The Medication(s) listed below have been approved by the Food & Drug Administration (FDA) as orphan products for treatment of this condition. There may be other products available
that are not considered orphan products.
Nitisinone (Brand name: Orfadin®)
- Manufactured by
Swedish Orphan AB
FDA-approved indication: Adjunctive therapy to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1. Available in capsule from since Januaray 2009. Available in oral suspension since April 2016.
National Library of Medicine Drug Information Portal