigator.
The expanded label for CANCIDAS is based on data from five prospective clinical studies involving 171 pediatric patients. Two clinical trials demonstrated the efficacy of CANCIDAS for the treatment of pediatric patients with indicated fungal infections, and demonstrated a safety and tolerability profile comparable to that of adults. The expanded label also includes consistent dosing for pediatric patients across all indications based on multiple pharmacokinetic studies.

As part of the expanded label, precise dosing of CANCIDAS in pediatric patients aged 3 months to 17 years should be based on the patient's body surface area (BSA). For all indications, a single 70-mg/m² loading dose (not to exceed an actual dose of 70 mg) should be administered on Day One, followed by 50 mg/m² daily thereafter (not to exceed an actual dose of 70 mg daily).² CANCIDAS is the only antifungal with well-studied BSA dosing in pediatric patients.
Safety and efficacy of CANCIDAS evaluated when used as empirical therapy
In one clinical trial, the safety and efficacy profile of CANCIDAS was assessed when used as empirical therapy against suspected fungal infections in patients 2 to 17 years of age with persistent fever and neutropenia (seriously ill patients with persistent fever and low white blood cell counts).

The study was a prospective randomized, double-blind, multi-center, comparative trial involving a total of 82 patients aged 2 to 17 years. Patients were randomized using a 2:1 ratio of CANCIDAS (n= 56) to AmBisome® liposomal amphotericin B, or AmBisome (n=26), a standard treatment option against invasive fungal infections, and were given either CANCIDAS 50 mg/m² daily following a 70 mg/m² loading dose on Day One (not to exceed 70 mg daily) or AmBisome 3 mg/kg daily. Patients were stratified based on risk category (high-risk patients had undergone allogeneic stem cell transplantation or had relapsed acute leukemia). Twenty-seven percent of patients in both treatment groups were high risk.

The study design and criteria for overall favorable response were similar to the corresponding study in adult patients. An overall favorable response required meeting each of the following criteria: no documented breakthrough fungal infections up to seven days after completion of treatment, survival for seven days after completion of study therapy, no discontinuation of the study drug because of drug-related toxicity or lack of efficacy, resolution of fever during the period of neutropenia, and successful treatment of any documented baseline fungal infection.
The favorable overall response rates were 46.4 percent (26 of 56) in the CANCIDAS group versus 32.0 percent (8 of 25) in the AmBisome group. The favorable response rates for high-risk patients were 60 percent (9 of 15) in the CANCIDAS group versus 0 percent (0 of 7) in the AmBisome group. The favorable response rates for low-risk patients were 41.5 percent (17 of 41) in the CANCIDAS group versus 44.4 percent (8 of 18) in the AmBisome group. Fewer patients experienced serious drug-related adverse events with CANCIDAS (0.6 percent; n=1) than AmBisome (11.5 percent; n=3). The therapy discontinuation rate due to drug-related adverse reaction was 1.2 percent with CANCIDAS (n=2) and 11.5 percent with AmBisome (n=3).
Efficacy and safety studied in documented fungal infections
Another prospective, open-label, non-comparative study estimated the safety and efficacy of CANCIDAS in pediatric patients aged 3 months to 17 years with candidemia and other Candida infections, esophageal candidiasis, and invasive aspergillosis (as salvage therapy).
The study design and criteria for favorable response were similar to the corresponding studies in adult patients. A favorable response for patients with candidemia and other Candida infections required both symptom/sign resolution/improvement and microbiological clearance of the Candida infection. Favorable overall response at five to seven days following discontinuation of study therapy for patients with esophageal candidiasis required both complete resolution of symptoms and significant endoscopic improvement. A favorable response for patients with invasive aspergillosis was defined as either complete resolution (complete response) or clinically meaningful improvement (partial response) of all signs and symptoms and attributable radiographic findings.
The efficacy of CANCIDAS in pediatric patients with these documented fungal infections was supported by the study results. Specifically, the favorable response rates were 81 percent (30 of 37) of patients with candidemia and other Candida infections, 100 percent (1 of 1) of patients with esophageal candidiasis, and 50 percent (5 of 10) of patients with invasive aspergillosis as salvage therapy.
Selected important risk information about CANCIDAS
CANCIDAS is contraindicated in patients with hypersensitivity to any component of the product. Concomitant use of CANCIDAS with cyclosporine should be limited to patients for whom the potential benefit outweighs the potential risk of increased hepatic enzyme abnormalities.
Laboratory abnormalities in liver function tests have been seen in healthy volunteers and patients treated with CANCIDAS. In some patients with serious underlying conditions who were receiving multiple medications concomitantly with CANCIDAS, isolated cases of clinically significant hepatic dysfunction, hepatitis, or hepatic failure have been reported; a causal relationship to CANCIDAS has not been established. Patients who develop abnormal liver function tests during therapy with CANCIDAS should be monitored for evidence of worsening hepatic function and evaluated for risk/benefit of continuing therapy with CANCIDAS. There is no clinical experience in adult patients with severe hepatic insufficiency (Child-Pugh score >9) and in pediatric patients with any degree of hepatic insufficiency.

Possible histamine mediated symptoms have been reported, including rash, facial swelling, pruritus, sensation of warmth and bronchospasm. Anaphylaxis has been reported during administration of CANCIDAS.
The most common adverse reactions in adult patients treated with CANCIDAS (greater than or equal to 10 percent), irrespective of causality, are: diarrhea, pyrexia, chills, alanine aminotransferase/aspartate aminotransferase (ALT/AST) increase, blood alkaline phosphatase increase and decrease of blood potassium.
The most common adverse reactions in pediatric patients treated with CANCIDAS, irrespective of causality, were pyrexia (29.2 percent), blood potassium decreased (15.2 percent), diarrhea (14.0 percent), increased AST (11.7 percent), rash (11.7 percent), increased ALT (11.1 percent), hypotension (11.1 percent), and chills (11.1 percent). The proportion of patients who experienced an infusion-related adverse reaction was 21.6 percent in the group treated with CANCIDAS and 34.6 percent in the group treated with AmBisome.

When CANCIDAS is co-administered to pediatric patients with inducers of drug clearance, such as rifampin, efavirenz, nevirapine, phenytoin, dexamethasone, or carbamazepine, a dose of CANCIDAS of 70 mg/m² daily (not to exceed 70 mg) should be considered. For patients receiving CANCIDAS and tacrolimus, standard monitoring of tacrolimus blood concentrations and appropriate tacrolimus dosage adjustments are recommended.
The efficacy and safety of CANCIDAS has not been adequately studied in prospective clinical trials involving neonates and infants under 3 months of age. Although limited pharmacokinetic data were collected in neonates and infants below 3 months of age, these data are insufficient to establish a safe and effective dose of caspofungin in the treatment of neonatal candidiasis. Invasive candidiasis in neonates has a higher rate of central nervous system and multi-organ involvement than in older patients; the ability of CANCIDAS to penetrate the blood-brain barrier and to treat patients with meningitis and endocarditis is unknown.
For more details about CANCIDAS, please see the prescribing information.
About CANCIDAS
CANCIDAS is a member of the echinocandin class of antifungals. CANCIDAS inhibits the synthesis of (1,3)-D-glucan, an integral component of the fungal cell wall. CANCIDAS should be administered via slow intravenous infusion over approximately one hour. CANCIDAS should not be administered by I.V. bolus administration.

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Tags: Drug Approvals, FDA Approval, pediatric patients, CANCIDAS, echinocandin therapy, Candidemia, Aspergillosis

NeuRx DPS RA/4 Respiratory Stimulation System Approved By FDA

FOR IMMEDIATE RELEASE June 18, 2008

Media Inquiries: Peper Long, 301-827-0599 Consumer Inquiries: 888-INFO-FDA

FDA Approves Diaphragm-Pacing Device
Device can help paralysis patients breathe without a ventilator for at least four hours

The U.S. Food and Drug Administration today announced that it approved the NeuRx DPS RA/4 Respiratory Stimulation System, an implantable electronic device that stimulates the diaphragm and allows certain spinal cord injury patients to breathe for at least four hours a day without a mechanical ventilator.

Spinal cord injuries can cause paralysis, which can impact the muscles of the chest and abdomen, including the diaphragm—the lower abdominal muscle essential for breathing. Normally, a person inhales when the diaphragm contracts and the lungs expand with air—a person exhales when the diaphragm relaxes and the air flows back out of the lungs.

"While the NeuRx RA/4 does not cure paralysis of the diaphragm, allowing patients to be free from a mechanical ventilator for at least four hours a day may enhance their quality of life," said Daniel G. Schultz, M.D., director of the FDA's Center for Devices and Radiological Health.

Patients with severe spinal cord injuries who cannot control their diaphragms often need mechanical ventilation to help them breathe. This usually requires a full-time connection to a ventilation machine.

The NeuRx DPS RA/4 uses four electrodes implanted in the muscle of the diaphragm to electronically stimulate contraction; this stimulation allows the patient to inhale.

The FDA approved the distribution of this device under a Humanitarian Device Exemption, an approval process for devices intended to treat or diagnose conditions that affect fewer than 4,000 people per year.

In a multi-center trial, the device has been demonstrated to be safe and to have probable benefit to the patient by allowing at least four hours per day of freedom from a mechanical ventilator.

NeuRx DPS RA/4 is manufactured by Synapse Biomedical of Cleveland, Ohio.

Tags: FDA Approval, Medical Devices, Diaphragm-Pacing Device, Respiratory Stimulation System, NeuRx DPS RA/4, Synapse Biomedical of Cleveland

New Formulation of Coagulation Therapy, NovoSeven RT Approved by FDA

FOR IMMEDIATE RELEASE May 9, 2008

Media Inquiries: Peper Long, 301-827-6242 Consumer Inquiries: 888-INFO-FDA

FDA Approves New Formulation of Coagulation Therapy
NovoSeven RT Can Be Stored at Room Temperature

The U.S. Food and Drug Administration today approved a new formulation of the genetically engineered version of Factor VIIa, a plasma protein essential for the clotting of blood. The new formulation allows the product to be stored at room temperature (up to 77 degrees Fahrenheit) for up to two years.

"Approval of this product for room temperature storage creates greater flexibility in disease management for both patients and physicians," said Jesse L. Goodman, M.D., M.P.H., director of the FDA’s Center for Biologics Evaluation and Research. "As with all FDA-approved products, the agency will monitor NovoSeven RT throughout its life cycle."

NovoSeven RT—the new formulation of NovoSeven Coagulation Factor VIIa (Recombinant)—contains sucrose and L-Methionine, which allow for storage at room temperature. This is helpful for health-care facilities with limited refrigerated space. The original formula could be stored for three years at temperatures between 36 and 46 degrees Fahrenheit.

NovoSeven RT shares the same uses as the earlier NovoSeven product. These uses include the treatment of bleeding and the prevention of surgical bleeding in patients with hemophilia A or B, who have antibodies that neutralize the action of clotting Factors VIII or IX; the treatment of bleeding and the prevention of surgical bleeding in patients with congenital Factor VII deficiency; and the prevention of surgical bleeding in patients with acquired hemophilia.

Coagulation factors are proteins found in plasma, which help blood clot. When one or more of these proteins are missing or inactive, bleeding can occur.

The most commonly observed adverse reactions with NovoSeven RT are fever, bleeding, injection site reaction, joint discomfort, headache, elevations or falls in blood pressure, nausea, vomiting, pain, swelling, and rash. Some elderly patients experienced an increased risk of arterial clotting when they were treated with NovoSeven RT outside of its approved indications.

NovoSeven RT and NovoSeven Coagulation Factor VIIa (Recombinant) are manufactured by Novo Nordisk A/S, located in Denmark.

Tags: FDA Approval, Coagulation Therapy, Factor VIIa, plasma protein, NovoSeven RT

Generic (Requip,)Ropinirole, For Treatment of Restless Legs Syndrome Approved By FDA

The U.S. Food and Drug Administration has approved the first generic versions of Requip (ropinirole hydrochloride) tablets for the treatment of moderate to severe Restless Legs Syndrome.

Ropinirole hydrochloride tablets have been approved in the following dosages: 0.25 milligram, 0.5 milligram, 1 milligram, 2 milligram, 3 milligram, and 4 milligram.

"This generic drug approval is an example of the FDA's efforts to increase access to safe and effective generic drugs as soon as the law permits," said Gary J. Buehler, director of the FDA's Office of Generic Drugs.

The following companies have received approval to market ropinirole hydrochloride tablets: Roxane Laboratories Inc., Teva Pharmaceuticals USA, Par Pharmaceuticals Inc., and Mylan Pharmaceuticals Inc.

The labeling of the generic versions of ropinirole hydrochloride may differ from that of Requip because some uses of the drug are protected by patents. In addition to treating Restless Legs Syndrome, Requip is also FDA-approved to treat symptoms of Parkinson's disease. The generic products are not approved for treatment of Parkinson's disease because this indication is protected by patent. Manufacturers of the generic drugs may seek approval for that use once the patent for the Parkinson's disease indication expires later this month.

The generic ropinirole hydrochloride tablets will have the same safety warnings as Requip, cautioning about patient reports of falling asleep while engaged in activities of daily living, including while driving. Although many of these patients reported sleepiness while on the drug, some patients perceived that they had no warning signs and believed that they were alert immediately prior to falling asleep. Some of these events have been reported as late as one year after the start of treatment.

For information:

Drugs@FDA
www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm

Consumer Education: Generic Drugs
www.fda.gov/cder/consumerinfo/generic_text.htm

GridTags: FDA Approval, Drug Approval, Requip, ropinirole hydrochloride, Restless Legs Syndrome, RLS, Roxane Laboratories Inc., Teva Pharmaceuticals USA, Par Pharmaceuticals Inc., Mylan Pharmaceuticals Inc