Pharma, BioPharma

Sanofi’s hemophilia A data set stage for regulatory filings, matchup with Roche

An experimental hemophilia A therapy from Sanofi and partner Sobi has met the goals of a pivotal study, and the pharmaceutical giant is now planning global regulatory submissions for later this year. The drug could compete against a blockbuster hemophilia A treatment marketed by Roche.

 

Sanofi is already a player in the hemophilia A market, but the company is developing another therapy that could better match up against a blockbuster product from rival Roche. The experimental Sanofi therapy has posted encouraging preliminary data in a pivotal study, and the pharmaceutical giant is now planning to submit applications seeking regulatory approvals.

Hemophilia A is a bleeding disorder in which a genetic mutation leads to insufficient amounts of factor VIII, a blood protein that’s important for clotting. Low levels of this protein make it difficult to stop the flow of blood from a wound. Patients who have severe hemophilia A may experience spontaneous bleeding episodes that affect the muscles and joints.

Treatment of severe hemophilia A includes frequent infusions of biological therapies intended to replace factor VIII or its function, providing a way to prevent bleeding episodes. Sanofi already markets one such product, Eloctate. That therapy, approved by the FDA in 2014, was developed for dosing every three to five days. With its experimental hemophilia A treatment, efanesoctocog alfa, Sanofi aims to extend the dosing interval to once a week.

Efanesoctocog alfa is an engineered version of factor VIII. The therapy achieves once-weekly dosing with technology from Amunix that extends how long the therapy lasts in circulation. The clinical trial results announced Wednesday are from a Phase 3 study that enrolled 159 severe hemophilia A patients who were previously treated with factor VIII replacement therapy. The open-label study was designed with two arms. One group received weekly prophylactic doses of the experimental Sanofi therapy for 52 weeks. The other group received “on demand” treatment as needed for 26 weeks, followed by weekly prophylactic treatment for another 26 weeks. The main goal was to measure the annualized bleeding rate in the prophylactic arm.

Sanofi and partner Swedish Orphan Biovitrum (Sobi) reported Wednesday that the median annualized bleeding rate of patients in the study was zero; the average annualized bleeding rate was 0.71. Comparing the experimental treatment to prior prophylactic factor VIII replacement therapy was one of the secondary goals. On this measure, Sanofi reported that its therapy showed a statistically significant reduction in annualized bleeding rates. One complication of factor VIII therapies is an immune response in which the body sees the biological treatment as foreign and produces antibodies against it. Sanofi said its hemophilia A treatment was well tolerated by patients and no antibodies to factor VIII were detected.

With the usual caveats about the limitations of cross-trial comparisons, Sanofi’s experimental hemophilia A therapy posted a better annualized bleeding rate than Eloctate, which accounted for €563 million in sales last year, according to the company’s annual report. But Sanofi may be eying how its experimental hemophilia A therapy matches up against Roche’s Hemlibra. That bispecific antibody, approved by the FDA in 2017, is designed to bind to two clotting proteins to replace the function of factor VIII. Hemlibra was developed for dosing every week, two weeks, or four weeks, depending on patient weight. Roche reported 2021 sales for the drug were 3 billion Swiss francs, a 41% increase over the prior year. The company said that market uptake for the drug is strong, particularly in the U.S. and Europe. However, Hemlibra’s label carries a boxed warning that flag risks such as dangerous cardiovascular problems.

In a prepared statement, Anders Ullman, Sobi’s head of R&D and chief medical officer, said that the Phase 3 results for efanesoctocog alfa will be presented at a future medical meeting. Sanofi and Sobi said that the data will form the basis for regulatory submissions that the company is planning to begin later this year. A filing in the European Union will follow the availability of pediatric study data, which are expected in 2023. Deitmar Berger, Sanofi’s global head of development and chief medical officer, said in a statement that the positive preliminary data show the experimental therapy’s potential to transform hemophilia A therapy.

“We believe efanesoctocog alfa provides higher protection for longer duration with reduced treatment burden of once-weekly dosing, and we look forward to working with regulators to bring this therapy to patients as soon as possible,” Berger said.

Photo: bluebeat76, Getty Images

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