MediciNova Announces FDA Granted Orphan Drug Designation to MN-166 (ibudilast) for Krabbe Disease

Industry News

The U.S. FDA has granted Orphan Drug Designation to MN-166 (ibudilast) for the treatment of Krabbe Disease.  According to the company’s website, MediciNova, a biotechnology company based in La Jolla, CA, has previously opened an Investigational New Drug application with the Division of Neurology Products for MN-166 and is planning to finalize a protocol for FDA submission in order to conduct a clinical trial of MN-166 in Krabbe Disease.  Krabbe Disease is a rare genetic disorder caused by mutations in the GALC gene, which encodes galactosylceramidase responsible for removing galactose from ceramide derivatives.  The mutations result in a deficiency of this enzyme and subsequently an accumulation of a class of ceramide derivatives called glycosphingolipid leading to a progressive loss of myelin that covers many nerves.  Currently, hematopoietic stem cell transplantation is the only available treatment option and it carries potential risk to the patients with limited efficacy.   MN-166 is an orally available small molecule capable of inhibiting phosphodiesterase (PDE)-4, PDE-10 and macrophage migration inhibitory factor (MIF).  Previous studies have shown that MN-166 attenuates activated glial cells and possesses anti-neuroinflammatory and neuroprotective properties providing the rationale for its potential therapeutic utility in neurodegenerative diseases.


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