DTMT Network

French pharma giant Sanofi and Stockholm based biopharmaceutical company, Swedish Orphan Biovitrum (Sobi) have jointly announced on June 1, 2022, that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its experimental drug efanesoctocog alfa (BIVV001) for the treatment of people with haemophilia A, a rare and life-threatening bleeding disorder.

In a statement, Sanofi said that the results from the trial demonstrated that efanesoctocog alfa met the primary endpoint, showing clinically meaningful prevention of bleeds in people with severe haemophilia A over a 52-week period.

The company further informed that the key secondary endpoint of the trial was also met, demonstrating that efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy in preventing bleeding events based on an intra-patient comparison.

Commenting on the development, Dr John Reed, the Global Head of Research and Development at Sanofi said, “The Breakthrough Therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with haemophilia A by providing higher protection for a longer duration.”

“This potential new class of factor VIII therapy represents how we are boldly advancing science to address unmet needs for the haemophilia community. We are excited to work with regulatory authorities during the filing and review of this innovative therapy.”

It may be recalled that the drug is jointly developed and commercialised by Sanofi and Sobi, which specialise in rare diseases.

Under the collaboration agreement, while  Sobi has final development and commercialisation rights in Europe, North Africa, Russia and most Middle Eastern markets, Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territories.

“This designation supports the innovation of efanesoctocog alfa and acknowledges its potential to fulfil an unmet medical need for people living with haemophilia A,” Dr Anders Ullman, Head of Research and Development and Chief Medical Officer at Sobi said.

“We are committed to transforming lives for people living with rare diseases, and this is a testament to the medical innovation that science can bring,” he added.

Efanesoctocog alfa is a new experimental investigational recombinant factor VIII therapy that is designed to extend protection from bleeding with once a week prophylactic dosing for people suffering from haemophilia A. It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority, the companies informed.

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