BioMarin Pharmaceutical Inc.’s experimental gene therapy, valoctocogene roxaparvovec, targeting severe hemophilia A in adults has reportedly met pre-specified clinical criteria to seek regulatory review in the U.S. and Europe. 

According to sources familiar with the development, 8 patients in the 20-patient cohort of the Phase 3 GENEr8-1 trial attained Factor VIII levels of 40 IU/dL, or more, at 23-26 weeks, conforming to the pre-specified standards for Factor VIII activity levels. The company is slated to meet up with the European Medicines Agency (EMA) and Food and Drug Administration (FDA) to assess the phase 3 data and the additional elements of submission and also plans to declare the timing for its strategic marketing applications in the third quarter of 2019.

Based on April 2019 data cutoff, amid weeks 23 to 26, in a unit of the 3^rd phase of GENEr8-1 study of the gene therapy with a dose of 6e13 vg/kg, 7 out of 16 study partakers touched or crossed the pre-specified Factor VIII levels of 40 IU/dL utilizing the chromogenic substrate assay.  Following the April 30 cutoff, one more patient met the criteria, making a total of 8 participants, cited sources close to the development.

Reportedly, valoctocogene roxaparvovec has been granted Breakthrough Therapy Designation by the FDA. The objective of the Breakthrough Therapy Designation program is to facilitate and accelerate the development and review of new drugs to suffice unmet medical requirements for treating severe medical conditions. Meanwhile, the European Medicines Agency has provided access to its Priority Medicines (PRIME) regulatory initiative for valoctocogene roxaparvovec. Qualifying for PRIME requires an investigational therapy to exhibit its probability of benefiting patients with unmet medical requirements on the grounds of early clinical data, claimed sources.

Reportedly, BioMarin's valoctocogene roxaparvovec has also been granted the orphan drug designation by FDA and EMA to treat severe hemophilia A. The Orphan Drug Designation program is aimed at advancing the evaluation and development of products that have the potential to treat rare diseases or conditions, reported sources.

Source Credits: https://investors.biomarin.com/2019-05-28-BioMarin-Announces-that-Phase-3-Cohort-of-Valoctocogene-Roxaparvovec-Gene-Therapy-Study-in-Severe-Hemophilia-A-Met-Pre-Specified-Criteria-for-Regulatory-Submissions-in-the-U-S-and-Europe