BALA CYNWYD, PA — Larimar Therapeutics announced that its novel therapy nomlabofusp has been chosen to participate in the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. This selection marks a significant step for the drug designed to treat Friedreich’s ataxia (FA), a rare neurodegenerative condition.
Nomlabofusp is a protein replacement therapy aimed at addressing the root cause of FA by delivering frataxin to mitochondria. The FDA’s decision to include it in the START program was based on several factors: the therapy’s potential to meet serious unmet medical needs, readiness of its development program, and a well-aligned plan for chemistry, manufacturing, and controls (CMC) development.
The START program, launched by the FDA in September 2023, aims to accelerate drug development for rare diseases and other serious conditions with high unmet needs. The program will initially select up to six novel drugs, divided between the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). Drugs in the program benefit from enhanced communication with the FDA, enabling more frequent and rapid interactions to facilitate the development process.
“We are thrilled nomlabofusp, the first potential therapy to increase frataxin levels in patients with FA, has been selected by the FDA to participate in the START pilot program,” said Carole Ben-Maimon, President, and CEO of Larimar. “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.”
Dr. Russell Clayton, Chief Medical Officer of Larimar, noted the importance of the increased communication enabled by the START program. “Participating in the START program enables increased communication with the FDA to help expedite the progression of our nomlabofusp development program to the pre-BLA meeting stage. This is important to patients who are continuing to live with this devastating disease.”
Nomlabofusp is currently being tested in an open-label extension (OLE) study to evaluate its long-term safety, tolerability, pharmacokinetics, and frataxin levels in peripheral tissues in FA patients. Interim data from this study is expected in the fourth quarter of 2024.
The inclusion of nomlabofusp in the START program could have far-reaching implications for the treatment of Friedreich’s ataxia. Enhanced communication with the FDA may shorten the timeline to pivotal clinical trials and eventual approval. This could bring hope to patients suffering from this debilitating condition, providing a therapy that targets the underlying cause.
Larimar’s progress with nomlabofusp reflects broader trends in biotechnology, where targeted therapies for rare diseases are gaining attention. The potential success of this program not only holds promise for FA patients but also sets a precedent for future treatments of rare conditions.
As the healthcare industry continues to evolve, initiatives like the START program play a crucial role in accelerating the availability of innovative therapies. For Larimar Therapeutics, this milestone signifies a promising future for both the company and the patients it aims to serve.
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