BioMarin Pharmaceutical Inc. BMRN re-submitted the marketing authorization application (“MAA”) for its gene therapy for severe hemophilia A, valoctocogene roxaparvovec to the European Commission.

Last month, the European Medicines Agency (EMA) had granted accelerated assessment for the review of valoctocogene roxaparvovec. The accelerated assessment is likely to reduce the time period for the EMA’s Committee for Medicinal Products for Human Use (“CHMP”) and Committee for Advanced Therapies to review the MAA for valoctocogene roxaparvovec from 210 days to 150 days. An opinion from the CHMP is expected in the first half of 2022.

The new submission includes 52 weeks’ data from the phase III GENEr8-1 study, along with four and three-year follow-up data from the ongoing phase I/II dose escalation study.

Last month, BioMarin presented five-year data for the 6e13 vg/kg dose cohort (n=7) and four-year data for the 4e13 vg/kg dose cohort (n=6) from the ongoing phase I/II study on valoctocogene roxaparvovec. The data showed that all participants in the 6e13 vg/kg and 4e13 vg/kg cohorts remained off prophylactic Factor VIII treatment.

In the high 6e13 vg/kg cohort, the mean annualized bleed rate (ABR) was 0.7 with a 95% reduction in ABR and a 96% reduction in mean Factor VIII usage five years post-treatment. Meanwhile, the mean ABR was 1.7 with a 92% reduction in ABR and a 95% reduction in mean FVIII usage at 4 years post-treatment for the 4e13 vg/kg cohort 

In the year so far, the stock price of BioMarin has declined 3.5% against the industry’s growth of 0.5%.


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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 the FDA was not satisfied with the available data and asked for two-year follow-up data on annualized bleed rates from the ongoing phase III GENEr8-1 study to provide additional evidence of a durable effect. The data is not expected to be available before November 2021.

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 now pushes potential approval of Roctavian to 2023 and is a major blow to BioMarin’s prospects. A BLA is expected to be re-filed to the FDA in the second quarter of 2022. BioMarin also withdrew its MAA in the EU for valoctocogene roxaparvovec.

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 and its partner Pfizer PFE are evaluating their gene therapy candidate, giroctocogene fitelparvovec (SB-525) in a phase III study for hemophilia A. Other companies developing gene therapy candidates to treat hemophilia A include Roche RHHBY and uniQure.

BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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