FDA Selects Larimar for START Pilot

Rare Daily Staff

The U.S. Food and Drug Administration selected Larimar Therapeutics’ experimental therapy nomlabofusp to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, an effort by the agency to accelerate the development of drugs to treat rare diseases.

The START pilot program was launched by the FDA in September 2023 with an initial selection of up to six novel drugs, three by the Center for Drug Evaluation and Research  and three by the Center for Biologics Evaluation and Research, intended to treat a rare disease or other serious condition with high unmet medical need through an enhanced mechanism for communication with the FDA.

Sponsors selected can benefit from more frequent and rapid ad-hoc interactions with the FDA to help facilitate the development of programs to the pivotal clinical study or pre-BLA meeting stage, and to generate high-quality and reliable data intended to support a BLA or New Drug Application.

Nomlabofusp is a recombinant fusion protein intended to deliver human frataxin to the mitochondria of patients with the rare, neuromuscular disease Friedreich’s ataxia who are unable to produce enough of this essential protein.

Nomlabofusp has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA), Orphan Drug Designation by the European Commission, and a PRIME designation by the European Medicines Agency.

START selection was based on demonstrated development program readiness, including the potential of nomlabofusp to address the serious and unmet medical needs in a rare neurodegenerative condition, alignment of chemistry, manufacturing, and controls development timelines with clinical development plans, and a proposed communications plan where enhanced communication could accelerate pivotal study initiation and path to potential Biologics License Application submission.

“This selection highlights the commitment of the FDA to augment currently available formal meeting communications with more rapid, ad-hoc communication mechanisms with the goal of accelerating the pace of development of nomlabofusp,” said Carole Ben-Maimon, president, and CEO of Larimar. “We look forward to participating in this pilot program and working with the FDA to further the development of nomlabofusp which has the potential to address the root cause of Friedreich’s Ataxia by increasing frataxin levels.”

Nomlabofusp is currently being evaluated in an ongoing open label extension (OLE) study to assess the long-term safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues in patients with FA. Interim data from the OLE study is expected in the fourth quarter of 2024.

Photo: Carole Ben-Maimon, president, and CEO of Larimar