The US Food and Drug Administration (FDA) has granted orphan-drug designation to biopharmaceutical company MediciNova Inc.'s MN-166 (ibudilast) for treatment of Krabbe disease. MediciNova previously opened an Investigational New Drug (IND) application with the Division of Neurology Products (DNP) for MN-166 (ibudilast).
Krabbe disease (also called globoid cell leukodystrophy) is a degenerative disorder that affects the nervous system. It is caused by the shortage (deficiency) of an enzyme called galactosylceramidase. This enzyme deficiency impairs the growth and maintenance of myelin, the protective covering around certain nerve cells that ensures the rapid transmission of nerve impulses. In the United States, Krabbe disease affects about 1 in 100,000 individuals. 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 second birthday.
The orphan drug designation programme provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
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. 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 Company 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.
Subscribe to PharmaTutor News Alerts by Email >>
