factor IX
FDA Rejects PTC’s Vatiquinone Application for Friedreich’s Ataxia, Requests Further Studies
FDA rejection; PTC Therapeutics; vatiquinone; Friedreich’s ataxia; complete response letter; drug efficacy; additional clinical studies
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure
PTC crashes as win for Novartis-partnered Huntington’s drug fails to convince investors
factor IX, PTC518, Accelerated, Approved, United States Food and Drug Administration
PTC’s Novartis-partnered Huntington’s drug disappoints in mid-stage data reveal
PTC518, factor IX, Huntington ‘s disease, Market
Biotech Restructuring and European Regulatory Setbacks
LifeMine Therapeutics, layoffs, PTC Therapeutics, Translarna, Duchenne muscular dystrophy, European Commission, regulatory decision
Novartis Invests $1 Billion in PTC’s Huntington’s Disease Program
Novartis, PTC, Huntington’s disease, pharmaceutical investment, gene therapy
Novartis Invests $1 Billion in PTC Therapeutics’ Huntington’s Disease Program
Novartis, PTC Therapeutics, Huntington’s disease, Phase 2 drug, global license agreement, collaboration, upfront payment, milestone payments.
PTC Therapeutics Sells Priority Review Voucher for $150M Following Phase 2 ALS Trial Failure
PTC Therapeutics, ALS, Phase 2 trial failure, priority review voucher, $150M sale
PTC Therapeutics Discontinues Utreloxastat Program Following Phase 2 ALS Trial Failure
PTC Therapeutics, Utreloxastat, ALS, Phase 2 Trial, CardinALS Study, Amyotrophic Lateral Sclerosis
FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency
PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder