Catalyst Pharmaceuticals

Overview

Catalyst Pharmaceutical Partners, Inc.

type	Public
traded as	nasdaq: CPRX Russell 2000 Component S&P 600 Component
founder	Patrick J. McEnany
location city	Coral Gables, Florida, U.S.
key people	Patrick J. McEnany (chairman Richard Daly (CEO and Board member)‡R1R‡
industry	Biotechnology
products	FIRDAPSE® (amifampridine) Tablets 10 mg (commercialized 2019) AGAMREE® (vamorolone) Oral suspension 40mg/ ml FYCOMPA® (perampanel) CIII
operating income	US$ 86.8 million (2023)‡R2R‡
assets	US$ 700 million (2023)‡R2R‡
equity	US$ 302.9 million (2023)‡R2R‡
num employees	176‡R2R‡‡R3R‡
homepage	www.catalystpharma.com
footnotes	‡R4R‡ ‡R5R‡

Catalyst Pharmaceuticals, Inc. is a biopharmaceutical company based in Coral Gables, Florida, United States. The company develops medicines for rare diseases, including the phosphate salt of amifampridine for the treatment of Lambert–Eaton myasthenic syndrome (LEMS). The drug is referred to under the trade name Firdapse, which was approved by the FDA for approved use in children 6 years and older with LEMS in addition to the prior approval for use in adults with LEMS on November 28, 2018. Firdapse commercially launched in January 2019.[6]

History

Catalyst was founded in 2002, and completed an IPO in 2006.[7] It focused primarily on developing therapies to prevent addiction until 2012.[8]

In 2009, Catalyst in-licensed worldwide rights to a family of GABA inhibitors including CPP-115 from Northwestern University.[9]

Litigation and Reception

In December 2015 a group of neuromuscular doctors published an editorial in the journal, Muscle & Nerve, with concerns about the potential for the price to be increased should Catalyst obtain FDA approval, and stating that 3,4-DAPP represented no real innovation and didn't deserve exclusivity under the Orphan Drug Act.[33][34] Catalyst responded to this editorial with a response in 2016 that explained they were conducting a full range of clinical and non-clinical studies necessary to obtain approval in order to specifically address the unmet need among the estimated 1500-3000 LEMs patients since about 200 were receiving the product through compassionate use – and that this is exactly what the Orphan Drug Act was intended to do.[35] Prior to the FDA approval, patients were able to get an investigational version of amifampridine for free through compassionate use programs in accordance with FDA Rules and Guidelines.[36][37]

On January 28, 2022, in Catalyst Pharmaceuticals, Inc. v. Becerra, the Eleventh Circuit upheld orphan exclusivity for Catalyst Pharmaceuticals and its drug Firdapse. With this decision, the Eleventh Circuit rejected the FDA's interpretation of orphan exclusivity and concluded that the agency had improperly approved a competitor product from Jacobus Pharmaceutical Co.[38][39]

On February 4, 2019, Bernie Sanders, United States Senator from Vermont, publicly sent a letter to Catalyst asking why they raised the price of their drug Firdapse to an annual cost of $375,000, considering Firdapse was previously free of charge through an FDA compassionate use program. Catalyst responded by saying, "The approval of Firdapse by the FDA means that all LEMS patients in the United States now have access to this much-needed medication". Sanders questioned the financial decision regarding the negative impact, specifically asking about how many patients would suffer or die, for patients who may no longer be able to afford the drug. The drug is used to treat Lambert–Eaton myasthenic syndrome (LEMS), which is a rare neuromuscular disorder.[40][41]

Catalyst Pharmaceuticals

History

Lambert-Eaton myasthenic syndrome

Duchenne muscular dystrophy

Amifampridine

Litigation and Reception

References