Catalyst Pharmaceuticals Announces FDA Approval of Firdapse for Lambert-Eaton Myasthenic Syndrome with Breakthrough Therapy and Orphan Drug Designations

VCBeat (WeChat ID: vcbeat) has learned that on November 28, 2018 (U.S. local time), Catalyst Pharmaceuticals (NASDAQ: CPRX, hereinafter referred to as “Catalyst Pharma”), a biopharmaceutical company based in Florida, announced that its New Drug Application (NDA) for Firdapse had received marketing approval and orphan drug designation from the U.S. Food and Drug Administration (FDA). Firdapse is a medication for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder. This marks the first drug approved by the FDA for the treatment of LEMS. The drug has also been approved for the treatment of congenital myasthenic syndromes (CMS) and myasthenia gravis.

Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare neuromuscular disorder characterized primarily by muscle weakness, with a global incidence of three cases per million people. LEMS triggers the patient’s own immune system to attack the junctions between nerves and muscles, impairing the ability of nerve cells to transmit signals to muscle cells, thereby resulting in muscle weakness.

Image source: Catalyst Pharma official website

“In the U.S., while some off-label medications used to treat LEMS symptoms have demonstrated varying degrees of efficacy and tolerability, Firdapse is the first drug to receive regulatory approval for this indication. Currently, there are no competing drugs for LEMS in development,” said Oppenheimer analyst Leland Gershell prior to the approval.

The U.S. FDA states that LEMS may be associated with other autoimmune disorders and is more common in cancer patients, such as those with small cell lung cancer.

This approval was granted through the FDA’s Priority Review pathway. It took only six months from the time Catalyst Pharma submitted its New Drug Application (NDA) to the FDA’s final approval. Previously, the agency had designated Firdapse as a Breakthrough Therapy, a status primarily awarded to drugs that may address serious conditions and thereby qualify for FDA Priority Review. Once approved, such drugs demonstrate significant improvements in the safety and efficacy of treatment, prevention, or diagnosis for serious diseases.

In a report, brokerage firm SunTrust Robinson Humphrey stated that Firdapse would generate $375 million in revenue for the company by 2025.

This drug received European Union approval in late 2009, was designated as an orphan medicinal product in the EU, and was granted 10 years of market exclusivity. It is marketed in Europe by BioMarin Pharmaceutical Inc. (hereinafter referred to as “BioMarin”). In 2012, BioMarin licensed its North American rights to Catalyst Pharma.

Catalyst Pharma, founded in 2002 and headquartered in Florida, USA, is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare, debilitating chronic neuromuscular and neurological disorders, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), MuSK antibody-positive myasthenia gravis, type 3 spinal muscular atrophy (SMA), and infantile spasms.

Catalyst Pharma was listed on the NASDAQ in the United States in 2006, with the stock ticker symbol CPRX. The company is currently developing a drug named CPP-115 for the treatment of refractory infantile spasms. CPP-115 has been granted U.S. Orphan Drug Designation by the FDA for the treatment of infantile spasms and has also received EU Orphan Medicinal Product Designation from the European Commission for the treatment of West syndrome. Furthermore, Catalyst is developing a generic version of Sabril (vigabatrin).