FDA grants Orphan Drug designation to nutritional supplement

In an unusual move that boosts the credibility of the nutritional supplement industry, the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to Tishcon Corp.'s (Westbury, NY) UbiQGel (Coenzyme Q10) treatment for mitochondrial cytopathies (MC).

Under the Orphan Drug Act, the FDA is given the authority to expedite drug approval under unusual circumstances. The agency may also award research grants for conducting clinical studies. Upon successful completion of the clinical trials, the FDA awards developers seven years of marketing exclusivity. These incentives allow companies to invest in the development of drugs aimed at treating rare or "orphan" diseases.

Mitochondrial Cytopathies encompass a group of debilitating diseases, which are in most cases caused by an inherited mitochondrial defect or dysfunction. MELAS syndrome, Kearns-Sayre syndrome, and MERRF syndrome are some of the more common mitochondrial diseases.

According to Raj Chopra, president of Tishcon, clinical trials of UbiQGel in mitochondrial cytopathy patients will begin this fall, and should be completed in approximately two years.

UbiQGel contains coenzyme Q10, a nutritional supplement, as its active component. As an essential cofactor in mitochondrial energy production, coenzyme Q10 is synthesized in the body and is also obtained through food. UbiQGel allegedly is a more bioavailable form of Q10.

"The significance of being granted an orphan drug designation is enormous for our small company," said Chopra. "Once the clinical trials have been successfully completed, we will be able to obtain marketing approval from the FDA to distribute UbiQGel as an approved prescription drug for mitochondrial cytopathies. This is a rare and a breakthrough designation for a dietary supplement. When prescribed by a physician for the approved indications it will be covered by Federal, State, and private insurance policies, just like other prescription drugs, thus lifting a huge financial burden off the shoulders of MC patients and their families."

For more information: Raj Chopra, President, Tishcon Corp., 30 New York Ave., Westbury, NY 11590-5910. Tel: 516-334-0479.

Edited by Angelo DePalma
Managing Editor, Drug Discovery Online and Pharmaceutical Online