NITISINONE MDK

What is Nitisinone MDK?

Nitisinone (2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione, or NTBC) is an inhibitor of the 4- hydroxyphenyl-pyruvate dioxygenase (HDDP), an enzyme involved in tyrosine degradation. The product is presented as hard capsules containing 2 mg, 5 mg, or 10 mg of nitisinone as the active substance. Nitisinone MDK® is a ‘generic medicine’, meaning it contains the same active substance and works in the same way as the ‘reference medicine’ already authorized called Orfadin®.

What is Nitisinone MDK approved for?

Nitisinone MDK is indicated for treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

What is Hereditary Tyrosinemia TYPE 1 (HT-1)?

HT-1 is a rare autosomal recessive genetic metabolic disorder characterized by lack of the enzyme fumarylacetoacetate hydrolase (FAH), which is needed for the final break down of the amino acid tyrosine. Failure to properly break down tyrosine leads to abnormal accumulation of tyrosine and its metabolites in the liver, potentially resulting in severe liver disease. Tyrosine may also accumulate in the kidneys and central nervous system.

Marketing Authorizations

Nitisinone MDK was granted marketing authorization by the EMA in August 2017. The EMA concluded that the product has comparable quality and is bioequivalent to reference product (Orfadin). Therefore, the Agency’s view was that, as for Orfadin, the benefit outweighs the identified risk.

This product was previously approved by Health Canada in September 2016, as MDK-Nitisinone.