ZELVYSIA (sapropterin dihydrochloride) by Pfizer is bh4, the cofactor for the enzyme phenylalanine hydroxylase (pah). Approved for blood phenylalanine (phe) levels in adult, older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin-(bh4-) responsive phenylketonuria (pku). First approved in 2025.
Drug data last refreshed 19h ago
BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of…
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PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC
The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease