Sanofi, poised to rival Roche in hemophilia, nabs FDA approval for Altuviiio

Sanofi and partner Sobi are coming back to the hemophilia A market with a vengeance after Roche’s Hemlibra ate up sales of their aging Eloctate.

Thursday, the duo scored an FDA approval for hemophilia A treatment Altuviiio almost a week ahead of schedule.

Altuviiio is an investigational factor VIII replacement therapy approved for routine prophylaxis and on-demand treatment to aid in bleeding episodes, plus for surgery management for adult and children. The treatment sets itself apart from the competition thanks to its extended half-life, which is three to four times longer than rival therapies, according to Sanofi.

Current factor VIII products cause levels to go up but decrease quickly, meaning patients have to go in for routine prophylaxis every two days. By contrast, Sanofi is touting Altuviiio's weekly dosing.

Hemophilia A is a rare and lifelong condition that causes patients' blood to not properly clot, leading to external bleeding, bruising and bleeding into joints. Joint bleeding can lead to mobility issues and chronic pain, which is why the long-term outcomes of Altuviiio are key, Dietmar Berger, M.D., Ph.D., chief medical officer and global head of development at Sanofi, said in a recent interview with Fierce Pharma.

The FDA based the drug's approval on results from a phase 3 study called XTEND-1. In the study, Altuviiio demonstrated supriority to prior factor prophylaxis treatment.

Hemophilia A patients are “driving a lot of their own treatment,” Berger said. Annual bleeding rates in the study were determined by patients' diaries, he explained.

Hemophilia A is a $10 billion-plus market, Berger said. As treatments go, there are factor therapies, such as Altuviiio, and non-factor therapies. Gene therapy is another emerging option, as evidenced by CSL and uniQure’s approval for Hemgenix in hemophilia B. 

Analysts from ODDO BHF project the drug's sales will reach €1.3 billion by 2029. Sanofi expects Altuviiio to be commercially available in April, the company said in a statement.

Sanofi and Sobi aren’t new players in the hemophilia game. Sobi and Biogen collaborated on hemophilia A treatment Eloctate, which was the first to tout less frequent injections. That drug later ended up in Sanofi’s hands when the French pharma bought hemophilia specialist Bioverativ, a Biogen spinout, for $11.6 billion in 2018.

Later, Roche’s Hemlibra took a huge bite out of Eloctate sales. Another competitor will be BioMarin’s Roctavian, which has a March 31 PDUFA date.

Sanofi won’t stop with Altuviiio. It’s currently working on developing fitusiran as a non-factor therapy for both hemophilia A and B, which would change the game with a broad mechanism.

The companies plan to submit Altuviiio to regulators in the EU during the second half of 2023, Sanofi said in a company statement.