BioMarin Pharmaceutical Inc. BMRN announced that the initial six-month data from the first three cohorts of a phase II proof-of-concept and dose-finding study on vosoritide (BMN 111) were presented at the annual meeting of the American Society for Bone and Mineral Research. Data from the study were previously announced in Jun 2015.
Vosoritide is being developed for the treatment of children with achondroplasia and has Orphan designation in the U.S. and the EU.
According to the World FactBook 2014 edition, about 1 in 25,000 live births in the world are affected by achondroplasia. The company estimates that 24,000 patients under 18 years of age, out of about 96,000 total patients in its territories, can be treated with vosoritide.
Meanwhile, BioMarin continues to progress on its pipeline. Currently, the company is evaluating two candidates in phase III – pegvaliase for the treatment of patients with phenylketonuria (PKU) and reveglucosidase alfa for the treatment of patients with of Pompe disease.
Moreover, the company’s lead pipeline candidate, drisapersen, is under FDA review with a response expected by Dec 27, 2015. BioMarin is seeking approval for drisapersen for the treatment of patients with Duchenne muscular dystrophy (DMD) with mutations in the dystrophin gene that are amenable to treatment with exon 51 skipping. In Aug 2015, drisapersen received rare pediatric disease designation from the FDA for the above indication.
BioMarin carries a Zacks Rank #2 (Buy). Other favorably-ranked stocks in the health care sector include Anika Therapeutics Inc. ANIK, Endo International plc ENDP and Nexvet Biopharma Public Limited Co. NVET, each sporting a Zacks Rank #1 (Strong Buy).
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