Patients Can't Clear Translarna's Data Hurdles As PTC Falls Short At FDA Panel
Patient testimony to the contrary, advisory committee members were not persuaded that PTC Therapeutics' ataluren, which failed its primary endpoint in two trials, is effective in treating nonsense mutation Duchenne muscular dystrophy.
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US FDA Panel For Sarepta’s Gene Therapy Heavy On Temporary Voting Members
Only four of 14 voting panelists are regular members of the Cellular, Tissues and Gene Therapies Advisory Committee, with scheduling conflicts precluding participation by many others, the FDA said. Three of the temporary members are veterans of prior muscular dystrophy drug reviews.
BrainStorm’s ALS Treatment NurOwn, Filed Over Protest, Will Get US FDA Panel Review
A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.
Keeping Track: A Burst of CRLs and BTDs, A Busy Week Of Submissions And Another Approval For Soliris
The latest drug development news and highlights from our US FDA Performance Tracker.