GlaxoSmithKline Muscular Dystrophy Drug Reaches Primary Objective in Study

Published: Apr 10, 2013

GlaxoSmithKline Plc (GSK)’s drisapersen achieved the primary objective of a mid-stage study, heating up competition with Sarepta Therapeutics Inc. (SRPT) in the race for a drug that could reverse the debilitating effects of Duchenne muscular dystrophy. Patients taking drisapersen showed a clinically meaningful difference from those on placebo after 48 weeks of treatment, according to an abstract of the study results posted on the website of CureDuchenne, an investor in Glaxo’s partner on the drug, Dutch biotechnology company Prosensa Therapeutics BV. Glaxo will present the data at a genetics conference in Cold Spring Harbor, New York, tomorrow. Duchenne muscular dystrophy is a neuromuscular disease with no known cure that affects one in 3,500 newborn boys. Drisapersen has been designated by U.S. and European regulators as an orphan drug, and may eventually reach 500 million pounds ($766 million) in sales if proven to be effective and approved for commercialization, according to Fabian Wenner, an analyst at Kepler Capital Markets in Zurich.

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