Sarepta's golodirsen DMD data elicit lackluster expert interest for accelerated approval; clinical benefits from Phase I/II dystrophin increase expected to be mild

03 Jan 2019

Sarepta Therapeutics’ (NASDAQ:SRPT) Phase I/II SRP-4053 (golodirsen) data is not strong enough for approval in Duchenne’s muscular dystrophy, experts said.

On 20 December, Sarepta announced that it had finished its rolling NDA for accelerated approval of golodirsen in DMD patients who have genetic mutations subject to skipping exon 53. One analyst report noted a likely approval based on the data’s strength; another analyst stated the results show a modest benefit but pointed out there is a lack of treatment options.

Whilst experts this news service interviewed were hesitant to suggest how the FDA will view the NDA, they noted that the 4053-101 Phase I/II (NCT02310906) showed an increase to roughly 1% of normal dystrophin levels, which is not very clinically meaningful data. The improvement in the surrogate marker is based on a small uncontrolled patient sample and is not expected to lead to significant clinical benefits, they noted.

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