Rare Disease Report

New Treatment Option of IPF in Development

JULY 24, 2017
James Radke
The U.S. Food and Drug Administration (FDA) has granted SM04646, a Wnt pathway inhibitor, an Orphan Drug Designation for treating idiopathic pulmonary fibrosis (IPF).
IPF is a chronic, progressive fibrotic interstitial pneumonia that does not have a clear cause. If left untreated, the disease is usually fatal within 2-3 years of initial symptoms. Until recently, treatment was limited to oxygen therapy, pulmonary rehabilitation, and/or lung transplant. However, the approvals of Esbriet (pirfenidone) and Ofev (nintedanib) in 2015 have provided these patients with additional options.
SM04646 is being tested as a treatment option for IPF. It is administered via a nebulizer and being investigated as a monotherapy or in combination with currently approved oral medications – Esbriet and/or Ofev. A phase 1 study showed the drug to be safe and well tolerated.

SMO4646 inhibits a Wnt pathway, which is a signaling pathway with cells that has been implicated in a number of diseases.


Per a news release: “The FDA’s decision to grant an Orphan Drug Designation to SM04646 for IPF is another important milestone in the development of SM04646,” said Dr. Yusuf Yazici, Samumed’s Chief Medical Officer. “IPF is a chronic, progressive, fibrotic disorder that causes deteriorating lung function and severe dyspnea in patients and ultimately ends in fatality. Early trials demonstrate the therapeutic potential of SM04646 to help address the unmet medical need of individuals with IPF.”
Last week, Samumed announced the completion of a Phase I study in which SM04646 was shown to be safe and well tolerated.

For the latest in orphan drug development, be sure to follow Rare Disease Report on Facebook and Twitter

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.