BioMarin Scraps Muscular Dystrophy Drug

8/2/10

BioMarin Pharmaceutical (NASDAQ: BMRN) said today it has halted development of an experimental drug for Duchenne Muscular Dystrophy after an early-stage clinical trial showed it was unlikely to be effective. While the result was discouraging for BMN 195, the company said in a separate statement that another drug, PEG-PAL, is showing “encouraging trends” in a small trial of patients with phenylketonuria (PKU). Full results from that study are expected during the fourth quarter of this year, the company said. BioMarin also lowered its financial forecast for 2010, based on anticipated delays in the U.S. introduction of Firdapse, a drug for a rare autoimmune disease.

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