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FDA has approved Cablivi in combination with the plasma exchange.

FDA Cleared Sanofi’s First Therapy for Rare-blood Disorder

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The U.S. Food and Drug Administration (FDA) has approved Sanofi’s therapy called Cablivi for a rare blood clotting disorder, acquired thrombocytopenic purpura (aTTP).

aTTP is a rare, life-threatening autoimmune blood disorder. It involves the extensive formation of blood clots in blood vessels and is considered to be an urgent medical need.

Cablivi is developed by Ablynx, a Sanofi company. Sanofi is a biopharmaceutical company that focuses on human health. Sanofi provides vaccines and treatments for illnesses and aims to help people fight from rare diseases.

Cablivi is known to be the first medicine that is approved in the U.S. for such a rare disorder. FDA has approved this in combination with the plasma exchange. Also, it will be combined with the immune suppression for the treatment of aTTP in adults.

This new drug by Sanofi targets von Willebrand factor (vWF) during the treatment. vWF is a protein in the blood. The therapy constrains the interface between vWF and platelets.

Olivier Brandicourt, M.D., Chief Executive Officer, Sanofi said, “The U.S. approval of Cablivi provides a much-needed treatment option for people facing this challenging disease. There have been limited medicines available to treat aTTP until now.”

Brandicourt further added, “Cablivi marks the first U.S. approval in our newly formed rare blood disorders franchise, and we look forward to continuing to provide important medicines for people living with these very serious diseases.”

Spero R. Cataland, M.D., Professor of Internal Medicine, Division of Hematology, Wexner Medical Center at the Ohio State University remarked, “aTTP is a very severe, life-threatening disease. For those faced with this rare diagnosis, the treatment and care can be difficult and the threat of recurrence is ever-present.”

Cataland further added, “Cablivi provides new hope for adults in the U.S. suffering with aTTP and provides a much-needed treatment option to help effectively manage aTTP episodes.”

According to the reports, Cablivi is the US’s first anti-vWF Nanobody medicine to be approved. Cablivi received FDA Fast Track designation after an evaluation under significant review. This review is reserved for those medicines that provide major improvements in care or effectiveness while treating serious diseases.

The Cablivi therapy was approved in the European Union in August 2018. It is the part of the rare blood disorders franchise within Sanofi Genzyme, which is a specialty care global business unit of Sanofi.

The report highlights that up to 20 percent of patients die from aTTP episodes. This happens despite the available treatments. Most of the deaths occur within 30 days of the diagnosis of disease.

In the aTTP disease, vWF accumulate to cause the formation of an extensive clot in small blood vessels in the body. This leads to a severe loss in platelet count, red blood cells, and low blood supply in body parts.

For few aTTP patients, major rescue measures are required. The prompt results of the treatment are not possible. Patients are required to get the routine treatment in the intensive care units especially in the initial days of aTTP diagnosis.

Cablivi medicine is likely to be available in the U.S. by the end of the first quarter. The U.S. wholesale acquisition cost for treating the aTTP occurrence through Cablivi is approximately $270,000 (approx 1.9 crores).

Sanofi is dedicated to aiding the patients who are treated with Cablivi for their medication. The company will be launching Cablivi Patient Solutions as well. It is a complete patient support program. This program will be providing financial help to needy patients.

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