FDA Approves Drug to Treat Rare Pediatric Liver Disease.

Business & Health/Medical Editors

NASHVILLE, Tenn.--(BW HealthWire)--Jan. 22, 2002

The FDA notified Swedish Orphan International AB based in Stockholm, Sweden that it has granted marketing approval for Orfadin(R) Capsules (nitisinone) an orphan designated product as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of Hereditary Tyrosinemia Type I (HTI). The product was designated an Orphan Drug On May 16, 1995 by FDA's Office for Orphan Product Development.

Orfadin(R) Capsules (nitisinone) is to be marketed and distributed by Rare Disease Therapeutics, Inc. based in Nashville, Tennessee.

Hereditary Tyrosinemia Type I is a rare genetic metabolic disorder characterized by the lack of an enzyme needed to break down the amino acid tyrosine. The most common form of the disease is Acute Tyrosinemia Type I which, if untreated, is fatal in the first year of life. There also exist a chronic and a sub-chronic form of the disease, with a more protracted course but still fatal if untreated. There are currently 54 known patients in the United States with an anticipated prevalence world wide of 300 patients.

"I am extremely pleased with this approval," said Milton Ellis, President of Rare Disease Therapeutics, Inc.

"The FDA approval of Orfadin(R) offers hope for the rare disease patients with HTI throughout the world," said Bo Jesper Hansen, Managing Director of Swedish Orphan International AB, "and is a very important recognition to the Swedish Orphan International Group of Companies for the past many years of dedicated development work of this true orphan product."

"Until now, Hereditary Tyrosinemia Type I has been a tragic and untreatable disease," said Abbey Meyers, President of the National Organization for Rare Disorders (NORD). "The potential of Orfadin(R) is very exciting."

There are no known contraindications to Orfadin(R) Capsules (nitisinone). Inadequate restriction of tyrosine and phenylalanine intake can result in elevations in plasma tyrosine which can lead to toxic effects to the eyes, skin, and nervous system. Slit lamp examination of the eyes should be performed before initiation of therapy and patients exhibiting ophthalmic symptoms should undergo reexamination and measurement of plasma tyrosine with adjustment to diet as needed.

Patients treated with Orfadin(R) Capsules (nitisinone) and dietary restriction in clinical trials developed transient thrombocytopenia (3%) and leucopenia (3%) or both (1.5%). Platelet and white blood cell counts should be monitored regularly during therapy. Regular liver monitoring by imaging and laboratory tests, including serum alpha-fetoprotein concentration is recommended, since hepatic neoplasm and liver failure have occurred on therapy.

Rare Disease Therapeutics, Inc. is an affiliate of the Swedish Orphan International Group of Companies, which is a global consortium of Orphan Pharmaceutical Companies throughout the United States, Europe, Japan, Australia, and Canada. The corporate mission being to provide patients, healthcare personnel, and the pharmaceutical industry with an independent global network specializing in the development, marketing and distribution of Orphan Products for the treatment of rare disorders and products for the treatment of unmet medical needs where current treatment is either unavailable or unsatisfactory. Globally the consortium has a 13-year history of multiple licensing agreements, comprehensive global patient advocacy networking, and significant successes in drug development and approvals.