FDA Names Ibudilast an Orphan Drug as Possible ALS Treatment

The U.S. Food and Drug Administration (FDA) has designated  MediciNova’s Ibudilast (MN-166) an orphan drug as a potential treatment for amyotrophic lateral sclerosis (ALS).

Ibudilast is a first-in-class, orally bioavailable small molecule phosphodiesterase (PDE)-4 and -10 inhibitor and a macrophage migration inhibitory factor (MIF) inhibitor that suppresses pro-inflammatory cytokines and promotes neurotrophic factors.

Ibudilast has been available on the Japanese and Korean markets since 1989 to treat post-stroke complications and bronchial asthma. MediciNova is now developing the drug to treat neurologic diseases and, in March, the FDA granted it fast track designation as a possible treatment of progressive multiple sclerosis, a move that aims to speed its development and regulatory review.

The drug’s anti-neuroinflammatory and neuroprotective action has been demonstrated in preclinical and clinical studies, including a double-blind Phase 2 trial (NCT02238626) in ALS patients, comparing the safety, tolerability and clinical responsiveness of Ibudilast, given as an adjunct to Rilutek (riluzole), against placebo. This study is taking place at the Carolinas HealthCare System.

“We are very pleased to receive orphan-drug designation for MN-166 for ALS, a rare disease for which riluzole is the only currently approved treatment option,” Yuichi Iwaki, MD, PhD, MediciNova’s president and chief executive officer, said in a press release. “Currently we have two ongoing clinical trials to evaluate 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 …  study.  This interim data was submitted to FDA to address the scientific rationale for orphan-drug designation to establish the medical basis for the use of MN-166 (ibudilast) for ALS.”

Orphan drug status is given to drugs and biologics that show safety and efficacy in the treatment, diagnosis or prevention of rare diseases affecting fewer than 200,000 people in the United States. The designation entitles the treatment’s developer to seven years of marketing exclusivity and other financial benefits, should it be approved for patient use.

MediciNova is a currently focusing on developing Ibudilast as a treatment for s ALS, progressive multiple sclerosis and for substance abuse or addiction (e.g. alcohol use, methamphetamine dependence, or opioid dependence).