WEP Clinical, a global pharmaceutical and services company, and MendeliKABS Inc., a Canadian pharmaceutical company dedicated to the treatment of rare diseases, announce an exclusive Named Patient agreement to commercialise MDK-Nitisinone (nitisinone), a drug for the treatment of hereditary tyrosinemia type 1, in a number of countries where the product has no valid Marketing Authorization in place.
MDK-Nitisinone is a Health Canada approved drug which is indicated for the treatment of patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
Tyrosinemia type I 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. MDK-Nitisinone stops the build-up of toxic substances which cause the severe liver and kidney problems in patients with HT-1. By doing that, it also prevents the porphyric crises associated with HT-1.
Under the terms of the agreement, WEP Clinical will manage any requests from physicians under a Named Patient Program in countries where the product is currently unlicensed.