BioMarin Gets EMA Permit for Accelerated Assessment of Valoctocogene Roxaparvovec

BioMarin Pharmaceutical Inc. (BMRN), a global biotechnology company received the European Medicines Agency (EMA) approval for accelerated assessment of valoctocogene roxaparvovec.

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BioMarin’s Worldwide R&D President Hank Fuchs said, “We continue to work closely with the EMA to make valoctocogene roxaparvovec, the potential first gene therapy to treat hemophilia A, available as soon as possible.”

Patients with hemophilia A lack adequate functioning Factor VIII protein, which helps blood to clot. These people are at risk of painful and significant life-threatening bleeds from even small injuries.

Notably, the permit will reduce the time for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a Marketing Authorization Application (MAA) for an Advanced Therapy Medicinal Product (ATMP). The company expects a CHMP opinion in the first half of 2022.

In June 2021, BioMarin plans to submit an MAA for valoctocogene roxaparvovec, which is designed to treat patients with severe hemophilia A. The application will include data related to the Phase 3 GENEr8-1 study, along with four and three years of follow-up from the respective dose cohorts in the ongoing Phase 1/2 dose escalation study, the company said. (See BioMarin stock analysis on TipRanks)

Additionally, BioMarin plans to submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study in support of the risk assessment of valoctocogene roxaparvovec, which was previously requested by the U.S. Food and Drug Administration (FDA). The company also intends to resubmit a Biologics License Application (BLA) in the second quarter of 2022, with the assumption of favorable study results, which will be followed by an expected six-month review procedure by the FDA.

Following the development, Truist Financial analyst Robyn Karnauskas reiterated a Buy rating and a price target of $125 (61.2% upside potential).

Karnauskas commented, “We see this as a positive not only from the perspective of reduced time for approval/ launch, but also as an acknowledgement of regulatory authority of the strength of the data and the unmet need in severe Hem A patients. Company still on track to submit 1 year pivotal data to the EMA for review in June 2021; this continues to be in-line with company’s guidance for a 2Q submission.”

Consensus among analysts is a Strong Buy based on 10 Buys and 3 Holds. The average analyst price target stands at $107.33 and implies upside potential of 38.4% to current levels.

TipRanks’ Stock Investors tool shows that investors currently have a Very Positive stance on BioMarin, with 5.5% of investors increasing their exposure to BMRN stock over the past 30 days.