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BioMarin (BMRN) to File for Hemophilia A Candidate in Q4

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that it will file regulatory applications seeking approval of valoctocogene roxaparvovec, its gene therapy, for severe hemophilia A, in both the United States and EU in the fourth quarter of 2019.

BioMarin claims that this is the first time a gene therapy product for any type of hemophilia will be filed with regulatory authorities.

This year so far, BioMarin’s shares have declined 5.6% while the industry witnessed an increase of 3.9%.



BioMarin is conducting two separate phase III studies on valoctocogene roxaparvove —GENEr8–1 (6e13 vg/kg dose) and GENEr8–2 (4e13 vg/kg dose) — in patients without pre-existing AAV5 antibodies. In May, BioMarin presented top-line data from the GENEr8-1 study.

Data from one cohort of the study showed that treatment with gene therapy candidate achieved factor VIII levels of 40 international units per deciliter (IU/dL) in eight out of 20 patients, a pre-specified criterion for accelerated approval. In this cohort, 20 patients were dosed 6e13 vg/kg of valoctocogene roxaparvovec for 23 to 26 weeks. Annual bleed rate (“ABR”) also reduced significantly from baseline. Back then, the company had said that enrolment for accelerated filing requirements is complete.

This interim phase III data from the GENEr8-1 study and updated three-year data from a long-term phase I/II study on valoctocogene roxaparvovec will form the basis of these regulatory submissions. The GENEr8-1 study will however continue to enroll to achieve its planned completion target of 130 total patients. Enrolment is expected to be completed by fall of 2019.

Three-year updated data from the phase I/II study on valoctocogene roxaparvovec was also announced in May, which demonstrated the durable benefit of the candidate. The data from the study showed that a single injection of valoctocogene roxaparvovec (both 6e13 vg/kg and 4e13 vg/kg doses) led to a substantial reduction in bleeding and the need for Factor VIII infusions. In the higher dose cohort, a 96% reduction in mean ABR as well as mean FVIII usage was observed.

Moreover, data also showed that significant reduction in ABR was sustained in the third year, following infusion of a high dose (6e13 vg/kg) of valoctocogene roxaparvovec. However, data showed that factor VIII levels decreased over the same period. However, the reduction in ABR for the lower dose (4e13 vg/kg) was sustained only into the second year.

Please note that valoctocogene roxaparvovec enjoys FDA’s Breakthrough Therapy Designation and orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A.

Hemophilia A is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Despite being prescribed current standard of care medicines, severe hemophilia A patients continue to experience painful bleeds, thereby creating significant need for medicines that can improve patients' quality of life. Several companies are developing gene therapy products to treat severe hemophilia A.

Sangamo Therapeutics, Inc. (SGMO - Free Report) and its partner Pfizer, Inc. (PFE - Free Report) presented promising new results from the phase I/II Alta study, evaluating their gene therapy candidate, SB-525, in patients with severe hemophilia A. Other companies developing gene therapy candidates for treating hemophilia A include Spark Therapeutics (ONCE - Free Report) and uniQure.

BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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