Sanofi Gets FDA Approval for Nanobody-Based Therapeutic

February 15, 2019

The drug is approved in the United States specifically for treating acquired thrombotic thrombocytopenic purpura, a rare blood-clotting disorder.

FDA has approved Sanofi’s Cablivi (caplacizumab-yhdp) in combination with plasma exchange and immunosuppression for treating acquired thrombotic thrombocytopenic purpura (aTTP) in adults. The drug is said to be the first FDA-approved therapy specifically indicated for aTTP, Sanofi noted in a Feb. 6, 2019 press release.

Cablivi is an anti-von Willebrand factor (vWF) nanobody and Sanofi’s first Nanobody-brand-based medicine to receive approval in the United States.

“The [US] 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,” said Olivier Brandicourt, MD, CEO, Sanofi, in the press release. “Cablivi marks the first [US] 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.”

Cablivi targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis. It is designed to inhibit the interaction between vWF and platelets.

Nanobodies are a novel class of proprietary therapeutic proteins based on single-domain antibody fragments that contain the unique structural and functional properties of naturally occurring heavy-chain-only antibodies.

Cablivi received fast track designation from FDA and was evaluated under priority review.

Meeting the needs of a rare blood disorder

aTTP is a rare, life-threatening, autoimmune blood disorder where accumulation of ultra-large vWF causes extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), and ischemia (restricted blood supply to parts of the body). It is considered an urgent, medical emergency.

Some patients require resuscitative measures, and the immediate outcome might not be predictable. Patients typically are routinely treated in intensive care units during the first few days following their aTTP diagnosis. It is estimated that up to 20% of patients die from TTP episodes, despite currently available treatments (plasma exchange and immunosuppression), with most deaths occurring within 30 days of diagnosis. In the US, aTTP affects fewer than 2,000 adults each year.

“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,” said Spero R. Cataland, MD, professor of internal medicine, Division of Hematology, Wexner Medical Center at the Ohio State University, in the company press release. “Cablivi provides new hope for adults in the US suffering with aTTP and provides a much-needed treatment option to help effectively manage aTTP episodes.”

Source: Sanofi