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BioMarin (BMRN) Hemophilia Gene Therapy Gets FDA's RMAT Tag

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BioMarin Pharmaceutical Inc.’s (BMRN - Free Report) gene therapy for severe hemophilia A, valoctocogene roxaparvovec, has been given Regenerative Medicine Advanced Therapy (“RMAT”) designation by the FDA.

The RMAT designation was created under the 21st Century Cures Act and is granted to speed up the development and review of regenerative therapies that target serious or life-threatening conditions.

The designation was bestowed on valoctocogene roxaparvovec due to its potential to address unmet need in the severe hemophilia A market. The candidate had earlier won a Breakthrough Therapy Designation from the FDA and orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A.

BioMarin’s shares have declined 12.6% this year compared with the industry’s decrease of 1.6%.



We remind investors that in August last year, the FDA issued a complete response letter (CRL) to BioMarin’s biologics license application (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

However, 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.  One year of follow-up of all patients in the study was announced in January.

With a mean follow-up of 71.6 weeks, data from the phase III study – GENEr8-1 – showed that treatment with a single dose (6e13 vg/kg) of the gene therapy candidate significantly reduced annualized bleeding rate (“ABR”) by 84% and the mean annualized factor VIII (a clotting protein) infusion rate in the rollover population by 99%. Meanwhile, the mean annualized bleed rate was 0.9 in a subset of patients dosed more than two years ago. 

Investors were expecting the FDA to grant accelerated approval to the drug on the PDUFA date of Aug 21 last year. 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 and is a major blow to BioMarin’s prospects. BioMarin also withdrew its marketing authorization application (MAA) in the EU for valoctocogene roxaparvovec. BioMarin plans to re-submit the MAA along with this latest one-year data to the EMA in the second quarter of 2021 and will also share the data with the FDA and discuss with the agency on potentially submitting a BLA with one-year results. The RMAT designation gives some sign of FDA’s faith in the candidate.

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 - Free Report) and its partner Pfizer (PFE - Free Report) 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 - Free Report) and uniQure

Zacks Rank and Stocks to Consider

BioMarin currently carries a Zacks Rank #4 (Sell).

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

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