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.
MDK-Nitisinone is used to treat hereditary tyrosinemia type 1 in combination with adherence to a reduced tyrosine dietary regime. MDK-Nitisinone’s mechanism of action involves reversible inhibition of 4-Hydroxyphenylpyruvate dioxygenase (HPPD). Use of the drug will prevent the formation of maleylacetoacetic acid and fumarylacetoacetic acid, which have the potential to be converted to succinyl acetone, a toxin that damages the liver and kidneys.
Currently, MDK-Nitisinone is only approved for use in Canada, and is not yet commercially available in any other jurisdiction.
MendeliKABS is a pharmaceutical company dedicated to the treatment of rare diseases. The organization integrates expertise in treatment of orphan diseases and drugs manufacturing, in compliance with international regulations and standards.
Inspired by the principle of offering premium quality affordable solutions to persons afflicted with hereditary tyrosinemia type 1, MendeliKABS’ vision embraces expanded access to orphan drugs in order to effectively meet the needs of patients affected by rare diseases. Produced in three dosages, 2 mg, 5 mg and 10 mg, MDK-Nitisinone is manufactured in Quebec, Canada.
Since its introduction in 1991, this treatment has replaced liver transplantation as a first line treatment for patients with this rare condition. Classified as an orphan disease, hereditary tyrosinemia type 1 affects one person out of 200,000 worldwide, but is especially prevalent in the province of Québec, Canada, with about one hundred persons affected.
About WEP Clinical
WEP Clinical is a rapidly growing niche pharmaceutical services company focused on providing patients and health institutions access to much needed drugs through Expanded Access Programs (EAP) and Named Patient Programs (NPP). The EU Headquarters were established in London in 2008, followed by the US Headquarters based in Research Triangle Park, North Carolina, in 2012.
The operational and leadership team has many years of pharmaceutical, clinical, and regulatory experience gained in companies such as Pfizer, AZ, MSD, GSK, UDG Healthcare, Waymade, Sanofi, Quintiles and Walgreens Boots Alliance. The facilities have been inspected and licensed by the MHRA, UK Home Office, FDA and all necessary local approval bodies.
WEP Clinical is compliant with all relevant GMDP regulations and operates to the highest standards of medicines supply. The company operates under a comprehensive Quality Management System with robust and compliant standard operating procedures (SOPs) and policies.
WEP Clinicals customers and partners include Pharma, Biotechs, Generic Manufacturers, CROs and Healthcare institutional providers. The company is fully committed to a culture of continual development and strive to service our partners needs beyond their expectations.
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