Firdapse’s Catalyst Pharmaceuticals Named BioFlorida’s Top Company

Firdapse is only therapy approved in US to treat LEMS symptoms

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by Mary Chapman |

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Catalyst Pharmaceuticals has been named BioFlorida’s David J. Gury Company of the Year for its breakthrough Lambert-Eaton myasthenia syndrome (LEMS) treatment Firdapse (amifampridine).

BioFlorida, which represents Florida’s life sciences industry, recognizes a single company each year for significant milestones and achievements that advance life sciences in the state.

“It is an honor to be recognized for such a prestigious award which represents the culmination of the hard work and commitment by our Catalyst teams that helped get us to where we are today,” Patrick J. McEnany, Catalyst’s founder, chairman, and CEO, said in a press release. “Since our founding, we have been inspired to develop and provide innovative treatment options for patients living with rare diseases. Our focus on patients and addressing important unmet medical needs is the foundation of our core values and accomplishments. We are honored to be acknowledged among the many deserving life science companies with these shared goals.”

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While Firdapse was originally developed by BioMarin Pharmaceutical, Florida-based Catalyst has held licensing rights to the treatment in North America since 2012. The medication works to improve muscle strength and mobility in LEMS, a rare autoimmune disorder that mainly affects lower limb muscles.

Firdapse is the first and only oral therapy available in the U.S. to treat the disease’s symptoms. Its 2018 regulatory approval in the U.S. for patients ages 17 and older was expanded last month to include children as young as 6.

The European Commission approved Firdapse for commercial use in 2009. It was  also approved in 2020 in Canada for treating adult patients. Catalyst amended its license agreement for Firdapse in 2019 to expand its commercial territory to Japan.

“We are excited to recognize Catalyst Pharmaceuticals as the BioFlorida Company of the Year and celebrate their efforts to make a meaningful difference in the lives of patients suffering from Lambert-Eaton Myasthenic Syndrome (“LEMS”) with their breakthrough treatment, Firdapse,” Nancy K. Bryan, BioFlorida’s president and CEO, said. “The Catalyst team has an unwavering commitment to the patient community it serves and dedication to ensuring that all LEMS patients nationwide have access to the treatment. We are proud to have Catalyst represent the Sunshine State as a shining example of industry advancement.”

Catalyst received the award at BioFlorida’s 25th Annual Conference, which brought together Florida’s life sciences industry to mark continued success in science, business, and public policy, and to spur collaboration and future growth. Catalyst was also one of the companies recognized during the conference’s Spotlight on Innovation session for demonstrating “transformational growth.”