2022-06-02| Trials & Approvals

Sanofi and Sobi Receive Breakthrough Therapy Designation for Hemophilia A Drug

by Reed Slater
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Sanofi, the Paris-based pharmaceutical giant, and its Swedish partner, Sobi, received news that its hemophilia A targeting drug, efanesoctocog alfa, received breakthrough therapy designation from the FDA, pushing it closer to a potential approval date. With new clinical study data, Sanofi hopes to submit efanesoctocog alfa to the FDA for regulatory approval to market the drug commercially. 


Tough Competition for Hemophilia A Treatment


Hemophilia A is a lifelong, rare blood disorder affecting about 1 in 5,000 males and is exceptionally rare in females. The disorder is characterized by an inability of a person’s blood to clot due to a coagulation factor deficiency. This can result in bleeding episodes that cause pain, joint damage, and even life-threatening hemorrhages. 

Efanesoctocog alfa is a factor VIII therapy which means it creates blood-clotting proteins absent in patients with hemophilia A. Efanesoctocog alfa received orphan drug designation from the FDA in 2017 and fast track designation in 2021. The most recent breakthrough therapy designation is based on data from the Phase 3 EXTEND-1 study, which will also be the basis for Sanofi’s approval submission planned for later this year. 

Even with its momentum behind it, efanesoctocog alfa faces tough competition among long-standing factor VIII therapies on the market. Novo Nordisk’s Novoeight was among the first factor VIII drugs approved to treat hemophilia A in 2013. Since then, several others have followed suit, including, Baxter International’s Obizur in 2014, Genentech’s Hemlibra in 2017, Bayer’s Jivi in 2018, and Wyeth Pharmaceuticals XYNTHA in 2020. Sanofi even owns Eloctate, which was originally approved by the FDA in 2014 under Biogen, however, Sanofi purchased Biogen’s blood disorder-focused company, Bioverativ, in 2018.


Related Article: Sanofi’s Trial to Make Cialis Available Over-the-Counter Meets FDA Hold


Standing Out Among the Rest


With so many available therapies on the market, Sanofi and Sobi had to highlight efanesoctocog alfa’s superior performance over previously approved drugs to meet the criteria for breakthrough therapy designation. To check that box, the two companies say the EXTEND-1 study showed meaningful prevention of bleeds in participants over a 52-week period. Sanofi and Sobi also said that the drug was superior to previous factor VIII replacement therapies in preventing bleed events in intra-patient comparison. 

Global head of research and development at Sanofi, Dr. John Reed, said, “The Breakthrough Therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with hemophilia A by providing higher protection for longer duration. This potential new class of factor VIII therapy represents how we are boldly advancing science to address unmet needs for the hemophilia community.”

Sanofi and Sobi hope to submit efanesoctocog alfa to the FDA for regulatory approval by the end of 2022. They also hope to publish data from EXTEND-Kids, the pediatric branch of the drug, by 2023.

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