The FDA has granted rare pediatric disease designation to ibudilast for the treatment of type 1 early infantile Krabbe disease, according to the drug’s manufacturer.
A degenerative genetic disorder characterized by irritability, limb spasticity, episodes of fever with no sign of infection, and progressive neurologic degeneration leading to death before the age of 2 years, Krabbe disease typically is diagnosed after development of symptoms from birth to age 6 months.
An autosomal recessive sphingolipidosis, Krabbe disease is caused by deficient activity of the GALC gene that severely reduces galactosylceramidase enzyme activity. Consequently, certain galactolipids, including galactosylceramide and psychosine, cannot be metabolized and instead accumulate in the cells that produce myelin.
Prior research indicate that psychosine accrual is toxic, causing the imperfect growth and development of myelin, and the resulting severe degeneration of motor skills.
Based on previous studies in progressive multiple sclerosis and other neurological conditions, ibudilast (MN-166, MediciNova) — an orally bioavailable, small molecule phosphodiesterase –4 and –10 inhibitor and a macrophage migration inhibitory factor inhibitor — may suppress pro-inflammatory cytokines and promotes neurotrophic factors.
The anti-neuroinflammatory and neuroprotective properties of ibudilast have been demonstrated in preclinical and clinical study results and provide the rationale for its therapeutic utility in neurodegenerative diseases, such as progressive multiple sclerosis and amyotrophic lateral sclerosis, as well as chronic neuropathic pain.
“We are very pleased to receive a rare pediatric disease designation for MN-166 for the treatment of Krabbe disease,” Yuichi Iwaki, MD, PhD, president and CEO of MediciNova, said in a press release. “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.”
The FDA defines “rare pediatric disease” as a disease that affects fewer than 200,000 individuals in the United States, primarily aged 0 to 18 years. Under the FDA’s rare pediatric disease priority review voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a rare pediatric disease may be eligible for a voucher that can be redeemed to obtain priority review for a subsequent marketing application for a different product.