Catalyst Pharmaceuticals Inc (NASDAQ:CPRX) received marketing authorization for Firdapse from Health Canada to treat patients with LEMS (Lambert-Eaton myasthenic syndrome) in Canada.
Marketing authorization under priority review
LEMS is a debilitating, rare, and life-threatening neurodegenerative condition. The national health care regulator agency gave the nod for Firdapse under Priority Review. Health Canada reviewed efficacy and safety data submitted by Catalyst from its previous two clinical studies that investigated Firdapse to cure patients suffering from LEMS.
Firdapse already received the approval of the US FDA and the EU in 2018 and 2009 to treat patients with LEMS in the US and over 15 nations overseas. Chief Medical and Regulatory Officer of Catalyst, Gary Ingenito, said the company is committed to improving people’s lives with rare neuromuscular disease. It is pleased to bring an approved drug to the patient population in Canada to cure LEMS. The company will work with Health Canada to explore using the drug for medical conditions.
The vice president (Business Development) of Catalyst, David Ailinger, said the company is negotiating with a Canadian partner to access Firdapse in Canada quickly.
Rare autoimmune disorder
The rare autoimmune disorder – LEMS is characterized by the weakness of muscles in the limbs. Patients suffer from LEMS because of an autoimmune reaction in their body, where the formed antibodies act against the potassium channels and interfere with the communication between the muscles and nerves. LEMS usually affects legs and causes difficulty in climbing the stairs and getting up from a sitting posture.
Well tolerated in the MSK-002 trial
In the MSK-002 clinical trial, Firdaspe exhibited well tolerance and safe in treating similar to that observed in curing LEMS. The primary endpoint is not achieved in this clinical study. Catalyst will further analyze the clinical data and decide for the MuSK-MG indication after discussing it with its neuromuscular advisors.
Catalyst is free from debt. It maintains investments, cash equivalents, and cash of $115.1 million by the end of Q2 2020. Catalyst posted a net income of $11.6 million in Q2 2020.
The company will expand the commercialization of Firdapse in the US. It will continue ongoing development programs that investigate Firdapse to treat SMA Type 3 and MuSK-MG.
Catalyst will also promote pre-commercialization activities of Firdapse in Canada. It is also working health regulators in Japan seeking Marketing Authorization for Firdapse.