FDA Approves Cablivi (caplacizumab-yhdp) injection by Ablynx, a sanofi company, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP)

FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder

The U.S. Food and Drug Administration today approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening disorder that causes blood clotting.

“Patients with aTTP endure hours of treatment with daily plasma exchange, which requires being attached to a machine that takes blood out of the body and mixes it with donated plasma and then returns it to the body. Even after days or weeks of this treatment, as well as taking drugs that suppress the immune system, many patients will have a recurrence of aTTP,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Cablivi is the first targeted treatment that inhibits the formation of blood clots. It provides a new treatment option for patients that may reduce recurrences.”

Patients with aTTP develop extensive blood clots in the small blood vessels throughout the body. These clots can cut off oxygen and blood supply to the major organs and cause strokes and heart attacks that may lead to brain damage or death. Patients can develop aTTP because of conditions such as cancer, HIV, pregnancy, lupus or infections, or after having surgery, bone marrow transplantation or chemotherapy.

The efficacy of Cablivi was studied in a clinical trial of 145 patients who were randomized to receive either Cablivi or a placebo. Patients in both groups received the current standard of care of plasma exchange and immunosuppressive therapy. The results of the trial demonstrated that platelet counts improved faster among patients treated with Cablivi, compared to placebo. Treatment with Cablivi also resulted in a lower total number of patients with either aTTP-related death and recurrence of aTTP during the treatment period, or at least one treatment-emergent major thrombotic event (where blood clots form inside a blood vessel and may then break free to travel throughout the body).The proportion of patients with a recurrence of aTTP in the overall study period (the drug treatment period plus a 28-day follow-up period after discontinuation of drug treatment) was lower in the Cablivi group (13 percent) compared to the placebo group (38 percent), a finding that was statistically significant.

Common side effects of Cablivi reported by patients in clinical trials were bleeding of the nose or gums and headache. The prescribing information for Cablivi includes a warning to advise health care providers and patients about the risk of severe bleeding.

Health care providers are advised to monitor patients closely for bleeding when administering Cablivi to patients who currently take anticoagulants.

The FDA granted this application Priority Review designation. Cablivi also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Cablivi to Ablynx, a sanofi company..

Octaplas Approved By FDA For Transfusion Or Plasma Exchange In Patients With Thrombotic Thrombocytopenic Purpura (TTP)

Octaplas has been approved for use in patients with certain medical condition who lacks sufficient levels of clotting proteins. This deficiency could lead to severe bleeding or excessive clotting of the blood. This condition is known as Thrombotic Thrombocytopenic Purpura (TTP), which could be congenital or acquired. It is known to have an incidence of 3.8 per million.

“Over more than 20 years, there have been more than 8 million units of Octaplas® transfused internationally, in more than 2.6 million patients, We look forward to bringing Octaplas® to the U.S. medical community. There have been no reports of Octaplas® being associated with TRALI in voluntarily reported adverse events data from all countries where Octaplas® is approved. The incidence of TRALI from plasma transfusion is not well documented, but has been reduced with the introduction of male-only donors in recent years. However, cases are still reported annually through global adverse event reporting systems.” according to Octapharma USA President Flemming Nielsen.

The FDA press release is below and for more information, follow the links after the press release.

FDA approves Octaplas to treat patients with blood clotting disorders

The U.S. Food and Drug Administration today approved Octaplas, a pooled plasma (human) blood product for the replacement of clotting proteins (coagulation factors) in certain medical conditions where patients have insufficient levels. Clotting protein deficiencies can cause excessive bleeding or excessive clotting.

Octaplas is a sterile, frozen solution of pooled human plasma from several donors that has been treated with a solvent detergent process. This process kills certain viruses and thereby minimizes the risk of serious virus transmission. The plasma used to manufacture Octaplas is collected from U.S. donors who have been screened and tested for diseases transmitted by blood, and determined to be suitable donors.

“For patients suffering with clotting disorders, this product provides a viable alternative to single-donor Fresh-Frozen Plasma and provides a reduced risk of certain viral transmissions,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.

Like Fresh Frozen Plasma, Octaplas should be matched to the recipient’s blood group to help avoid transfusion reactions. An additional benefit to Octaplas is that each lot is tested for composition of key clotting factors and is only released if the levels are within acceptable ranges.

Octaplas has been used extensively in Europe and other countries. A previous generation of Octaplas was first marketed in 1992, and the current version has been marketed since 2009. All generations of the product have had similar manufacturing processes and comparable ingredients and properties. In total, more than 2 million patients have been treated with over 7 million doses of Octaplas outside of the United States.

The licensing of Octaplas was primarily based on clinical studies conducted in patients with liver disease, liver transplant, heart surgery and TTP. Additional data supporting the safe use of Octaplas for the U.S. market came from prior use of the products in Europe and other approved markets. Use of the product in Europe was not associated with transfusion-related acute lung injury, an uncommon but serious risk of transfusion with single units of plasma.

The most common adverse reactions observed in clinical studies included shortness of breath, dizziness, chest discomfort, skin itchiness and rashes, headache and tingling sensations.

The product is manufactured by Octapharma, Vienna, Austria.
For Immediate Release: Jan. 17, 2013
Media Inquiries: Rita Chappelle, 301-796-4672, rita.chappelle@fda.hhs.govConsumer Inquiries: 888-INFO-FDA, OCOD@fda.hhs.gov

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