According to some sources, the FDA removed some clinical restrictions on the drug after conducting a phase II data review of the Nomlabofusp clinical project under the new drug developer Larimar Therapeutics.
The Zhitong Finance App learned that according to some sources, the US Food and Drug Administration (FDA) lifted some clinical restrictions on the drug after conducting a phase II data review of the Nomlabofusp clinical project under Larimar Therapeutics (LRMR.US), a developer of new drugs for the treatment of rare diseases. Boosted by this news, Larimar Therapeutics's US stock rose nearly 23% after the market on Monday.
According to reports, Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company's Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria. Interim data from the drug's Open Label Extension (OLE) study is expected to be released in the fourth quarter. The company aims to submit an FDA marketing application in the second half of 2025.
Other companies developing Friedreich's ataxia (FA) treatments include PTC Therapeutics (PTCT.US) and Design Therapeutics (DSGN.US). BIIB.US (BIIB.US)'s Friedreich ataxia (FA) treatment drug SkyClarys was approved by the FDA last year to treat adult and adolescent Friedreich ataxia (FA) patients aged 16 and above. The drug was approved for marketing in the European Union in February this year to treat adult and adolescent Friedreich ataxia (FA) patients aged 16 and above.