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HomeLatest Pharma-NewsThe first oral small molecule targeting splicing Risdiplam approved by US FDA...

The first oral small molecule targeting splicing Risdiplam approved by US FDA for the treatment of spinal muscular atrophy

Nov 25, 2019: PTC Therapeutics, Inc announced that the United States Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for risdiplam (RG7916) for the treatment of spinal muscular atrophy (SMA).

It gives improvements in the safety or efficiency of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. The SMA program is a collaboration between PTC, the SMA Foundation and Roche.

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It is designed to supply sustained increase in SMN protein centrally and peripherally through daily dosing and is being evaluated for its potential aptitude to help the SMN2 gene produce more functional SMN protein to all over the body.

Risdiplam is being studied in a clinical trial for patients with type 1 SMA, called FIREFISH, in pre-symptomatic babies, RAINBOWFISH, in patients who have been in previous clinical trials for SMA, JEWELFISH and in SUNFISH, a placebo-controlled study in people aged 2-25 years with type 2 or 3 SMA.
http://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-grants-priority-review-risdiplam

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