MediciNova, Inc., a biopharmaceutical company, has received Fast Track designation from the US Food and Drug Administration (FDA) for MN-001 (tipelukast) for the treatment of patients with idiopathic pulmonary fibrosis (IPF).
Fast Track is a process designed to facilitate the development and expedite the review of drugs that are intended to treat serious or life-threatening diseases and demonstrate the potential to address unmet medical needs for such diseases. An important feature of the FDA's Fast Track programme is that it emphasizes frequent communication between the FDA and the sponsor throughout the entire drug development and review process to improve the efficiency of product development. Accordingly, Fast Track status can potentially lead to a shortened timeline to ultimate drug approval.
In addition to the granting of Fast Track designation to MN-001 for treatment of IPF, the FDA granted Orphan Drug designation to MN-001 for treatment of IPF in October 2014, as previously announced.
Yuichi Iwaki, MD, PhD, president and chief executive officer of MediciNova, Inc., commented, "We are very pleased that MN-001 has received Fast Track designation for IPF and believe this validates its potential to address unmet medical needs in this life-threatening disease. We look forward to providing further updates as our development program progresses."
According to the FDA, in order to be granted Fast Track designation, a drug must (1) be intended for the treatment of a serious or life-threatening disease or condition; and (2) demonstrate the potential to address unmet medical needs for the disease or condition. A drug that receives Fast Track designation may be eligible for more frequent meetings with the FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval; accelerated approval, i.e., approval based on an effect on a surrogate, or substitute endpoint reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality; Priority Review, with an FDA goal for completing review within six months of submission; and Rolling Review, which means that a sponsor can submit completed sections of its New Drug Application (NDA) for review by the FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed.
Pulmonary fibrosis (PF) is a progressive disease characterized by scarring of the lungs that thickens the lining, causing an irreversible loss of the tissue's ability to transport oxygen. The causes of PF vary and can be due to anti-cancer drug therapy or exposure to chemicals. Idiopathic pulmonary fibrosis (IPF) is one type of PF without a clear cause. According to the Coalition for Pulmonary Fibrosis, IPF affects approximately 128,000 individuals in the US, with about 48,000 new cases diagnosed annually. The prognosis for IPF is poor and about two-thirds of IPF patients die within five years of diagnosis.
MN-001 is a novel, orally bioavailable small molecule compound thought to exert its effects through several mechanisms to produce its anti-inflammatory and anti-fibrotic activity in preclinical models, including leukotriene (LT) receptor antagonism, inhibition of phosphodiesterases (PDE) (mainly 3 and 4), and inhibition of 5-lipoxygenase (5-LO). The 5-LO/LT pathway has been postulated as a pathogenic factor in fibrosis development and MN-001's inhibitory effect on 5-LO and the 5-LO/LT pathway is considered to be a novel approach to treat fibrosis. MN-001 has been shown to down-regulate expression of genes that promote fibrosis including LOXL2, collagen type 1 and TIMP-1. MN-001 has also been shown to down-regulate expression of genes that promote inflammation including CCR2 and MCP-1. In addition, histopathological data shows that MN-001 reduces fibrosis in multiple animal models.
Previously, MediciNova evaluated MN-001 for its potential clinical efficacy in asthma and had positive phase 2 results. MN-001 has been exposed to more than 600 subjects and is considered generally safe and well-tolerated.