Saudi Food and Drug Administration (SFDA) has approved Swedish Orphan Biovitrum AB (Sobi)’s Orfadin (nitisinone) capsules in all strengths (2 mg, 5 mg, 10 mg and 20 mg) for the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. HT-1 is a progressive, rare genetic disease that may result in liver and kidney complications and in most cases fatal if untreated. In the most common form of the disease, symptoms arise within the first six months of the child’s life.
Significance of the approval:
- The Kingdom of Saudi Arabia is the largest market in the Middle East.
- This approval will open opportunity to expansion in entire middle east countries.
- Nitisinone, also known as NTBC is a medication used to slow the effects of hereditary tyrosinemia type 1. Since its first use for this indication in 1991, it has replaced liver transplantation as the first-line treatment for this rare condition.
- It is also being studied in the related condition alkaptonuria.
- It is marketed under the brand name Orfadin by the company Swedish Orphan Biovitrum (Sobi); it was first brought to market by Swedish Orphan International. It was originally developed as a candidate herbicide.