EMA Validates BioMarin' MAA For Valoctocogene Roxaparvovec To Treat Severe Hemophilia A

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.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Nestle USA Inc., affiliated to Swiss food and beverage giant Nestle SA, is recalling around 27,872 pounds of frozen DiGiorno Crispy Pan Crust pepperoni pizza citing misbranding and undeclared soy protein, a known allergen, according to the U.S. Department of Agriculture's Food Safety and Inspection Service or FSIS. Eli Lilly and Co. is recalling one lot of GLUCAGON Emergency Kit for low blood sugar to the consumer/user level citing loss of potency, the U.S. Food and Drug Administration announced. The recall involves lot D239382D with expiration date of April 2022 of Glucagon Emergency Kit containing 1mg of freeze-dried (lyophilized) product in a 3 mL vial and a pre-filled diluent syringe. Twitter has rolled out the tipping feature called "Tips" for everyone around the globe using the microblogging site with more payment options to choose from, including cryptocurrencies such as Bitcoin. This is a new way for people to receive and show support on Twitter with money. The feature is currently available on iOS and will be available on Android over the coming weeks.
Follow RTT