FDA grants rare pediatric disease designation to MediciNova’s MN-166 to treat Krabbe disease

Under the FDA’s rare pediatric disease priority review voucher program, the sponsor may be eligible for a voucher that can be used to obtain a priority review for a subsequent human drug application. The FDA has previously granted orphan drug designation to MN-166 (ibudilast) for treatment of Krabbe disease.

MediciNova president and CEO Yuichi Iwaki said: "We are very pleased to receive Rare Pediatric Disease Designation for MN-166 for treatment of Krabbe Disease. 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."

About Krabbe Disease

Krabbe disease is a rare genetic degenerative disorder for which there is no cure and is generally fatal before two years of age. Krabbe disease has 4 clinical subtypes (Types 1 – 4), distinguished by age of onset. In the vast majority of cases, the symptoms of Krabbe disease begin at age 0 – 6 months (Type 1 early infantile form).

Initial signs and symptoms typically include irritability (e.g., excessive crying), limb spasticity, absent reflexes, muscle weakness, feeding difficulties, episodes of fever with no sign of infection, stiff posture, and slowed or regressed neurocognitive development. As the disease progresses, muscles continue to weaken, affecting the infant’s ability to move, chew, swallow, and breathe.

Affected infants also experience vision loss and seizures, regress rapidly to a decerebrate condition and usually succumb to the disease before their 2nd birthday. Approximately 10% of patients present symptoms later in life, including in adulthood (Types 2 – 4).

Progressive loss of vision, difficulty walking, decline in cognitive skills, loss of manual dexterity, and muscle weakness are the most common initial symptoms in this form of the disorder, although signs and symptoms vary considerably among affected individuals. Patients with late-onset Krabbe Disease regress at a slower pace and have the potential to live significantly longer than patients diagnosed in early infancy.

About Rare Pediatric Disease Designation

According to the FDA, a rare pediatric disease 1) primarily affects individuals aged from birth to 18 years and 2) is a rare disease or condition that affects fewer than 200,000 persons in the U.S. The rare pediatric disease priority review voucher program is intended to stimulate new drug development for rare pediatric diseases by offering additional incentives for obtaining FDA approval of these products.

Under the program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease product may be eligible for a voucher which can be used to obtain priority review for a subsequent human drug application. If obtained, the priority review voucher may be sold or transferred to another sponsor.

About MN-166 (ibudilast)

MN-166 (ibudilast) has been marketed in Japan and Korea since 1989 to treat post-stroke complications and bronchial asthma. MediciNova licensed MN-166 (ibudilast) from Kyorin Pharmaceutical Co., Ltd. for potential utility in relapse-remitting multiple sclerosis (RRMS). Intellectual property was additionally established or obtained by MediciNova in progressive MS and other neurological conditions.

MN-166 (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. It attenuates activated glia cells, which play a major role in certain neurological conditions.

Ibudilast’s anti-neuroinflammatory and neuroprotective actions have been demonstrated in preclinical and clinical study results and provide the rationale for its therapeutic utility in neurodegenerative diseases (e.g., progressive MS and ALS), substance abuse/addiction and chronic neuropathic pain.