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.
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Additionally, efanesoctocog alfa was found to be well-tolerated, and no inhibitor development to factor VIII was reported.
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By GlobalDataHeadache, 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.
