Avicena Wants to Market Creatine as Orphan Drug

The Avicena Group of San Francisco says it has a creatine product that's much purer than what you can now buy over the counter, and that it plans to market the drug as a specific treatment for ALS.

In February, Avicena received permission from the U.S. Food and Drug Administration to designate its creatine product, NEOtine, an "orphan drug." Orphan drugs are those developed to treat diseases affecting fewer than 200,000 people in the United States or those for which there's no reasonable expectation that development costs can be recovered from sales.

NEOtine is being tested in a multicenter clinical trial in ALS. Trial sites include several MDA/ALS centers, and the MDA/ALS Center at Carolinas Medical Center in Charlotte, N.C., is coordinating the study.

For more information about the trial, which remains open, contact Ruth King, research coordinator at the Carolinas Center, at (704) 355-8699 or ruth.king@carolinashealthcare.org

If the FDA is convinced that NEOtine is safe and effective in ALS and that it's sufficiently different from available creatine products, it could receive approval as an ALS treatment under the FDA's Orphan Products Program.

"Orphan drug designation is the first step toward final drug approval," said Belinda Tsao-Nivaggioli, vice president of Operations at the Avicena Group. "The next step for us is to meet with the FDA in a pre-IND [investigational new drug] meeting and decide the design of the final, phase 3 clinical trial (if one is needed) before applying for a new drug approval (NDA)."

People with ALS probably will be able to get reimbursement through insurance and government programs if the drug is approved for the disease, Tsao-Nivaggioli said. "Doctors can [then] prescribe this like any other prescription drug."

The Orphan Products Program gives pharmaceutical companies various financial incentives, such as marketing rights, tax credits and assistance with testing.

For details about this trial and other ALS clinical trials, see www.mdausa.org/research/ctrials.html

Source: Muscular Dystrophy Association - USA