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BioMarin Issues Encouraging Update on Hemophilia A Drug

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved the resumption of enrollment in the open-label phase I/II study on its pipeline candidate, BMN 270, which is an experimental gene therapy being evaluated for the treatment of severe hemophilia A.

The regulatory agency has also approved the company's proposed amendments to the study, including the elimination of the requirement for prophylactic corticosteroids and increasing additional enrollment from up to three additional patients to up to six additional patients.

We remind investors that BioMarin had suspended the dosing of patients in the study after enrolling the first nine patients. This was due to increases in alanine aminotransferase levels that were observed to exceed the pre-specified threshold set by the company. Subsequent to the suspension of the study, the company reviewed safety and efficacy data on the nine patients with the MHRA. On the basis of the review, the MHRA approved the continuation of the study.

BioMarin plans to resume enrollment in the study before the end of 2016.  Positive safety and efficacy data from the study would allow the company to commence a phase IIb study on the candidate in the second half of 2017.


We are encouraged by the latest development. Earlier this March, BMN 270 received orphan drug status in the EU for the treatment of hemophilia A.

Current prescribed treatments for hemophilia A include Biogen Inc.’s (BIIB - Free Report) Eloctate and Shire plc’s (SHPG - Free Report) Adynovate among others.

Apart from BMN 270, BioMarin has a robust pipeline of candidates targeting orphan diseases. Interesting candidates include Brineura, currently under review in the U.S. for the treatment of children with CLN2 disease, which is a form of Batten disease. A response from the FDA is expected by Apr 27, 2017.

Zacks Rank and Stock to Consider

BioMarin currently carries a Zacks Rank #2 (Buy). Incyte Corporation (INCY - Free Report) is another favorably placed stock in the health care sector sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Incyte’s earnings estimates for 2016 and 2017 were up a respective 29.4% and 9.8% over the last 60 days. The company has beaten earnings estimates thrice in the last four quarters with an average surprise of 335.16%.

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