US-based biopharmaceutical company PTC Therapeutics Inc (NASDAQ:PTCT) announced on Friday a negative opinion from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) on the renewal of the conditional marketing authorisation of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
This follows the return of the previously issued negative opinion by the European Commission for re-review.
PTC intends to request re-examination of the opinion.
The marketing authorisation remains valid until the end of 2024, even if the negative opinion is maintained and ratified.
PTC argues that real-world evidence supports Translarna's use and said it will fight to keep it available for DMD patients.
HELP Therapeutics' HiCM-188 IND application receives US FDA approval
Ananda's cannabinoid medicines to feature in NHS epilepsy trials
AstraZeneca's Wainzua recommended for EU approval
Astellas Pharma's VYLOY receives US FDA approval
Accord Healthcare wins CHMP approval for Stelara biosimilar Imuldosa
Ionis and AstraZeneca's WAINZUA recommended for EU approval
Novartis receives positive CHMP opinion for Kisqali
Viatris signs exclusive licensing agreement with Lexicon Pharmaceuticals
Adicet Bio receives FDA clearance to expand ADI-001 trials to additional autoimmune diseases
GSK PLC announces acceptance of NDA for gepotidacin by US FDA
SystImmune receives FDA clearance for Phase 1 trial of novel AML therapy
Accord BioPharma's IMULDOSA receives US FDA approval to treat chronic inflammatory conditions
PTC Therapeutics' sepiapterin receives US FDA target regulatory action date
RedHill Biopharma's opaganib selected for Ebola treatment development by BARDA