MediciNova Gets FDA Orphan Status For IPF Drug

Biopharmaceutical firm MediciNova announced the U.S. Food and Drug Administration (FDA) designation of MN-001 (tipelukast) as an Orphan Drug for the treatment of idiopathic pulmonary fibrosis (IPF).

MN-001 is a novel, orally bioavailable small molecule compound that serves as an anti-fibrotic and anti-inflammatory agent through several mechanisms of action: antagonism of the leukotriene (LT) receptor, phosphodiesterases (PDE) inhibition mainly PDE 3 and 4, and inhibition of 5-lipoxygenase (5-LO). The 5-LO/LT pathway in particular is being investigated as a pathogenic factor in fibrosis development. MN-001 has demonstrated its ability to reduce inflammation by targeting the genes that promote fibrosis. Histopathological data also shows that MN-001 decreased fibrosis in several animal models of disease.

Dr. Yuichi Iwaki, President and CEO of MediciNova, said, “We are very pleased to receive this orphan-drug designation, which is an important part of our development strategy for MN-001 in IPF. As we already have an open IND, we plan to finalize a protocol and submit it to the FDA in order to conduct a Phase 2 clinical trial of MN-001 in IPF.” The company has filed for an Investigational New Drug (IND) application for MN-001 with the Division of Pulmonary, Allergy, and Rheumatology Products (DPARP) of the FDA.

The orphan designation will award MediciNova with 7 years of marketing exclusivity for MN-001 should it be approved for its indication.

Idiopathic Pulmonary Fibrosis (IPF) is a type of pulmonary fibrosis that has no clear cause. The disease is characterized by scarring of the lungs that progressively and irreversibly weakens the organs’ ability to transport oxygen. IPF affects an estimated 128,000 patients in the U.S., with around 48,000 new diagnosed cases every year.

Earlier this year, the company reported positive results for MN-001 in a mouse model of nonalcoholic steatohepatitis (NASH), a condition in which the liver carries excess fat but with no inflammation or damage. NASH currently has no treatment and can lead to liver cirrhosis.