The Ministry of Health of the Russian Federation has announced the registration of Sapropterin PSK, the first Russian drug based on sapropterin, a synthetic cofactor used in the treatment of phenylketonuria (PKU). Developed by PSK Pharma, a member of the Rus Biopharm group, this drug has been included in the list of vital and essential medicines (VED) and will be available in 100 mg tablet form, the press service of the organization told GxP News. According to the manufacturer, the drug is manufactured at the production site in Dubna in accordance with GMP requirements.
About 1 in 10,000 to 15,000 babies is born with PKU, an inborn error of metabolism that results in decreased metabolism of the amino acid phenylalanine. Due to a gene mutation, the body is unable to process this amino acid, which leads to the accumulation of toxic metabolic products.
Sapropterin is a synthetic form of the naturally occurring enzyme cofactor that is responsible for the conversion of phenylalanine to another amino acid, tyrosine. Sapropterin prevents the pathological accumulation of phenylalanine and its metabolites in the blood, which helps reduce the risk of serious concomitant complications.
Evgeniia Shapiro, General Director of PSK Pharma, noted that in 2024, the Russian market for public procurement of sapropterin reached 3,200 packages.
