MedPath

Nomlabofusp

Generic Name
Nomlabofusp
Brand Names
-
Drug Type
Biotech
Chemical Formula
-
CAS Number
2548202-05-5
Unique Ingredient Identifier
V6S49GL5GS
Associated Conditions
-
Associated Therapies
-

A Promising Candidate for Friedreich's Ataxia

Larimar presented positive early clinical results for nomlabofusp (CTI-1601), a drug designed to correct frataxin deficiency in FRDA patients. The phase 1 study involved 55 patients, with primary endpoints of safety and tolerability. A phase 2 trial showed increased frataxin levels with doses of 25 mg and 50 mg. An ongoing open-label extension study will include adolescents and children, with preliminary data expected in Q4 2024. Larimar aims for accelerated FDA approval with a BLA submission planned for mid-next year.

A Broad View of the Future of Friedreich's Ataxia Therapy

Recent review highlights advancements in Friedreich’s ataxia (FRDA) therapy, including the approval of Skyclarys (omaveloxolone) and emerging gene therapies, emphasizing the need for sensitive outcome measures and personalized medicine approaches.
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