EU Panel Recommends Orphan Drug Status for Ibudilast for ALS Treatment

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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EMA ibudilast recommendation

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending that ibudilast (MN-166) receives orphan medicinal product (orphan drug designation) for the treatment of amyotrophic lateral sclerosis (ALS).

MediciNova is currently focusing on the development of ibudilast for neurological disorders such as ALS, progressive multiple sclerosis, and drug use disorders (including methamphetamines and opioids).

“We are very pleased to receive a positive opinion recommending orphan-drug designation for [ibudilast] for ALS in the European Union, a recommendation that complements our recently granted orphan-drug designation in the U.S.,” Yuichi Iwaki, MD, PhD, MediciNova’s president and CEO, said in a press release.

“This is an important milestone for the development of a promising new therapeutic treatment for ALS, a life-threatening, rare disease for which riluzole is the only currently-approved treatment option in the EU and U.S.,” Iwaki said.

Ibudilast is an oral 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, a family of molecules that support the development and growth of new and mature neurons.

The investigational drug reduces the effect of activated glia cells, which are now known to play an important role in certain neurological conditions.

Ibudilast has been marketed in Japan and Korea since 1989 to treat post-stroke complications and bronchial asthma, but its anti-neuroinflammatory and neuroprotective actions have also been proven in preclinical and clinical studies, providing the basis for its therapeutic potential in neurodegenerative diseases.

This is the first orphan medicinal product designation that MediciNova has received in Europe.

MediciNova has two ongoing clinical trials to evaluate ibudilast for ALS patients in collaboration with Carolinas HealthCare System’s Neuromuscular/ALS-MDA Center and Massachusetts General Hospital, Iwaki said.

He said ibudilast showed positive trends in interim effectiveness data from the mid-study analysis of the CHS Neuromuscular/ALS-MDA Center study.

“This interim data, along with previously reported positive ALS preclinical study data, was submitted to EMA COMP to address the scientific rationale for orphan medicinal product designation to establish the medical basis for the use of [ibudilast] for ALS,” Iwaki added.

An orphan medicinal product in Europe refers to pharmaceuticals that have been developed for the treatment of life-threatening or chronically debilitating rare diseases with a prevalence of fewer than five patients per 10,000 people. The status offers potential benefits like protocol assistance, fee waivers, and market exclusivity periods.