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BioMarin Announces Positive Pivotal Data for PALYNZIQ® (pegvaliase-pqpz) in Adolescents with Phenylketonuria

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BioMarin Pharmaceutical (Nasdaq: BMRN) announced positive Phase 3 PEGASUS trial results for PALYNZIQ® (pegvaliase-pqpz) in treating adolescents with phenylketonuria (PKU). The study met its primary efficacy endpoint, showing statistically significant reduction in blood Phe levels among patients aged 12-17 compared to diet alone.

The safety profile aligned with known results, and PALYNZIQ remains the first and only enzyme therapy approved for adult PKU treatment. BioMarin plans to present detailed findings at an upcoming medical meeting and will submit to global health authorities later this year to expand PALYNZIQ's label for adolescent use.

BioMarin Pharmaceutical (Nasdaq: BMRN) ha annunciato risultati positivi della fase 3 dello studio PEGASUS per PALYNZIQ® (pegvaliase-pqpz) nel trattamento degli adolescenti affetti da fenilchetonuria (PKU). Lo studio ha raggiunto il suo obiettivo primario di efficacia, mostrando una riduzione statisticamente significativa dei livelli di Phe nel sangue tra i pazienti di età compresa tra 12 e 17 anni rispetto alla sola dieta.

Il profilo di sicurezza è in linea con i risultati noti e PALYNZIQ rimane la prima e unica terapia enzimatica approvata per il trattamento della PKU negli adulti. BioMarin prevede di presentare risultati dettagliati in un prossimo incontro medico e presenterà domanda alle autorità sanitarie globali entro la fine di quest'anno per ampliare l'etichetta di PALYNZIQ per l'uso negli adolescenti.

BioMarin Pharmaceutical (Nasdaq: BMRN) anunció resultados positivos del ensayo PEGASUS de fase 3 para PALYNZIQ® (pegvaliase-pqpz) en el tratamiento de adolescentes con fenilcetonuria (PKU). El estudio cumplió con su objetivo primario de eficacia, mostrando una reducción estadísticamente significativa en los niveles de Phe en sangre entre los pacientes de 12 a 17 años en comparación con la dieta sola.

El perfil de seguridad se alineó con los resultados conocidos, y PALYNZIQ sigue siendo la primera y única terapia enzimática aprobada para el tratamiento de la PKU en adultos. BioMarin planea presentar hallazgos detallados en una próxima reunión médica y presentará a las autoridades sanitarias globales más adelante este año para ampliar la etiqueta de PALYNZIQ para su uso en adolescentes.

BioMarin Pharmaceutical (Nasdaq: BMRN)은 페닐케톤뇨증(PKU) 청소년 치료를 위한 PALYNZIQ® (pegvaliase-pqpz)의 3상 PEGASUS 시험 결과가 긍정적이라고 발표했습니다. 이 연구는 주요 효능 목표를 달성했으며, 12세에서 17세 환자에서 단순한 식이요법과 비교하여 혈중 Phe 수치의 통계적으로 유의미한 감소를 보여주었습니다.

안전성 프로필은 알려진 결과와 일치하며, PALYNZIQ는 성인 PKU 치료를 위해 승인된 첫 번째이자 유일한 효소 치료제입니다. BioMarin은 다가오는 의료 회의에서 자세한 결과를 발표할 계획이며, 올해 말에는 청소년 사용을 위한 PALYNZIQ의 라벨을 확대하기 위해 글로벌 보건 당국에 제출할 예정입니다.

BioMarin Pharmaceutical (Nasdaq: BMRN) a annoncé des résultats positifs de l'essai PEGASUS de phase 3 pour PALYNZIQ® (pegvaliase-pqpz) dans le traitement des adolescents atteints de phénylcétonurie (PKU). L'étude a atteint son objectif principal d'efficacité, montrant une réduction statistiquement significative des niveaux de Phe dans le sang chez les patients âgés de 12 à 17 ans par rapport à un régime alimentaire seul.

Le profil de sécurité était conforme aux résultats connus, et PALYNZIQ reste la première et unique thérapie enzymatique approuvée pour le traitement de la PKU chez les adultes. BioMarin prévoit de présenter des résultats détaillés lors d'une prochaine réunion médicale et soumettra aux autorités sanitaires mondiales plus tard cette année une demande pour élargir l'étiquette de PALYNZIQ à l'utilisation chez les adolescents.

BioMarin Pharmaceutical (Nasdaq: BMRN) hat positive Ergebnisse der Phase-3-Studie PEGASUS für PALYNZIQ® (pegvaliase-pqpz) zur Behandlung von Jugendlichen mit Phenylketonurie (PKU) bekannt gegeben. Die Studie erreichte ihr primäres Wirksamkeitsziel und zeigte eine statistisch signifikante Reduktion der Blut-Phe-Spiegel bei Patienten im Alter von 12 bis 17 Jahren im Vergleich zur alleinigen Diät.

Das Sicherheitsprofil entsprach den bekannten Ergebnissen, und PALYNZIQ bleibt die erste und einzige Enzymtherapie, die für die Behandlung von PKU bei Erwachsenen zugelassen ist. BioMarin plant, detaillierte Ergebnisse auf einer bevorstehenden medizinischen Tagung zu präsentieren und wird später in diesem Jahr bei den globalen Gesundheitsbehörden einen Antrag stellen, um das Label von PALYNZIQ für die Anwendung bei Jugendlichen zu erweitern.

Positive
  • Phase 3 PEGASUS trial met primary efficacy endpoint
  • Statistically significant reduction in blood Phe levels vs diet alone
  • Potential market expansion to adolescent population (ages 12-17)
  • Safety results consistent with known profile
Negative
  • None.

Company plans to submit for regulatory approval later this year

SAN RAFAEL, Calif., April 2, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone. Safety results were consistent with the known profile of the medicine. PALYNZIQ is the first and only enzyme therapy approved to treat adults with PKU.

Detailed results from the PEGASUS study will be presented at an upcoming medical meeting and submitted to global health authorities starting later this year to request a label expansion for PALYNZIQ to include adolescents.

"For more than two decades, BioMarin has made strides for people with PKU – pioneering the first treatment, bringing a meaningful new option in PALYNZIQ, and continuing to innovate as we advance our research pipeline. We are encouraged to see these positive data that build on that legacy and show how PALYNZIQ can make an impact for adolescents as they begin their transition to adult living," said Greg Friberg, M.D., executive vice president and chief research & development officer at BioMarin. "We look forward to sharing these data with the scientific community and to submitting them to global regulators with the goal of making PALYNZIQ available for younger people living with PKU."

About PEGASUS

PEGASUS is a Phase 3 multi-center open-label randomized controlled study evaluating the safety and efficacy of PALYNZIQ compared to diet alone in 55 adolescents aged 12-17 with phenylketonuria. The primary endpoints are change in blood Phe concentration and characterization of the safety profile in adolescents. Secondary endpoints include change in total dietary protein intake and pharmacokinetics. 

The study is being conducted in two parts: the primary treatment phase ranging from weeks 1-73 (Part 1), and the extension phase (Part 2), which lasts for up to an additional 80 weeks of monitoring for the PALYNZIQ arm and allows for crossover for those in the diet-only arm.

For more information, please visit clinicaltrials.biomarin.com

About PALYNZIQ

PALYNZIQ substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. PALYNZIQ is administered using a dosing regimen designed to facilitate tolerability; 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.

PALYNZIQ is approved to reduce blood Phe concentrations for adults in the U.S., for people 16 and older in the EU, Canada and Brazil, and for people 15 and older in Japan with PKU who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.

Patient Support Accessing PALYNZIQ

To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-866-906-6100 or e-mail support@biomarin-rareconnections.com. For more information about PALYNZIQ, please visit www.palynziq.com. For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic condition affecting approximately 70,000 people in the regions of the world where BioMarin operates. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If functional enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms.

As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU born after this period in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after.

PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most individuals to adhere to the lifelong strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

PALYNZIQ U.S. Indication and Important Safety Information

PALYNZIQ® (pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater than 600 micromol/L on existing management.

BOXED WARNING: RISK OF ANAPHYLAXIS

  • Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment
  • Administer the initial dose of PALYNZIQ under the supervision of a healthcare provider equipped to manage anaphylaxis, and closely observe patients for at least 60 minutes following injection. Prior to self-injection, confirm patient competency with self-administration, and patient's and observer's (if applicable) ability to recognize signs and symptoms of anaphylaxis and to administer auto-injectable epinephrine, if needed
  • Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use
  • Prescribe auto-injectable epinephrine. Prior to the first dose, instruct the patient and observer (if applicable) on its appropriate use. Instruct the patient to seek immediate medical care upon its use. 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). Further information, including a list of qualified pharmacies, is available at www.PALYNZIQREMS.com or by telephone at 1-855-758-REMS (1-855-758-7367)

WARNINGS AND PRECAUTIONS

Anaphylaxis

  • Signs and symptoms of anaphylaxis reported include syncope, hypotension, hypoxia, dyspnea, wheezing, chest discomfort/chest tightness, tachycardia, angioedema (swelling of face, lips, eyes, tongue), throat tightness, skin flushing, rash, urticaria, pruritus, and gastrointestinal symptoms (vomiting, nausea, diarrhea)
  • Anaphylaxis generally occurred within 1 hour after injection; however, delayed episodes occurred up to 48 hours after PALYNZIQ administration
  • Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use
  • Anaphylaxis requires immediate treatment with auto-injectable epinephrine. Prescribe auto-injectable epinephrine to all patients receiving PALYNZIQ and instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment. Prior to the first dose, instruct the patient and observer (if applicable) on how to recognize the signs and symptoms of anaphylaxis, how to properly administer auto-injectable epinephrine, and to seek immediate medical care upon its use. Consider the risks associated with auto-injectable epinephrine use when prescribing PALYNZIQ. Refer to the auto-injectable epinephrine prescribing information for complete information
  • Consider the risks and benefits of readministering PALYNZIQ following an episode of anaphylaxis. If the decision is made to readminister PALYNZIQ, administer the first dose under the supervision of a healthcare provider equipped to manage anaphylaxis and closely observe the patient for at least 60 minutes following the dose. 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

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, nausea, abdominal pain, vomiting, cough, oropharyngeal pain, pruritus, diarrhea, nasal congestion, fatigue, dizziness, and anxiety
  • Of the 285 patients exposed to PALYNZIQ in an induction/titration/maintenance regimen in clinical trials, 44 (15%) patients discontinued treatment due to adverse reactions. The most common adverse reactions leading to treatment discontinuation were hypersensitivity reactions (6% of patients) including anaphylaxis (3% of patients), angioedema (1% of patients), arthralgia (4% of patients), generalized skin reactions lasting at least 14 days (2% of patients), and injection site reactions (1% of patients)
  • The most common adverse reactions leading to dosage reduction were arthralgia (15% of patients), hypersensitivity reactions (9% of patients), injection site reactions (4% of patients), alopecia (3% of patients), and generalized skin reactions lasting at least 14 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), and injection site reactions (4% of patients)
  • Angioedema and serum sickness: In clinical trials, 22 out of 285 (8%) patients experienced 45 episodes of angioedema (symptoms included: pharyngeal edema, swollen tongue, lip swelling, mouth swelling, eyelid edema, and face edema) occurring independent of anaphylaxis. In clinical trials, serum sickness was reported in 7 out of 285 (2%) patients

Blood Phenylalanine Monitoring and Diet

  • Obtain blood Phe levels every 4 weeks until a maintenance dosage is established. Periodically monitor blood Phe levels during maintenance therapy
  • Counsel patients to monitor dietary protein and Phe intake, and adjust as directed by their healthcare provider

DRUG INTERACTIONS

Effect of PALYNZIQ on Other PEGylated Products

  • In a single-dose study of PALYNZIQ in adult patients with PKU, two patients receiving concomitant injections of medroxyprogesterone acetate suspension (a formulation containing PEG 3350) experienced a hypersensitivity reaction. One of the two patients experienced anaphylaxis
  • The clinical effects of concomitant treatment with different PEGylated products is unknown. Monitor patients treated with PALYNZIQ and concomitantly with other PEGylated products for hypersensitivity reactions including anaphylaxis

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

  • PALYNZIQ may cause fetal harm when administered to a pregnant woman
  • Advise women who are exposed to PALYNZIQ during pregnancy or who become pregnant within one month following the last dose of PALYNZIQ that there is a pregnancy surveillance program that monitors pregnancy outcomes. Healthcare providers should report PALYNZIQ exposure and encourage these patients to report their pregnancy to BioMarin (1-866-906-6100)
  • Monitor blood Phe levels in breastfeeding women treated with PALYNZIQ

Pediatric Use

  • The safety and effectiveness of PALYNZIQ in pediatric patients have not been established

Geriatric Use

  • Clinical studies of PALYNZIQ did not include patients aged 65 years and older

You are encouraged to report suspected adverse reactions to BioMarin at 1-866-906-6100, or to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see accompanying full Prescribing Information, including Boxed Warning.

About BioMarin

BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the Phase 3 PEGASUS trial evaluating PALYNZIQ, including plans to present results at an upcoming medical conference; the safety profile and potential benefit of PALYNZIQ for adolescents, including ability to lower blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone; the development of BioMarin's PALYNZIQ program generally, including plans to submit detailed results to global health authorities later this year to request a label expansion for PALYNZIQ to include adolescents with the goal of making PALYNZIQ available to younger people living with PKU; and the continued clinical development of PALYNZIQ. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of PALYNZIQ; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2024, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, BioMarin RareConnections® and PALYNZIQ® are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:




Investors

Media

Traci McCarty 

Katherine Powell

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558 

(415) 827-2968

 

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SOURCE BioMarin Pharmaceutical Inc.

FAQ

What were the results of BioMarin's (BMRN) Phase 3 PEGASUS trial for PALYNZIQ?

The trial met its primary efficacy endpoint, showing statistically significant reduction in blood Phe levels in adolescents aged 12-17 with PKU compared to diet alone.

When will BioMarin (BMRN) submit PALYNZIQ for adolescent approval?

BioMarin plans to submit to global health authorities later in 2025 to request label expansion for adolescent use.

What age group is currently approved for BioMarin's (BMRN) PALYNZIQ treatment?

PALYNZIQ is currently approved only for adults with PKU, as the first and only enzyme therapy for this population.

What is the safety profile of PALYNZIQ in BioMarin's (BMRN) adolescent trial?

Safety results in the Phase 3 PEGASUS trial were consistent with the known profile of the medicine.
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