Saudi FDA Approves Sobi’s Orfadin Capsules

NSwedish Orphan Biovitrum AB (Sobi), has announces that the Saudi Food and Drug Administration (SFDA) has approved 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. People with hereditary tyrosinemia type 1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver, renal and neurological complications. Approximately 1,000 persons worldwide are identified as living with HT-1.

Orfadin (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine by-products in the body. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine is not adequately broken down. Orfadin is a proprietary product and is developed by and made available globally by Sobi.
Before Orfadin became available, the survival rate in HT-1 was 29 per cent after two years for children who developed symptoms before two months of age. After the introduction of Orfadin, the survival rate is 93 per cent after two years in patients with treatment initiation before two months of age.