1. Scrip
  2. >>Therapy Areas

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).

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

More from Therapy Areas

In Brief: AbbVie’s Rinvoq Secures US Approval For Giant Cell Arteritis

 
• By Sushmita Panda

JAK inhibitor gains ninth indication in US after a strong performance in the first quarter.

CSPC’s Global-First Monospecific ADC Promising In Advanced NSCLC

 
• By Xu Hu

CSPC’s SYS6010, the first monospecific EGFR-targeting antibody-drug conjugate in the clinic for advanced NSCLC, has shown Phase I promise, but could face close competition with another China-originated bispecific.

Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.