gene therapy
FDA nears decision on Duchenne therapy
Sarepta Therapeutics is expecting to hear from the FDA on Thursday about granting conditional approval to its gene therapy for Duchenne muscular dystrophy.
A marketing clearance is expected, although it will likely be limited to boys aged 4 to 5, where the gene therapy has shown greater efficacy. Sarepta has been seeking a broader approval to cover all boys under 10 who can still walk, but the company said last month that the FDA was more likely to grant a narrower approval, pending additional data from an ongoing clinical trial.
public shaming
U.K. trade group scolds Leo Pharma, others
Leo Pharma, a Danish maker of dermatological drugs, has been chastised by the Association of the British Pharmaceutical Industry for "serious and extremely concerning" code violations. A manager at Leo tried to get pricing information from a rival, so that its products could ultimately appear more cost effective to regulators and insurers. The company has admitted to the transgression, saying "a comprehensive internal investigation was launched, followed by appropriate disciplinary action."
The U.K. trade group also rebuked AstraZeneca and Daiichi Sankyo, saying both had omitted or provided misleading safety information. It will run advertisements noting the various company violations as part of an effort to bolster the industry's image.
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