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BioMarin's Hemophilia Candidate Included in PRIME Initiative

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that BMN 270, being evaluated for the treatment for severe hemophilia A, has been granted access to Priority Medicines (PRIME), a regulatory initiative by the European Medicines Agency (EMA). For a candidate to be accepted under PRIME, it has to demonstrate the potential to benefit patients with unmet medical needs based on early clinical data.

BioMarin’s share price has gained 23.3% in the past one year, while the Zacks classified Medical - Biomedical and Genetics industry fell 6.2%.

As mentioned earlier, PRIME is an initiative undertaken by the EMA, which aims to provide better scientific guidance and support accelerated review of investigational therapies that address high unmet medical need. The initiative also offers early and enhanced regulatory support to optimize regulatory applications. This will help patients benefit as early as possible from therapies that might significantly improve their quality of life.

In Mar 2016, BioMarin announced that both the FDA and the European Commission granted Orphan Drug designation to BMN 270 for the treatment of patients with hemophilia A. The candidate is currently in phase I/II for hemophilia A. We remind investors that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) had approved the resumption of enrollment in the open-label phase I/II study for BMN 270 in Oct 2016. The regulatory agency has also approved the company's proposed amendments in the study, including elimination of the requirement for prophylactic corticosteroids and increasing additional enrollment from up to three patients to a maximum of six patients. The company expects to enroll patients in the study in the third quarter of 2017.

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 MHRA, on the basis of which it approved the continuation of the study.

Zacks Rank & Key Picks

BioMarin currently carries a Zacks Rank #3 (Hold).  Some better-ranked stocks in the health care sector include Sunesis Pharmaceuticals, Inc. SNSS, Enzo Biochem, Inc. ENZ  and Anika Therapeutics Inc. ANIK. All of them carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.

Enz Biochem’s loss estimates for 2017 narrowed 5.88% over the past 60 days. The company recorded a positive earnings surprise in three of the last four quarters, the average being 22.50%. Its share price was up 48.7% in the past one year.

Anika’s earnings estimates for 2016 and 2017 were up 3.9% and 0.5%, respectively, over the last 60 days. The company recorded a positive earnings surprise in each of the last four quarters, the average being 33.14%. Its share price was up 32.8% in the past one year.

Sunesis’ loss estimates narrowed 5.06% and 8.80% for 2016 and 2017, respectively, over the past 60 days. The company recorded a positive earnings surprise in three of the last four quarters, the average being 0.54%.

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