The US Food and Drug Administration (FDA) has accepted Sanofi’s efanesoctocog alfa (BIVV001) Biologics License Application (BLA), intended to treat haemophilia A, for priority review.
A decision from the regulatory agency on approval for the therapy is anticipated on 28 February next year.
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The application is based on findings from the open-label, non-randomised, interventional Phase III XTEND-1 clinical trial designed to evaluate the safety, efficacy and pharmacokinetics of efanesoctocog alfa given once a week in subjects aged 12 years or above.
The trial enrolled 159 severe haemophilia A patients who had previously received factor VIII replacement therapy.
According to the trial findings, the treatment offered clinically meaningful bleeding prevention and was superior to previous factor prophylaxis.
Additionally, efanesoctocog alfa was found to be well-tolerated, and no inhibitor development to factor VIII was reported.
Headache, arthralgia, fall and back pain were the most frequent treatment-emergent adverse events seen in the trial.
A new and investigational recombinant factor VIII therapy, efanesoctocog alfa is developed under a partnership between Sanofi and Sobi.
It can potentially increase bleeding protection in haemophilia A patients with once-a-week prophylactic dosing.
Sanofi Development global head and chief medical officer Dietmar Berger said: “The results from the pivotal XTEND-1 Phase III study demonstrate efanesoctocog alfa’s ability to reduce annualised bleeding rates, which supports its potential as therapy with best-in-disease efficacy.
“We look forward to working closely with the FDA during the review process as we aim to bring this novel therapy to the haemophilia A community.”
On reporting findings from the XTEND-Kids paediatric trial underway, the company will make a regulatory submission to the EU next year.
The latest development comes after the company signed a research partnership with Atomwise to use the latter’s artificial intelligence (AI)-driven AtomNet platform to discover and research up to five drug targets.
