BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the company's PALYNZIQ® (pegvaliase-pqpz) supplemental Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2026.
These data were recently presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan. PALYNZIQ's safety profile consists primarily of immune-mediated responses, which can include anaphylaxis, for which robust risk management measures effective in clinical trials are in place. instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment.
PALYNZIQ is available only through a restricted program called PALYNZIQ REMS (Risk Evaluation and Mitigation strategy). Subsequent PALYNZIQ dose titration should be based on patient tolerability and therapeutic response. Consider premedication with an H1-receptor antagonist, H2-receptor antagonist, and/or antipyretic prior to PALYNZIQ administration based upon individual patient tolerability.
Other Hypersensitivity Reactions: Hypersensitivity reactions other than anaphylaxis have been reported in 204 of 285 (72%) patients treated with PALYNZIQ in clinical trials; Management of hypersensitivity reactions should be based on the severity of the reaction, recurrence of the reaction, and the clinical judgment of the healthcare provider, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids Injection Site Infections: Serious injection site infections including abscess, cellulitis, necrosis, and ulcer have been reported. Advise patients not to administer PALYNZIQ into the affected area until the infection has resolved. ADVERSE REACTIONS: The most common adverse reactions (at least 20% of patients in either treatment phase) were injection site reactions, arthralgia, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days (2% of patients); The most common adverse reactions leading to dosage reduction were arthralgia (15% of patients), injection site reactions (4% of patients), alopecia (3% of patients), and generalized skin reactions lasting at least14 days (2% of patients).
The most common adverse reactions leading To temporary drug interruption were hypersensitivity reactions (14% of patients), arthralgia (13% of patients), anaphylaxis (4% of patients), anaphyretics, and injection site reactions (4%of patients); Angioedema and serum sickness: In clinical trials, 22 out of 285 (8%) patients experienced 45 episodes ofangioedema (sym symptoms included: pharyngeal edema, swollen tongue, lip swelling, mouth swelling, eyelid edema, and face edema) occurring independent of anaphylaxis. In clinical trials, serum reactions, serum reactions, serum reactions (4% of patients; and serum sickness: In clinical trial, serum reactions, serum reactions were observed in the U.S. and serum sickness: In clinical studies, 22 out of 285 (8%) patients experienced 45 episodes of angioedema (sym symptoms include: pharyngeal edem, swollen tongue, lip swelling), lip swelling, mouth swelling, eyeid edema, and face Edema) occurring independent of anphylaxis. In clinical trials.
In clinical trials, serum samples, serum reactions, and serum sickness, serum samples were observed in patients with anaphylaxis.
