Research & Development
PTC Therapeutics Inc receives FDA priority review for NDA for risdiplam to treat spinal muscular atrophy
25 November 2019 -

Biopharmaceutical company PTC Therapeutics Inc (NASDAQ:PTCT) on Monday announced that it has received US Food and Drug Administration (FDA) priority review for the New Drug Application (NDA) for risdiplam.

PTC Therapeutics stated that risdiplam is an investigational medicine designed for the safety or effectiveness of the treatment, diagnosis or prevention of spinal muscular atrophy (SMA) when compared to standard applications, in collaboration with the SMA Foundation and Roche.

The company added that the US FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of 24 May 2020 for a decision.

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that is the leading genetic cause of mortality in infants and toddlers caused by deletion or mutation in the survival of motor neuron 1 (SMN1) gene, which results in reduced levels of SMN protein.

Risdiplam (RG7916) is intended to provide sustained increase in SMN protein centrally and peripherally through daily dosing and is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body, added the company.

In addition, the filing acceptance by the US FDA triggers a USD15m milestone payable to PTC by Roche.

This NDA filing is based on 12-month data from the dose-finding portion of the pivotal FIREFISH and SUNFISH studies and clinical and preclinical pharmacokinetic and pharmacodynamic data. SUNFISH part two recently met its primary endpoint of change from baseline in the Motor Function Measure 32 scale, concluded the company.



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