MediciNova has received rare paediatric disease status from the US Food and Drug Administration (FDA) for its MN-166 (ibudilast) to treat Type 1 Early Infantile Krabbe disease.
Krabbe disease is a rare genetic degenerative disorder, which is generally fatal before two years of age. It has four clinical subtypes, which can be distinguished by age of onset.
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MN-166 is a first-in-class, orally bioavailable and small molecule phosphodiesterase (PDE) -4 and -10 inhibitor and a macrophage migration inhibitory factor (MIF) inhibitor, which suppresses pro-inflammatory cytokines and promotes neurotrophic factors.
The ibudilast weakens activated glia cells, which plays a crucial role in certain neurological conditions.
The company also recently obtained orphan drug designation for ibudilast to treat Krabbe disease.
MediciNova president and CEO Dr Yuichi Iwaki said: “We are very pleased to receive Rare Pediatric Disease Designation for MN-166 for treatment of Krabbe disease.
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By GlobalData“There is an unmet medical need for treatment of Krabbe disease in the pediatric population and we plan to work closely with the FDA and clinical experts to evaluate MN-166 in the pediatric population.”
Since 1989, the company has been marketing ibudilast in Japan and Korea to treat post-stroke complications and bronchial asthma.
MediciNova received licence for ibudilast from Kyorin Pharmaceutical for use in the treatment of relapse-remitting multiple sclerosis (RRMS).
The company also obtained additional intellectual property right for ibudilast to be used for progressive MS and other neurological conditions.
