EMA Names Investigational Gene Therapy, BMN 270, a Priority Medicine for Hemophilia A

BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is preparing new clinical trials expected to begin this year.

The European Medicines Agency (EMA)’s Committee for Advanced Therapies (CAT) and its Committee for Medicinal Products for Human Use (CHMP) agreed that BMN 270 should have access a new EMA regulatory initiative called Priority Medicines (PRIME).

PRIME offers enhanced support in the development of medicines that target an unmet medical need. This voluntary program is meant to optimize development plans and speed up evaluation so these medicines, considered priority medicines within the European Union, can reach patients earlier.

CHMP ruled in favor of access to the PRIME scheme based on results reported by BioMarin, showing that BMN 270 addresses unmet clinical need based on:

• Breakthrough bleeds in preventive treatment settings, leading to sequelae, such as permanent joint disease as a result of repeated bleeding in the joints (hemophilic arthropathy);

• Preliminary clinical data demonstrating that a single IV administration of BMN 270 leads to sustained restoration of factor VIII activity, reduces annualized bleeding rates, and improving patients’ quality of life.

“We are thrilled that the EMA has recognized the potential of BMN 270 to change the course of hemophilia A and provide a treatment paradigm currently unmet by available therapies,” Hank Fuchs, MD, president of worldwide research and development at BioMarin, said in a press release.  “We look forward to preparing to enroll patients in a registration enabling study in the third quarter of this year, as we work closely with the EMA to accelerate development and hopefully to facilitate earlier access to the first gene therapy treatment for patients with severe hemophilia A, the most common form of the disease.”

BMN 270, a recombinant AAV vector coding for human-coagulation factor VIII, is an investigational gene therapy for hemophilia A. In mouse models of hemophilia A, BMN 270 restored factor VIII plasma concentrations to levels projected to be adequate for normal clotting in humans.

BioMarin reported positive interim results of a Phase 1/2 clinical trial (NCT02576795) in July 2016. The trial is evaluating the efficacy and safety of escalating doses of BMN 270 in up to 15 patients with severe disease (defined by the WFH as less than 1% of the blood clotting factor VIII). The results, presented at the XXXII International Congress of the World Federation of Hemophilia (WFH), showed that patients with severe disease treated with BMN 270 had improved and showed sustained clotting function.