The Food and Drug Administration has selected Bala Cynwyd-based Larimar Therapeutics’ lead drug candidate for its new program inspired by the Operation Warp Speed initiative, which was used for the rapid approval of COVID-19 vaccines, writes John George for the Philadelphia Business Journal.
Larimar’s Nomlabofusp, an experimental therapy for Friedreich’s ataxia, will be the focus of the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.
“I think this really validates that the FDA is very interested in this program and that they think there is a real opportunity to get this product to patients — as long as the data pans out,” said Carole Ben-Maimon, CEO of Larimar.
Following the news, Larimar’s stock increased by 14.25 percent to close at $9.14 per share on Monday.
Friedreich’s ataxia is a rare neurodegenerative condition that is inherited and causes progressive damage to the nervous system, which results in movement difficulties.
Larimar is developing Nomlabofusp as a novel protein-replacement therapy specifically aimed at addressing the root cause of Friedreich’s ataxia by delivering frataxin, an iron-storage protein, to mitochondria. People affected by Friedreich’s ataxia cannot produce the required amount of this essential protein.
Read more about Larimar Therapeutics in the Philadelphia Business Journal.
