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Sephience: FDA approved
treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU)
Kuvan: FDA approved
Indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet.
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
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Patient Assistance Programs2
Palynziq
BioMarin Pharmaceutical Inc.
Sephience
PTC Therapeutics Inc.
Palynziq
(PEGVALIASE-PQPZ)Orphan drugBioMarin Pharmaceutical Inc.
Sephience
(SEPIAPTERIN)Orphan drugPTC Therapeutics Inc.
Kuvan
(sapropterin)Orphan drugBioMarin Pharmaceutical, Inc.
12.1 Mechanism of Action KUVAN is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through a...