Everything You Need to Know About Larimar Therapeutics' Nomlabofusp and the FDA's START Program

Over 7,000 rare diseases affect millions of people worldwide, with the majority lacking effective treatments. In rare diseases, where treatment options are often limited, hope can sometimes feel like a distant light. Finding cures for these conditions is usually a long and challenging process, but hope is on the horizon.

A human mitochondrial protein. Reduced expression causes Friedreich's ataxia. © Studio Molekuul/Wirestock - stock.adobe.com

In a groundbreaking initiative to accelerate the development of treatments for rare diseases, the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program has emerged as a beacon of hope for patients and researchers alike. This program can potentially transform the landscape of rare disease treatments, offering promise for those affected by these conditions.

Due to their low prevalence and the need for more awareness, rare diseases present unique challenges in terms of research and treatment options. Traditional drug development processes may not always be feasible for these conditions. The START program is a crucial step toward addressing these challenges and improving the situation for those affected by rare diseases.

The START Pilot Program, which focuses on fostering innovative research and collaboration, is set to revolutionize the landscape of rare disease therapeutics. Its mission is to bridge the gap between innovation and access, providing a platform for accelerated clinical trials in rare disease therapeutics. This program could pave the way for life-changing breakthroughs in rare disease treatment.

To achieve this accelerated format, novel therapies for rare diseases will be developed by enhancing communication with the FDA. The sponsors chosen can take advantage of increased and swift, spontaneous engagements with the FDA to advance programs toward the crucial clinical study phase or pre-BLA meeting, aiming to produce dependable and credible data to back a BLA or NDA. ​

This past May, Larimar Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, announced that its lead compound for Friedreich's Ataxia, Nomlabofusp, would participate in the START pilot program. Nomlabofusp is one of the three Center for Drug Evaluation and Research programs and one of the six programs selected by the FDA.

Nomlabofusp is a protein replacement therapy designed to deliver frataxin to mitochondria, addressing the root cause of Friedreich's Ataxia. It has received various designations from regulatory bodies, such as Rare Pediatric Disease designation, Fast Track designation, Orphan Drug designation, and PRIME designation. ​

The selection of nomlabofusp for the START program allows Larimar Therapeutics to communicate more effectively with the FDA to expedite the development program towards the pre-BLA meeting stage. Interim data from an ongoing open-label extension (OLE) study are expected in the fourth quarter of 2024. ​ The study assesses the long-term safety, tolerability, pharmacokinetics, and frataxin levels in peripheral tissues of Friedreich's ataxia patients. ​