FDA Approves Sanofi and Sobi’s Once-Weekly Hemophilia A Med

After Sanofi and Sobi’s Eloctate fell to the wayside, making room for more effective therapies, the duo is back at the Hemophilia A therapy table after the FDA approved Altuviiio (formerly efanesoctocog alfa). The company claims the once-weekly medication delivers normal to near-normal factor activity levels and significantly reduces bleeds compared to its competitors. 

Another Option for Hemophilia A Patients

Hemophilia A is a rare, lifelong condition caused by an inability for blood to clot. This phenomenon can result in excessive bleeds and bleeds in joints which can lead to permanent joint damage and chronic pain. 

Factor VIII protein is vital to creating blood clots, and factor activity levels negatively correlate with a person’s risk of excessive bleeding. Sanofi and Sobi’s Altuviiio got its namesake from its ability to return factor VIII protein levels to normal or near-normal levels. 

The FDA evaluated Altuviiio’s application under its Priority Review program after granting the drug Breakthrough Therapy, Fast Track, and Orphan Drug designation. 

Sanofi CEO, Paul Hudson, said, “Today’s approval of ALTUVIIIO allows patients and physicians to reimagine living with hemophilia. The high sustained factor activity levels that can be achieved with ALTUVIIIO have the potential to change the hemophilia landscape. For the first time, with a once-weekly dose, powerful bleed protection is a reality for patients.”

Related Article: CSL’s Hemgenix Wins First Gene Therapy Approval For Hemophilia In Europe

A Stellar Clinical Trial Performance Supporting the Approval

The FDA made its approval decision based on Sanofi’s XTEND-1 Phase 3 clinical trial, which evaluated Altuviiio’s safety and efficacy in 159 participants. The study reached completion in February last year after researchers studied the drug’s effects in patients after consistent doses over 52 weeks. 

The clinical trial met its primary endpoint of providing significant bleed protection in patients with severe Hemophilia A. The study also met its secondary endpoint by demonstrating a 77% reduction in annualized bleeding rate (ABR) compared to prior factor prophylaxis therapies.

The data also showed that nearly all bleeding episodes resolved after a single Altuviiio injection. Participants reported improved physical health at 52 weeks compared to baseline measurements. 

Sanofi and Sobi’s Altuviiio approval is exciting news in the hemophilia arena, and Sanofi is not stopping there. The French company is working on fitusiran, a non-factor hemophilia therapy, in Phase 3 trials. In the meantime, Sanofi and Sobi can celebrate Altuviiio’s FDA approval while preparing for an EU submission, which Sanofi said it anticipates submitting in the second half of this year. 

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Author

Reed Slater