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

August 7, 2024 By Andrew Cox, Pharm.D., MBA. www.managedhealthcareexecutive.com 

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. ​

Lexeo Therapeutics' LX2006 Shows Promise in Treating Friedreich Ataxia Cardiomyopathy

August 9, 2024. By Andrew Cox, Pharm.D., MBA. Managed Healthcare Executive.

Although the initial results are promising, LX2006's ultimate success will depend on ongoing safety evaluations, long-term efficacy data, and the ability to navigate the regulatory landscape. Lexeo Therapeutics' interim clinical data for LX2006 represents a potentially transformative advancement in treating Friedreich Ataxia cardiomyopathy. While the early results are encouraging, ongoing clinical evaluations and regulatory engagements will be vital in determining the future of this promising gene therapy.

New and Emerging Drug and Gene Therapies for Friedreich Ataxia

Scott V, Delatycki MB, Tai G, Corben LA. New and Emerging Drug and Gene Therapies for Friedreich Ataxia. CNS Drugs. 2024 Aug 8. doi: 10.1007/s40263-024-01113-z. Epub ahead of print. PMID: 39115603. 

 This review provides a contemporary position of drug and gene therapies for FRDA currently in phase 1 clinical trials and beyond. Despite significant scientific advances in the specificity of both compounds and targets developed and investigated, challenges remain for the advancement of treatments in a limited recruitment population. Currently therapies focus on reducing oxidative stress and improving mitochondrial function, modulating frataxin controlled metabolic pathways and gene replacement and editing. Approval of omaveloxolone, the first treatment for individuals with FRDA aged 16 years and over, has created much excitement for both those living with FRDA and those that care for them.