RTTNews | May 24, 2021 08:49AM EDT

08:48 Monday, May 24, 2021 (RTTNews.com) - The European Medicines Agency or EMA has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A, BioMarin Pharmaceutical Inc. (BMRN) said in a statement.

Accelerated assessment reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is expected in the first half of 2022.

BioMarin said it plans to submit a Marketing Authorization Application (MAA) for valoctocogene roxaparvovec for the treatment of severe hemophilia A in June 2021.

In addition, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by Food and Drug Administration.

BioMarin noted that it is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA.

Additionally, BioMarin's valoctocogene roxaparvovec has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

Read the original article on RTTNews ( https://www.rttnews.com/3196994/biomarin-ema-grants-accelerated-assessment-for-review-of-valoctocogene-roxaparvovec.aspx)

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