Af­ter se­ri­ous side ef­fect, Sarep­ta and FDA re­align on next-gen Duchenne drug with tri­al hold lift­ed

FDA’s month­s­long tri­al hold on Sarep­ta Ther­a­peu­tics’ next-gen Duchenne mus­cu­lar dy­s­tro­phy drug is out the door af­ter the biotech and reg­u­la­tor hashed out a new glob­al tri­al pro­to­col to ex­pand mon­i­tor­ing of urine bio­mark­ers.

The US agency took no­tice in June af­ter a se­ri­ous ad­verse event of low mag­ne­sium lev­els in one pa­tient’s blood — a con­di­tion known as hy­po­mag­ne­semia — was ob­served in part B of the MO­MEN­TUM tri­al. The Phase II study is slat­ed to test vesleteplirsen, for­mer­ly SRP-5051, in about 60 pa­tients be­tween ages 7 and 21 in the US, Cana­da and EU.

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