1. Scrip
  2. >>Therapy Areas
  3. >>Anticancer

Catalyst Collapses On FDA Firdapse Refusal

 
• By Donna Young

Investors hit Catalyst Pharmaceuticals Inc. hard on Feb. 17, driving shares down 50%, after the company disclosed the FDA refused to file (RTF) the Florida firm's new drug application (NDA) for Firdapse (amifampridine phosphate), a 3,4-diaminopyridine phosphate, as a symptomatic treatment for two very rare neuromuscular diseases – Lambert Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS).

Investors hit Catalyst Pharmaceuticals Inc. hard on Feb. 17, driving shares down 50%, after the company disclosed the FDA refused to file (RTF) the Florida firm's new drug application (NDA) for Firdapse (amifampridine phosphate), a 3,4-diaminopyridine phosphate, as a symptomatic treatment for two very rare neuromuscular diseases – Lambert Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS).

The Catalyst stock ended the day at $1.16, down 69 cents, or a loss of 37.3%.

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Anticancer

Vepdegestrant Up For Grabs As Pfizer And Arvinas Step Aside

 
• By Manas Mishra

Pfizer and Arvinas have agreed to out-license their PROTAC breast cancer candidate, vepdegestrant, with Arvinas cutting jobs and refocusing on early-stage assets.

China’s Therapeutic mRNA Vaccine Pipeline For Oncology The Largest Globally

 
• By Xu Hu

Nearly half of the mRNA-based therapeutic vaccines for cancer in the R&D pipeline globally are from Chinese companies, although financing activities in the area have been relatively quiet so far this year in China compared with 2020-23.

Biokin/BMS, Genfleet/Verastem’s Drug Candidates Impress At WCLC

 
• By Dexter Jie Yan

With data generated from China-only trials and consistency issues lingering, Chinese drug makers presented impressive results from early-stage studies of their assets at the World Conference on Lung Cancer annual conference.

Revolution’s Daraxonrasib Data Bolster Phase III Case Amid RAS Cancer Push

 
• By Alaric DeArment

The company announced positive monotherapy and chemotherapy-combination data for the RAS(ON) inhibitor in first-line pancreatic ductal adenocarcinoma.

More from Therapy Areas

Roivant Sets The Stage For Commercial Return With Brepocitinib Data

 
• By Jessica Merrill

Positive Phase III data positions Roivant to launch the oral TYK2/JAK1 inhibitor for dermatomyositis, with an NDA filing planned in early 2026.

AstraZeneca’s Fasenra RESOLUTE Failure Highlights The Limits Of Biologics In COPD

 
• By Alexandra Shimmings

AstraZeneca’s IL-5 blocker has again missed in COPD, adding to a number of disappointments for biologics in this disease. Complex biology and overlapping inflammatory pathways seem to be at the heart of the problem.

Lilly’s Orforglipron Bests Novo Rival In First Oral Head-To-Head Study

 
• By Andrew McConaghie

The company’s cardiometabolic leader Ken Custer discusses the topline ACHIEVE-3 results in diabetes and just-published full results of ATTAIN-1 in obesity of orforglipron, with a pledge to maximize access to the oral GLP-1 therapy worldwide, once approved.