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BioMarin (BMRN) Refiles BLA With FDA for Hemophilia Gene Therapy

BioMarin Pharmaceutical Inc. BMRN resubmitted the biologics license application (BLA) to the FDA, seeking approval for valoctocogene roxaparvovec (valrox) gene therapy to treat adult patients with severe hemophilia A.

BioMarin had previously submitted a BLA in 2019 for valrox to address hemophilia A. However, the FDA issued a complete response letter (CRL) to the BLA ahead of the PDUFA date in August 2020. This was due to the regulatory agency’s dissatisfaction with the available data. As a result, it asked for two-year follow-up information on the annualized bleed rates from the GENEr8-1 study only to provide additional evidence of a durable effect.

The refiled BLA is based on two-year outcomes data from the phase III study, GENEr8-1, evaluating Roctavian in patients with hemophilia A. Data from the GENEr8-1 study showed that treatment with valrox led to stable and durable bleed control. Participants who were treated with the therapy achieved significantly reduced annualized bleeding rate and the mean annualized factor VIII (a blood clotting protein) infusion rate. Treatment with gene therapy also demonstrated superiority to the current standard of care, Factor VIII prophylactic therapy.

While resubmissions to the FDA are followed by a six-month review procedure, BioMarin also expects to submit additional data to strengthen its BLA filing. Based on these data submissions, management expects the review procedure to extend by three months.

Shares of BioMarin have fallen 3.7% in the year-to-date period compared to the industry’s 25.2% decline.


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A one-time infusion, valrox is designed to enable the body to produce Factor VIII on its own. Moreover, the patients are also not required to be administered a continued hemophilia prophylaxis, which in other marketed therapies is required.

If valrox is approved by the FDA, it will be the first gene therapy in the United States for hemophilia A. The gene therapy has been granted Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy and orphan drug designations by the FDA.

Last month, the European Commission (EC) granted conditional approval to valrox for treating adult patients with severe hemophilia A. The therapy is being marketed by BioMarin under the trade name Roctavian.

Following the EC decision, Roctavian became the first approved gene therapy for hemophilia A in the European Union (EU). Per the EC, the use of Roctavian carries significant benefits over currently marketed therapies in treating hemophilia A. In fact, the EC has also endorsed the EMA’s recommendation and granted Roctavian orphan drug designation (ODD). Following the grant of ODD, the drug now has a 10-year market exclusivity in the EU.

 

Zacks Rank & Stocks to Consider

BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the overall healthcare sector include Morphic MORF, Sesen Bio SESN and Sanofi SNY. While Morphic and Sesen Bio sport a Zacks Rank #1 (Strong Buy) at present, Sanofi carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Morphic’s 2022 loss per share have narrowed from $3.38 to $1.80. Loss estimates for 2023 have narrowed from $3.91 to $3.62 during the same period. Shares of Morphic have lost 40.9% in the year-to-date period.

Earnings of Morphic beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 48.29%, on average. In the last reported quarter, MORF delivered an earnings surprise of 183.95%.

Estimates for Sesen Bio’s 2023 bottom line have narrowed from 27 cents to 1 cent in the past 60 days. Share prices of Sesen Bio have fallen 52.2% in the year-to-date period.

Earnings of Sesen Bio beat estimates in each of the last four quarters, the average surprise being 89.49%. In the last reported quarter, Sesen Bio delivered an earnings surprise of 61.54%.

In the past 60 days, estimates for Sanofi’s 2022 earnings per share have increased from $4.08 to $4.14. Earnings estimates for 2023 have increased from $4.25 to $4.29 during the same period. Shares of Sanofi have lost 24.2% in the year-to-date period.

Earnings of Sanofi beat estimates in each of the last four quarters, witnessing a surprise of 9.37%, on average. In the last reported quarter, SNY delivered an earnings surprise of 8.24%.


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