Slovakia – Medicinal products for the alimentary tract and metabolism – Liek s účinnou látkou cerliponáza alfa 150 mg, infúzny roztok

The subject of the contract is the drug with the active substance cerliponase alfa 150 mg, infusion solution. Drug form: sol inf 5 ml/150 mg+5 ml diluent (inj. vials). Administration: intracerebroventricular use. The drug cerliponase alfa belongs to the group of drugs known as enzyme replacement therapies. It is used to treat patients with the disease neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidylpeptidase 1 (TPP1) deficiency. People with CLN2 disease do not have the enzyme called TPP1 or have too little of it, which causes the accumulation of substances called lysosomal storage materials. In people with CLN2 disease, these substances accumulate in certain parts of the body, mainly in the brain. This drug replaces the missing enzyme called TPPA, thereby minimizing the accumulation of lysosomal storage materials. This drug works by slowing the progression of the disease. The recommended dose is 300 mg of cerliponase alfa, which is administered once every two weeks by intracerebroventricular infusion.