Friday, May 31, 2024
Larimar Therapeutics, Inc. (Nasdaq: LRMR), a company in the biotechnology sector focusing on rare diseases, has announced that the FDA has selected nomlabofusp to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. Nomlabofusp is a novel protein therapy designed for the treatment of Friedreich’s ataxia (FA) by delivering frataxin to mitochondria, targeting the root cause of the disease. The selection for the START program was based on nomlabofusp's readiness for development, its potential to address the significant medical needs in FA, alignment of development timelines, and a proposed communication strategy to expedite pivotal study initiation and potential Biologics License Application (BLA) submission.
The START pilot program, initiated by the FDA in September 2023, aims to accelerate drug development, initially selecting up to six novel drugs, three each by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), to address rare diseases or other serious conditions with high unmet needs. Sponsors selected for START benefit from more frequent and rapid interactions with the FDA to facilitate development towards pivotal clinical studies or pre-BLA meetings, aiming for high-quality data to support a BLA or New Drug Application (NDA).
Larimar, expressed enthusiasm about nomlabofusp's selection, emphasizing the FDA's commitment to accelerating development through enhanced communication. Dr. Russell Clayton, Larimar's Chief Medical Officer, highlighted the importance of increased FDA communication in progressing nomlabofusp's development, particularly for patients with FA.
Nomlabofusp is currently undergoing evaluation in an ongoing open-label extension study to assess long-term safety, pharmacokinetics, and frataxin levels in FA patients. Interim data is expected in Q4 2024.
Nomlabofusp is a recombinant fusion protein designed to deliver human frataxin to mitochondria in FA patients. It has received various designations from regulatory bodies, including Rare Pediatric Disease, Fast Track, and Orphan Drug designations from the FDA, Orphan Drug Designation from the European Commission, and PRIME designation from the European Medicines Agency.
Source: larimartx.com
