{"id":828059,"date":"2025-03-20T09:09:42","date_gmt":"2025-03-20T13:09:42","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/biomarin-presents-new-data-demonstrating-favorable-safety-and-strong-adherence-in-real-world-clinical-practice-with-voxzogo-vosoritide-in-children-under-3-with-achondroplasia-at-2025-american\/"},"modified":"2025-03-20T09:09:42","modified_gmt":"2025-03-20T13:09:42","slug":"biomarin-presents-new-data-demonstrating-favorable-safety-and-strong-adherence-in-real-world-clinical-practice-with-voxzogo-vosoritide-in-children-under-3-with-achondroplasia-at-2025-american","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/biomarin-presents-new-data-demonstrating-favorable-safety-and-strong-adherence-in-real-world-clinical-practice-with-voxzogo-vosoritide-in-children-under-3-with-achondroplasia-at-2025-american\/","title":{"rendered":"BioMarin Presents New Data Demonstrating Favorable Safety and Strong Adherence in Real-World Clinical Practice with VOXZOGO\u00ae (vosoritide) in Children Under 3 with Achondroplasia at 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting"},"content":{"rendered":"
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PR Newswire<\/p>\n<\/p><\/div>\n

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\n New data also to be presented for PALYNZIQ\u00ae<\/sup> (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with phenylketonuria<\/i>\n <\/p>\n

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\n SAN RAFAEL, Calif.<\/span>
\n <\/span>, March 20, 2025<\/span><\/span> \/PRNewswire\/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO\u00ae<\/sup>\u00a0<\/i>(vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ\u00ae\u00a0<\/sup><\/i>(pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established safety profiles of both medicines, which continue to play a pivotal role for people living with these genetically defined conditions.<\/p>\n

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\n \"BioMarin
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The data will be presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Los Angeles<\/span>.<\/p>\n

\n Data Highlight VOXZOGO Treatment Adherence and Favorable Safety Profile in Infants<\/b>\n <\/p>\n

New data from a study in Japan<\/span> showed strong treatment adherence with VOXZOGO in children with achondroplasia under the age of 3, with no reported treatment-related adverse events nor any dose interruptions among 63 children followed for up to 23.7 months. These real-world findings validate VOXZOGO’s established safety profile and reinforce the therapeutic benefit seen in clinical studies. The study’s safety results, including in infants as young as 1 month old, add to the growing body of evidence supporting early treatment initiation.<\/p>\n

“VOXZOGO is the first and only approved treatment for children with achondroplasia, and it is encouraging to see favorable safety and strong adherence in very young children receiving the medicine in real-world clinical practice. As the effects of restricted growth are apparent from birth in children with achondroplasia, we expect that earlier treatment should translate to greater benefits,” said Greg Friberg<\/span>, M.D., executive vice president and chief research & development officer at BioMarin. “In addition to data in achondroplasia, scientific research at ACMG furthers our genetic understanding of skeletal conditions like hypochondroplasia, in which we hope to complete enrollment for our pivotal study with VOXZOGO in the first half of 2025.”<\/p>\n

\n PALYNZIQ Data Show Importance of Sustained Blood Phe Level Reduction and Improvement in Quality of Life in Adults with PKU<\/b>\n <\/p>\n

New data from the OPAL study, a post-marketing observational trial to assess the real-world safety and efficacy of PALYNZIQ, showed lowered blood Phe levels following treatment and positive health-related quality of life (HRQoL) outcomes. Mean blood Phe level was 1029 \u03bcmol\/L at baseline (n=51) and lowered to 293 \u03bcmol\/L at week 96 (n=16), representing a 67.8% reduction. Improvements in HRQoL were also observed, as measured by the PKU quality of life questionnaire (PKU-QOL) and the PKU Symptom Severity and Impacts Scale (PKU-SSIS), which use a scale of 1-100, with lower scores indicating better\u00a0HRQoL. Mean change from baseline of -12.4 and -11.8 respectively were observed over the same time period.<\/p>\n

Additionally, secondary data analyses from the Phase 3 PRISM clinical trial program demonstrated that sustaining lowered blood Phe\u00a0levels with PALYNZIQ led to improvements in attention and mood. Most notably, scores for sustained Phe\u00a0levels \u2264120 were significantly better than those at sustained levels of \u2264600 or \u2264360, suggesting blood Phe\u00a0in the normal range may provide additional benefit for adults living with PKU.<\/p>\n

Real-world data from the Assessment of the Treatment and management LAndScape of PKU (ATLAS) study also highlighted a shift in the treatment landscape in part attributable to PALYNZIQ, with an increased proportion of people with PKU treated across 19 U.S. clinics achieving blood Phe levels \u2264360 \u03bcmol\/L and a reduction in the proportion of individuals with blood Phe levels >1200 \u03bcmol\/L.<\/p>\n

These findings demonstrate PALYNZIQ’s efficacy in treating PKU, as the company works to advance this option for younger people living with the genetically defined condition, with results from a Phase 3 study in adolescents expected later this year.<\/p>\n

Below are BioMarin’s key presentations at ACMG, all listed in Pacific Time:<\/p>\n

\n Interdisciplinary Variant Re-Classification: FGFR3<\/i> as an Example of Genotypic Investigation in Suspected Skeletal Dysplasia Population
<\/b>Poster #P213
Thursday, March 20<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n How Helpful are Sleep Studies in Determining Surgical Need in Infants with Achondroplasia?
<\/b>Poster #P313
Thursday, March 20<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Beyond Boundaries: The Continuous Spectrum in FGFR3<\/i>-Related Conditions
<\/b>Poster #P373
Thursday, March 20<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Index of Sustained Phe Response and Improvements in PKU Clinical Outcome Assessments in Patients Receiving Pegvaliase
<\/b>Poster #P009
Thursday, March 20<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Initial Psychometric Evaluation of the Adult Symptom Severity and Impacts Scale (PKU-SSIS) Using Interim Data from the OPAL Study
<\/b>Poster #P029
Thursday, March 20<\/span>, 10:30 \u2013 11:30 a.m.\u00a0<\/p>\n

\n The Assessment of the Treatment and Management Landscape of Phenylketonuria Survey Study: Findings from 19 Clinics in the United States<\/span>
<\/b>Poster #P045
Thursday, March 20<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Safety Profile and Adherence of Vosoritide in Young Children with Achondroplasia in Japan<\/span>
<\/b>Poster #P208
Friday, March 21<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Vosoritide as a Targeted Therapy for FGFR3<\/i>-Related Thanatophoric Dysplasia
<\/b>Poster #P354
Friday, March 21<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Improvements in Blood Phenylalanine and Health-Related Quality of Life Outcomes Among Adults with PKU Receiving Pegvaliase in the OPAL Study
<\/b>Poster #P002
Friday, March 21<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n Occurrence of Anaphylaxis in Adult Incident Pegvaliase-Treated PKU Patients in a Post-Marketing Safety Analysis in the United States<\/span>
<\/b>Poster #P046
Friday, March 21<\/span>, 10:30 \u2013 11:30 a.m.<\/span><\/p>\n

\n About Achondroplasia<\/b>\n <\/p>\n

Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3<\/i> gene, a negative regulator of bone growth.<\/p>\n

More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still “open,” typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.<\/p>\n

\n About Phenylketonuria<\/b>\n <\/p>\n

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.<\/p>\n

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.<\/p>\n

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.\u00a0Dietary 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.<\/p>\n

\n About VOXZOGO<\/b>\n <\/p>\n

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3<\/i>. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3<\/i> to promote endochondral bone growth.<\/p>\n

VOXZOGO is approved in the U.S., Japan<\/span> and Australia<\/span> to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.<\/p>\n

\n Patient Support Accessing VOXZOGO<\/b>\n <\/p>\n

To reach a BioMarin RareConnections\u00ae<\/sup> Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail VOXZOGOSupport@biomarin-rareconnections.com<\/a><\/u>. For more information about VOXZOGO, please visit www.voxzogo.com<\/a><\/u>. For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com<\/a><\/u>.<\/p>\n

\n About\u00a0PALYNZIQ<\/b>\n <\/p>\n

PALYNZIQ\u00a0substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe.\u00a0PALYNZIQ 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.<\/p>\n

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

\n Patient Support Accessing PALYNZIQ<\/b>\n <\/p>\n

To reach a BioMarin RareConnections\u00ae<\/sup> Case Manager, please call, toll-free, 1-866-906-6100 or e-mail\u00a0support@biomarin-rareconnections.com<\/a><\/u>. For more information about PALYNZIQ, please visit\u00a0www.palynziq.com<\/a><\/u>. For additional information regarding this product, please contact BioMarin Medical Information at\u00a0medinfo@bmrn.com<\/a><\/u>.<\/p>\n

\n VOXZOGO U.S. Important Safety Information<\/b>\n <\/p>\n

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\n What is VOXZOGO used for?<\/u>
\n <\/b>\n <\/p>\n