Close
Close
Treatment News

Liver disease drug receives orphan drug designation from FDA

2.33333333333
2.3 from 12 votes
Tuesday, October 01, 2013

The FDA has granted orphan drug designation to Lumena Pharmaceuticals for LUM001, a drug being developed for treatment of four rare liver diseases, according to a press release.

LUM001 is intended for use in patients with Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), primary biliary cirrhosis (PBC) and primary sclerosing cholangitis (PSC). The drug, administered orally once daily, reduces serum bile acid levels induced by these conditions, preventing damage to the liver and improving severe itching.The announcement follows 12 studies of LUM001 in more than 1,400 patients. The company recently initiated a phase 2 study of the drug among children with ALGS, and also plans additional phase 2 studies of LUM001 later this year. Two of those studies will incorporate adult patients with PBC and PSC, and one will assess LUM001 use in pediatric patients with PFIC. Mild, transient GI disturbance was the most commonly reported adverse event during prior studies.

“Orphan drug designation from the FDA allows us to pursue an accelerated pathway for development of LUM001 so we can deliver new drug treatments for the patients in desperate need,” Alejandro Dorenbaum, MD, chief medical officer for the company, said in the release. “Current therapeutic options for cholestatic liver diseases are of limited efficacy, and surgical approaches to reducing bile acid levels to manage disease symptoms can be disfiguring and prone to complications. The only remaining option for many patients is liver transplant.”

 

Source: Healio
2.33333333333
2.3 from 12 votes
Free Newsletter
Trending News
Videos
by Abidemi Uruejoma
12 views
by Abidemi Uruejoma
53 views