Ucyclyd Pharma gains FDA approval for the treatment of urea cycle disorders.HUNT VALLEY, Md.--(HealthWire)--Sept. 24, 1996--After nearly two years of discussions and reviews, the United States Food and Drug Administration United States Food and Drug Administration (FDA), n.pr a unit of the Public Health Service created to protect the health of the nation against impure and unsafe foods, drugs, and cosmetics. (FDA FDA abbr. Food and Drug Administration FDA, n.pr See Food and Drug Administration. FDA, n.pr the abbreviation for the Food and Drug Administration. ), recently granted Ucyclyd Pharma Inc. of Hunt Valley, final approval to distribute sodium phenylbutyrate, an orphan drug orphan drug, drug developed under the U.S. Orphan Drug Act (1983) to treat a disease that affects fewer than 200,000 people in the United States. The orphan drug law offers tax breaks and a seven-year monopoly on drug sales to induce companies to undertake the marketed under the trade name Buphenyl, for treating urea cycle disorders, a rare disease currently affecting some 400 known patients around the world. The drug, now being manufactured by Pharmaceutics International Inc., also of Hunt Valley, began commercial shipping to pharmacies on Sept. 3, 1996. "For this very small group of patients, Buphenyl is a life-saving drug. Our company was formed solely to ensure that these individuals have uninterrupted access to the therapy they require," said Norbert Wiech, Ph.D., Ucyclyd Pharma's president. "We are very pleased finally to have the FDA's approval. It's been a long road to get this drug to market," Wiech added. The sodium phenylbutyrate treatment originally was developed over 10 years ago by investigators at The Johns Hopkins School of Medicine. The drug had been provided to patients as an investigative new drug (IND) for over five years funded by the National Institute of Child Health, the FDA, and private foundations. Ucyclyd Pharma coordinated the development of the tablet and powder dosage forms, tabulation tab·u·late tr.v. tab·u·lat·ed, tab·u·lat·ing, tab·u·lates 1. To arrange in tabular form; condense and list. 2. To cut or form with a plane surface. adj. Having a plane surface. of safety and efficacy data, and preparation of the new drug application. All this was accomplished without financial help from large pharmaceutical companies, the county or state governments. "We made this project succeed without any significant outside capital -- and, that isn't easy for a start-up company start-up company A new business. providing an orphan drug -- because we saw it as an important responsibility to a small number of patients and their families," said Wiech. Urea cycle disorders may be detected at any age and is characterized by the body's failure to synthesize and excrete excrete /ex·crete/ (eks-kret´) to throw off or eliminate by a normal discharge, such as waste matter. ex·crete v. To eliminate waste material from the body. waste nitrogen (a by-product of protein breakdown). As a result, patients accumulate glutamine glutamine (gl  `təmēn), organic compound, one of the 20 amino acids commonly found in animal proteins. and ammonia in their blood, which can cause brain injury and death. Buphenyl provides patients with an alternate ability to excrete nitrogen which otherwise would accumulate as ammonia. CONTACT: Ucyclyd Pharma Inc. Norb Wiech, 410/584-8188 or Cornerstone Rich Badmington, 410-727-2131, 410/296-6989 (evenings) |
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