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BioMarin (BMRN) Hemophilia Gene Therapy Gets EMA's Speedy Review

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the European Medicines Agency (EMA) has granted accelerated assessment for the review of its investigational gene therapy valoctocogene roxaparvovec for treating adult patients with severe hemophilia A.

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 marketing authorization application (“MAA”) for valoctocogene roxaparvovec from 210 days to 150 days.

An opinion from the CHMP is expected in the first half of 2022.

BioMarin plans to re-submit the MAA for valoctocogene roxaparvovec next month. The new submission will have 52 weeks’ data from the phase III GENEr8-1 study, along with four and three years follow-up data from the ongoing phase I/II dose escalation study.

The company had earlier withdrawn the MAA for valoctocogene roxaparvovec in the EU.

Shares of BioMarin have declined 11.6% so far this year compared with the industry’s decrease of 4.4%.

price chart for BMRN

We remind investors that in August 2020, the FDA issued a complete response letter (“CRL”) to BioMarin’s BLA for valoctocogene roxaparvovec. The BLA application was based on interim data from an ongoing phase III study and the updated three-year results from a long-term phase I/II study.

The FDA was not satisfied with the data and asked for two-year follow-up safety and efficacy data on annualized bleed rates from the ongoing phase III 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 in August 2020. 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 now pushes potential approval of valoctocogene roxaparvovec to 2022, which is a major blow to BioMarin’s prospects.

The company is looking to re-submit the BLA for valoctocogene roxaparvovec to the FDA with two-year follow-up safety and efficacy data from the GENEr8-1 study in the second quarter of 2022.

Notably, hemophilia A is a genetic disorder caused by missing or defective factor VIII. Despite being prescribed with the current standard-of-care medicines, severe hemophilia A patients persistently experience painful bleedings, 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 - Free Report) and its partner Pfizer (PFE - Free Report) are evaluating their gene therapy candidate giroctocogenefitelparvovec in a phase III study for hemophilia A. Other companies developing gene therapy candidates to treat hemophilia A include Roche (RHHBY - Free Report) and uniQure.

Zacks Rank

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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