RTTNews | Jul 15, 2021 09:00AM EDT

09:00 Thursday, July 15, 2021 (RTTNews.com) - The European Medicines Agency or EMA validated BioMarin Pharmaceutical Inc.'s (BMRN) Marketing Authorization Application or MAA for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A, the company said in a statement.

A CHMP opinion is anticipated in the first half of 2022.

BioMarin said it looks forward to working with the agency as it evaluates the robust data set in the application, which the company believes address the requests made during the prior MAA review.

The company noted that the submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study.

BioMarin resubmitted a Marketing Authorization Application to the European Medicines Agency on June 25, 2021. In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration.

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

Read the original article on RTTNews ( https://www.rttnews.com/3209397/ema-validates-biomarin-maa-for-valoctocogene-roxaparvovec-to-treat-severe-hemophilia-a.aspx)

For comments and feedback: contact editorial@rttnews.com

Copyright(c) 2021 RTTNews.com All Rights Reserved