PTC Therapeutics

PTC Therapeutics updates 2024 revenue guidance to $750-$800 million following strong Q3 earnings, driven by DMD franchise. The company has over $1 billion in cash reserves and plans global launches for sepiapterin and vatiquinone. Challenges include Translarna's uncertain approval timelines in Europe and revenue variability from Translarna.

The global spinal muscular atrophy market is projected to grow from USD 2,013.5 million in 2024 to USD 4,823.9 million by 2031, at a CAGR of 13.3%. Key drivers include FDA approvals for drugs like Zolgensma and Evrysdi, rising awareness, and increasing gene therapies. North America is expected to dominate due to its established healthcare infrastructure.

FDA considers removing oral phenylephrine from OTC guidelines due to lack of efficacy, not safety concerns. Public comments invited until May 7, 2025, before final decision. Phenylephrine is in many nasal decongestants like Sudafed and Mucinex. FDA's proposal does not affect nasal spray decongestants.

PTC Therapeutics reports Q3 2024 revenue of $197 million, raises full-year revenue guidance to $750-800 million. FDA reviews regulatory filings for sepiapterin, Translarna™, and AADC gene therapy. On track for 2024 milestones, including NDA submission for vatiquinone. Financial highlights include total revenue of $196.8 million, net loss of $106.7 million, and cash, cash equivalents, and marketable securities of $1,013.4 million.

Despite failed trials, hope is renewed in Huntington's disease treatment with a gene therapy showing 80% slowing of disease progression and another treatment seeking FDA accelerated approval.

FDA to decide on Journey's rosacea drug DFD-29, Merus' bispecific antibody for NSCLC and PDAC, PTC's gene therapy Upstaza for AADC deficiency, and Autolus' CAR T therapy obe-cel for ALL by November 4, 6, 13, and 16 respectively.

Regeneron's Eylea sales miss estimates, UCB sees potential in Alzheimer's drug bepranemab, Madrigal's Rezdiffra sales exceed expectations, AbbVie partners with EvolveImmune for cancer drugs, Lundbeck acquires Longboard Pharmaceuticals, and PTC Therapeutics awaits FDA decision on Translarna for Duchenne muscular dystrophy.

PTC Therapeutics announces FDA acceptance of Translarna NDA resubmission for nonsense mutation Duchenne muscular dystrophy (nmDMD). The NDA is supported by significant benefits in a global placebo-controlled trial and long-term treatment data from the STRIDE registry. Translarna aims to restore functional protein formation in patients with genetic disorders caused by nonsense mutations.

PTC Therapeutics plans to submit a New Drug Application to the FDA for vatiquinone, its oral therapy for Friedreich’s ataxia, based on positive results from the MOVE-FA trial and extension studies, despite missing the primary goal. Vatiquinone showed significant benefits in upright stability and a strong safety profile, particularly relevant for pediatric patients.