Dive Brief:
- Rare disease company Catalyst Pharmaceuticals Inc. on Monday unveiled positive topline data from a Phase 3 study of Firdapse as a symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disease affecting the muscles in the limbs.
- In the trial, Firdapse met both of its primary endpoints, leading to improvements in patients' quantitative myasthenia gravis (QMG) score and subject global impression (SGI). Catalyst called the 6.4 point difference between Firdapse and placebo in QMG scores "clinically significant."
- These results will be used to support a New Drug Application to the Food and Drug Administration, which Catalyst hopes to submit in the first quarter of 2018.
Dive Insight:
Almost two years ago, Catalyst received a "refusal-to-file" letter from the FDA, followed by a demand from the regulator for additional clinical study. The setback sent Catalyst shares into a slide and lengthened the company's time to market.
Fast-forward to the present and Catalyst is now anticipating a re-filing of Firdapse with the FDA to come soon.
Catalyst conducted the most recent Phase 3 study under a Special Protocol Assessment process, giving the company a green light to measure the drug against the QMG and SGI co-primary endpoints.
It's not clear how many people develop Lambert-Eaton myasthenic syndrome (LEMS) each year, with estimates ranging from several hundred to a few thousand. About half of cases are related to an underlying cancer, most commonly small cell lung cancer.
In the past, Catalyst's attempt to win approval of Firdapse has sparked some controversy, given the drug's similarity to an unapproved drug for the condition known as 3,4-Dap and made by the privately owned small pharma Jacobus Pharmaceuticals.
Approval of Firdapse, along with the seven years of orphan drug exclusivity, could shut Jacobus out and threaten the supply of 3,4-Dap, which Jacobus has previously made available for the cost of shipping only.
Catalyst picked up the North American rights to Firdapse from BioMarin Pharmaceuticals Inc. in October 2012 and launched the drug in Europe back in 2010.
Last week, Catalyst moved Firdapse into a Phase 2 trial in ambulatory patients with spinal muscular atrophy type 3. The drug is also in clinical development for congenital myasthenic syndromes, and MuSK antibody positive myasthenia gravis (MuSK-MG).
Catalyst announced a public offering of 11 million shares of common stock on Monday.