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# FDA Orphan Status for ALS Drug
- URL: https://www.fdaweb.com/fda-orphan-status-for-als-drug/
- Published: 2016-10-12T12:00:00.000Z
- Updated: 2026-09-14T21:39:03.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137062

FDA has granted MediciNova an orphan drug designation for MN-166 (ibudilast) for treating amyotrophic lateral sclerosis (ALS). Currently, the company has two ongoing clinical trials that are evaluating MN-166 in ALS in collaboration with researchers at Carolinas HealthCare System’s (CHS) Neuromuscular/ALS-MDA Center and Massachusetts General Hospital. “MN-166 demonstrated positive trends in the interim efficacy data from the mid-study analysis of the CHS Neuromuscular/ALS-MDA Center study,” it says. The interim data were submitted to FDA to support the orphan drug designation.

Ibudilast has been marketed in Japan and Korea since 1989 to treat post-stroke complications and bronchial asthma. MediciNova says it is also developing MN-166 for progressive multiple sclerosis and other neurological conditions such as ALS and substance abuse/addiction. The company describes MN-166 as a “first-in-class, orally bioavailable, small molecule phosphodiesterase-4 and -10 inhibitor and a macrophage migration inhibitory factor inhibitor that suppresses pro-inflammatory cytokines and promotes neurotrophic factors. It attenuates activated glia cells, which play a major role in certain neurological conditions.”