FDA approves once-weekly hemophilia drug from Sobi and Sanofi

USA — The U.S. Food and Drug Administration (FDA) has granted approval for a new hemophilia treatment co-developed by Sanofi and Sobi that has the potential to offer greater convenience for certain patients.

The treatment, known as Altuviiio, is indicated for both the prevention and treatment of bleeding episodes in adults and children with hemophilia A, which is the more prevalent type of this rare blood disorder.

In hemophilia A, genetic mutations hinder the production of Factor VIII, a vital blood-clotting protein.

While many individuals with this condition receive laboratory-engineered Factor VIII protein via intravenous infusions two to three times weekly, Altuviiio is designed to last longer, thereby enabling weekly dosing.

Altuviiio was designed by incorporating essential features from von Willebrand factor, a critical protein for blood clotting.

This protein binds with Factor VIII, aiding in its transportation and protection from breakdown by the body.

To further slowdown the degradation of the molecule, scientists incorporated two “XTEN” polypeptides into Altuviiio.

Collectively, these attributes permit the drug to maintain higher levels of clotting protein for more extended periods.

In the clinical trial that resulted in Altuviiio’s approval, the average Factor VIII levels remained above 40% for most of the week between doses.

Notably, only individuals with severe hemophilia A participated in the trial, and they typically have less than 1% of the Factor VIII levels observed in people without the disease.

Regarding treatments for hemophilia, there are two types: factor therapies, such as Altuviiio, and non-factor therapies, with gene therapy being an emerging option.

CSL and uniQure’s approval for Hemgenix in hemophilia B demonstrates this. Sanofi has announced that Altuviiio is expected to be commercially available in April.

Five big players on the bleeding disorder treatment market, including Roche and Novo Nordisk, will be facing more competition as a new hemophilia A drug has just been approved in the U.S.

Sanofi and Sobi have been involved in hemophilia treatments for some time. Sobi and Biogen collaborated on Eloctate, a treatment for hemophilia A that required less frequent injections.

Sanofi later acquired the drug when it bought Bioverativ, a Biogen spinout specializing in hemophilia, for US$11.6 billion in 2018.

Hemlibra, developed by Roche, became a strong competitor to Eloctate, leading to a decline in its sales. Another competitor in the market is BioMarin’s Roctavian, which has a Prescription Drug User Fee Act (PDUFA) date of March 31.

Sanofi is not just focused on Altuviiio, but is also working on developing fitusiran, a non-factor therapy that could revolutionize treatment for both hemophilia A and B.

The company plans to submit Altuviiio to regulators in the EU in the second half of 2023, according to a statement by Sanofi.

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