CytRx Corporation, a biopharmaceutical company engaged in the development and commercialization of human therapeutics, announced that its lead drug candidate arimoclomol has been granted orphan medicinal product status for the treatment of amyotrophic lateral sclerosis (ALS) by the European Commission.
This orphan medicinal product status grants CytRx market exclusivity in the European Union (EU) for 10 years in the event that arimoclomol receives marketing approval, and provides for incentives such as reduced fees for protocol assistance and scientific advice.
"We appreciate the European Commission's recognition of arimoclomol as a potential drug for ALS," said CytRx president and CEO Steven A. Kriegsman. "This is a life-threatening and debilitating condition for which there is currently no effective treatment and we believe that arimoclomol is based on a novel mechanism of action that may hold a key in ALS therapy."
CytRx senior vice president of drug development Jack Barber, Ph.D., commented, "We intend ultimately to pursue product approval for arimoclomol in ALS in the EU, which will require us to follow a modified regulatory process from that in the US. The granting of orphan medicinal product status provides us the mechanism with which to meet with the EMEA and other regulatory officials in the EU in pursuit of EU approval going forward."
The European Commission grants orphan medicinal product status to promote the innovation of drugs that are developed to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. Arimoclomol received orphan drug status from the U.S. Food and Drug Administration for the treatment of ALS in May 2005. Orphan drug status in the US holds numerous potential benefits, including opportunities for grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, seven years of US market exclusivity should the FDA grant marketing approval for the drug and an added mechanism for more frequent communication with the FDA.
Based on the company's September 2006 announcement of meeting the primary safety and tolerability endpoints in a phase IIa trial with arimoclomol as an ALS treatment, CytRx plans to initiate a phase IIb trial in the US and Canada in mid-2007, subject to FDA approval. The company believes that positive efficacy and safety results from the phase IIb clinical trial could be sufficient for arimoclomol product registration in the US for this indication.
Arimoclomol is one of CytRx's three orally-administered, small molecule compounds. This small molecule drug candidate is believed to function by stimulating a normal cellular protein repair pathway through the activation of "molecular chaperones." Since damaged proteins called aggregates are thought to play a role in many diseases, CytRx believes that activation of molecular chaperones could have therapeutic efficacy for a broad range of diseases.
ALS is a progressive degeneration of the brain and spinal column nerve cells that control the muscles that allow movement. Over a period of months or years, ALS causes increasing muscle weakness, inability to control movement and problems with speaking, swallowing and breathing. According to the ALS Survival Guide, 50 per cent of ALS patients die within 18 months of diagnosis and 80 per cent die within five years. More than 120,000 people are living with ALS worldwide.