BioMarin Pharmaceutical Inc. BMRN announced that the FDA has issued a complete response letter (CRL) to its biologics license application (BLA) for Roctavian (valoctocogene roxaparvovec/valrox), an investigational gene therapy, developed for treating patients with severe hemophilia A.

Shares of the company took a massive hit from this news on Wednesday as the stock shed 35.3% of value. In fact, so far this year, the stock has lost 9.3% against the industry’s increase of 3.9%.

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 is not satisfied with the available data and needs more information before it can approve the product.Per the regulatory agency, the differences between the phase I/II and the phase III analyses limited its ability to rely on the results from the phase I/II alone to support durability of effect.

According to management, the FDA introduced a recommendation in the CRL. The regulatory body requires BioMarin to provide substantial proof of a durable effect from the ongoing phase III study (270-301) using Annualized Bleeding Rate (ABR) as the primary endpoint.

The FDA authorities asked BioMarin to complete the phase III study and submit two-year follow-up safety and efficacy data on all participants from the program. The study is fully enrolled and the last patient in the same will complete two years of follow-up in November 2021. This could push the potential approval of Roctavian to 2022.

The CRL from the FDA is a massive setback for the company as this was the first marketing application submission for a gene therapy product for any type of hemophilia in the United States. BioMarin expects Roctavian to be transformational as it has the potential to dramatically change the treatment paradigm.

The company plans to meet the FDA officials in the coming weeks for deciding on the next plan of action to gain an approval for Roctavian.

The FDA already granted a priority review and Breakthrough Therapy plus Orphan Drug designations to valoctocogene roxaparvovec for severe hemophilia A.

Meanwhile, in Europe, the marketing application for valoctocogene roxaparvovec remains under accelerated assessment. However, BioMarin has stated earlier, that the review process in the EU got extended by at least three months due to the ongoing COVID-19 pandemic. Therefore, an opinion from the CHMP is expected in late 2020 or early 2021.

Hemophilia A is a genetic disorder caused by the missing or defective factor VIII, a clotting protein. Despite being prescribed the current standard of care medicines, patients with severe hemophilia A persistently experience painful bleeding, thereby signalling an urgent need for medicines that can improve the quality of life of those affected. Several companies are developing gene therapy products to treat severe hemophilia A.

Notably, Sangamo Therapeutics, Inc. SGMO along with partner Pfizer, Inc. PFE is also evaluating their gene therapy candidate SB-525 in a phase III study for severe hemophilia A. Other companies developing gene therapy candidates to that end include Roche and uniQure QURE.

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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