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BioMarin Begins Pediatric Study on Achondroplasia Candidate

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BioMarin Pharmaceutical Inc BMRN stated that it has dosed the first participant in a phase II study, evaluating its pipeline candidate, vosoritide, in pediatric patients with achondroplasia, a genetic disorder causing dwarfism in infants and toddlers.

The phase II placebo-controlled study (n=70) on vosoritide will be conducted on children with achondroplasia in the age bracket of 0-5 years. The primary objective of the study will be to evaluate safety, tolerability and effect of vosoritide on height Z-scores.

We would like to remind investors that vosoritide is already being assessed in a phase III trial on children aged five to 11 years with achondroplasia. Top-line data from this study are expected to be announced in the second half of 2019, followed by an open-label extension analysis with the lower dose (15 µg/kg/day). Vosoritide has an Orphan Drug status in both the United States and the EU for achondroplasia.

Earlier in May, BioMarin received an FDA nod for its pipeline candidate, pegvaliase, to be marketed by the trade name of Palynziq injection, developed to reduce blood phenylalanine (Phe) levels in Phenylketonuria (PKU) patients.

BioMarin already has a PKU drug in its portfolio called Kuvan. Revenues of the same rose 7% to $99.1 million in the first quarter of 2018. Kuvan is a key growth driver of BioMarin’s revenues. Per settlement with Dr Reddy’s RDY and Par Pharmaceuticals, a subsidiary of Endo International plc ENDP, Kuvan generics are expected to enter the U.S. market from October 2020 onward.

Shares of BioMarin have inched up 0.9% year to date against the industry’s decline of 6.9%.


BioMarin currently carries a Zacks Rank #2 (Buy). Another top-ranked stock in the biomedical sector is CRISPR Therapeutics AG CRSP, sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here

CRISPR Therapeutics’ loss per share estimates has been narrowed by 7.8% for 2018 and 9% for 2019 in the last 60 days. The stock has skyrocketed 151.5% year to date.

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