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BioMarin (BMRN) Gets EMA Validation for Hemophilia Gene Therapy

BioMarin Pharmaceutical Inc. BMRN announced that the European Medicines Agency (“EMA”) has validated its marketing authorization application (“MAA”) seeking approval for its investigational gene therapy, valoctocogene roxaparvovec, for treating adult patients with severe hemophilia A.

Following this validation, the review of the MAA for valoctocogene roxaparvovec can finally begin. An opinion from the EMA’s Committee for Medicinal Products for Human Use (“CHMP”) is expected in the first half of 2022.

The MAA filing includes safety and efficacy data from the phase III GENEr8-1 study which evaluated valoctocogene roxaparvovec in 134 subjects, along with four- and three-year follow-up data from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, of the ongoing phase I/II dose escalation study.

Last month, BioMarin re-submitted the MAA for valoctocogene roxaparvovec to the European Commission. The company had withdrawn the MAA for valoctocogene roxaparvovec in the EU last year.

In May 2021, the EMA granted accelerated assessment for the review of valoctocogene roxaparvovec. The accelerated assessment is likely to reduce the time period for the EMA’s CHMP and Committee for Advanced Therapies to review the MAA for valoctocogene roxaparvovec from 210 days to 150 days.

Share of BioMarin have plunged 9.3% so far this year compared with the industry’s decrease of 4.1%.


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We remind investors that in August last year, the FDA issued a complete response letter (“CRL”) to valoctocogene roxaparvovec’s biologics license application (“BLA”) ahead of the Aug 21 PDUFA date as it was not satisfied with the available data. The FDA asked for two-year follow-up data on annualized bleed rates from the ongoing phase III GENEr8-1 study in order to have additional evidence of a durable effect. The data is not expected to be available before November 2021. Back then, BioMarin also withdrew its marketing application in the EU.

Investors were expecting the FDA to grant accelerated approval to the drug on the PDUFA date. It was expected that valoctocogene roxaparvovec, if approved, would be a transformational product as it has the potential to dramatically change the treatment paradigm. However, the CRL and the delayed BLA filing have now pushed potential approval of valoctocogene roxaparvovec to 2023, which is a major blow to BioMarin’s prospects. A BLA on the same is expected to be re-filed to the FDA in the second quarter of 2022.

Hemophilia A is a genetic disorder caused by missing or defective factor VIII. Despite being prescribed the current standard-of-care medicines, severe hemophilia A patients persistently experience painful bleeds, thereby creating significant need for medicines that can improve patients' quality of life. Several companies are developing gene therapy products to treat severe hemophilia A.

Sangamo Therapeutics SGMO, along with partner Pfizer PFE, is evaluating their gene therapy candidate, giroctocogene fitelparvovec (SB-525), in a phase III study for hemophilia A. Swiss pharma giant Roche RHHBY and Dutch biotech, uniQure, are also developing gene therapy candidates to treat hemophilia A.

Zacks Rank

BioMarin currently carries a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.


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