BioMarin seeks license for gene therapy to treat hemophilia

Kim Yun-mi  Published 2020.01.06  16:44  Updated 2020.01.06 16:44


The world might be one step closer to the eradication of hemophilia.

BioMarin Pharmaceutical said it has submitted a biologics license application to the European and U.S. health authorities for BMN 270, a candidate treatment for hemophilia A. The submission was the first as gene therapy for hemophilia.

The company said the therapy reduced the frequency of treatments for bleeds significantly.

“The European Medicines Agency validated the company's Marketing Authorization Application (MAA) to the EMA. BioMarin anticipates the start of the MAA review to commence in January 2020 under accelerated assessment,” the company said in a press release dated Dec. 23.

BioMarin also said in a public filing on the same day that it submitted the license application to the U.S. Food and Drug Administration and expected that the FDA would start the review in February. As both the EMA and the FDA designated BMN 270 for expedited review, the company will receive the result within this year, observers said.

BMN 270 is a single-dose, adeno-associated virus (AAV) mediated gene therapy, for the production of Factor VIII, a clotting protein. The license application included an interim analysis of a phase-3 study and the three-year phase-1/2 data.

The phase-1/2 study evaluated the efficacy and safety of BMN 270 in 15 adult patients with severe hemophilia A with less than 1 percent of Factor VIII levels. The data included the follow-up results of the maintenance effect three years after the administration.

The results showed that in two people, who received doses of six 10 to the power of 12, and two 10 to the power of 13 vector genomes per kilogram body weight (vg/kg), respectively, did not experience the maintenance effect, as their Factor VIII was less than 1 IU/dL.

However, the median Factor VIII of seven patients treated with six 10 to the power of 13 vg/kg was 20 IU/dL, and the median number of treatments due to bleeding was zero.

Patients who received six 10 to the power of 13 vg/kg and four 10 to the power of 13 vg/kg maintained the clinical benefits in the third year. The results were published in the New England Journal of Medicine on Thursday.

The phase-3 study on BMN 270 is also taking place in Korea.

The Food and Drug Safety Ministry’s data on clinical trial approval, the authorities granted two phase-3 trials on BMN 270, and one of them was complete.

The first one was a multinational study on preventive therapy, testing the four 10 to the 13 vg/kg of BMN 270 in patients with severe hemophilia A who were receiving Factor VIII injections. The regulator granted the nod in April last year, and the trial has been completed. In Korea, four male patients participated in the study at three institutions.

The other study received approval on April 8 last year, and the regulator did not disclose the information on the dose. Five male patients are participating in the study at five institutions in Korea.

As local institutions are testing the treatment in phase-3 trials, the company is expected to seek Korean approval after winning the go-ahead in the U.S. and Europe, observers said.

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