
ROCKVILLE, Maryland-The US Food and Drug Administration (FDA) has approved the orphan drug Orfadin (nitisinone capsules, Swedish Orphan International) for treating hereditary tyrosinemia type 1 (HT-1), a rare pediatric disease that causes progressive liver failure and liver cancer. In announcing its approval decision, the agency warned that only physicians experienced in treating the disease should prescribe Orfadin. Rare Diseases Therapeutics, Inc., of Nashville, Tennessee, is the drug’s US distributor.