FDA approves Orphan Pharmaceutical's drug to treat rare pediatric liver disease.Orphan Pharmaceuticals Inc., Nashville, TN has received approval from the 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. to market nitisinone capsules under the name of Orfadin to treat hereditary tyrosinemia type 1 (HT-1), a rare pediatric pediatric /pe·di·at·ric/ (pe?de-at´rik) pertaining to the health of children. pe·di·at·ric adj. Of or relating to pediatrics. disease causing progressive liver failure liver failure Clinical medicine Liver insufficiency that results in death, requires a liver transplant, or is characterized by recovery after encephalopathy, or while awaiting a transplant; also defined as a condition with ≥ 3 of following: albumin < 3. and liver cancer Liver Cancer Definition Liver cancer is a relatively rare form of cancer but has a high mortality rate. Liver cancers can be classified into two types. in young children. The drug was given 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 approval to treat the fewer than 100 children in the US who are affected by the disease. Orphan products are developed to treat rare diseases, or conditions that affect fewer than 200,000 people in the US. The Orphan Drug Act provides Orphan Pharmaceuticals with 7-year exclusive marketing rights as the first sponsor to obtain marketing approval for the drug. An FDA Talk Paper noted that because of liver failure or liver cancer, children with hereditary tyrosinemia type 1 rarely survive into their twenties without a liver transplant. However, the FDA said liver failure and liver cancer occur at much-reduced rates when treated early enough with nitisinone. |
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