This section highlights FDA-related milestones and regulatory updates for drugs developed by BioMarin Pharmaceutical (BMRN).
Over the past two years, BioMarin Pharmaceutical has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
vosoritide, PALYNZIQ, Valoctocogene, and BRINEURA®. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Vosoritide FDA Regulatory Timeline and Events
Vosoritide is a drug developed by BioMarin Pharmaceutical for the following indication: Children with achondroplasia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- vosoritide
- Announced Date:
- May 2, 2026
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical Inc announced new research from studies of VOXZOGO® (vosoritide) in children with achondroplasia demonstrating positive impact on important health indicators, including arm span and bone density..
AI Summary
BioMarin reported new research showing long-term treatment with VOXZOGO (vosoritide) in children with achondroplasia produces meaningful health improvements. Studies presented evidence of increased arm span and gains in bone density, indicating benefits beyond height alone. Researchers also observed improvements in other clinical measures tied to skeletal health, suggesting sustained vosoritide treatment can positively affect growth and bone quality during key developmental years.
The company will also present data on VOXZOGO in hypochondroplasia, including a 12-month study that showed significant increases in bone mineral density and content. BioMarin plans to share pivotal Phase 3 topline results for hypochondroplasia in the first half of 2026, which could support broader use if outcomes are favorable. These findings highlight ongoing research into how vosoritide may improve multiple aspects of bone health for children with these conditions.
Read Announcement- Drug:
- vosoritide
- Announced Date:
- March 12, 2026
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical Inc. announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia in ongoing clinical trials and real-world studies.
AI Summary
BioMarin announced new positive data for VOXZOGO (vosoritide) from ongoing clinical trials and real‑world studies in children with achondroplasia. Children who began treatment before age 2 showed durable, multi‑year improvements versus untreated peers, including better body proportionality, increased arm span, maintained arm‑to‑height ratios, and sustained height gains. Early treatment was also linked to significant improvements in body mass index. Researchers noted these benefits persisted during early life when growth plates are still open.
Real‑world evidence from Europe, Japan and the U.S. supports the trial findings: more than 5,000 infants and children across 50+ countries have received VOXZOGO, contributing to over 10,000 patient‑years of data. BioMarin says VOXZOGO is the only approved treatment for children starting at birth and has the most extensive published clinical evidence among achondroplasia therapies, including measures beyond height. Detailed results will be presented at the upcoming ACMG meeting.
Read Announcement- Drug:
- vosoritide
- Announced Date:
- May 12, 2025
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical Inc. announced new data from studies of VOXZOGO® (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions.
AI Summary
BioMarin recently announced promising new study data on VOXZOGO (vosoritide), which showed a meaningful impact on tibial bowing in children with achondroplasia. In the Phase 2 CANOPY clinical studies, young patients treated with VOXZOGO experienced a significant and sustained reduction in tibial bowing—a complication that often leads to pain and surgical intervention. This improvement, observed over several years of treatment, highlights VOXZOGO’s potential to improve physical function and overall quality of life by addressing issues beyond height growth. Additionally, early investigational efficacy was noted in other skeletal conditions, including hypochondroplasia and Turner syndrome, indicating broader therapeutic benefits. These findings support the value of early and continuous treatment with VOXZOGO and offer encouraging prospects for children living with these challenging skeletal dysplasias.
Read Announcement- Drug:
- vosoritide
- Announced Date:
- March 20, 2025
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU).
AI Summary
BioMarin Pharmaceutical recently revealed promising new study results for two of its treatments. Data for VOXZOGO® (vosoritide) showed that young children with achondroplasia, including infants as young as one month, maintained strong treatment adherence with no reported treatment-related adverse events. These findings support its safety profile and reinforce its benefits for children affected by this skeletal condition. Researchers are also continuing to explore the use of VOXZOGO in clinical trials for other skeletal disorders, such as hypochondroplasia.
Additionally, new results for PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU) have been encouraging. In the OPAL study, PALYNZIQ demonstrated a significant reduction in blood phenylalanine levels and improvements in health-related quality of life. These outcomes underscore the long-term value and effectiveness of PALYNZIQ for managing PKU.
Read Announcement - Drug:
- vosoritide
- Announced Date:
- September 18, 2024
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical Inc announced that positive data from the CANOPY clinical program evaluating VOXZOGO® (vosoritide) in children with achondroplasia and other genetic skeletal conditions will be presented at the 16th International Skeletal Dysplasia Society meeting (ISDS) in Madrid, Sept. 18-21, 2024. .
AI Summary
BioMarin Pharmaceutical Inc. announced that positive data from its CANOPY clinical program with VOXZOGO® (vosoritide) in children with achondroplasia and other genetic skeletal conditions will be showcased at the 16th International Skeletal Dysplasia Society meeting in Madrid, from September 18 to 21, 2024. The program has produced promising results indicating that VOXZOGO not only helps improve height but also enhances health-related quality of life for children with achondroplasia. Researchers have observed significant increases in bone length while maintaining bone strength, suggesting a key role for the drug in supporting proper skeletal development. Additionally, early studies in children with other genetic skeletal conditions, such as hypochondroplasia and Noonan syndrome, show encouraging improvements in growth parameters. This presentation highlights VOXZOGO’s potential as a standard treatment approach and its role in addressing unmet medical needs for families impacted by these disorders.
Read Announcement- Drug:
- vosoritide
- Announced Date:
- June 17, 2024
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical Inc announced the presentation of new data from an investigator-led analysis of the Phase 2 111-205 study, which demonstrate that children with achondroplasia treated with VOXZOGO® experienced increases in bone length while maintaining bone strength..
AI Summary
BioMarin Pharmaceutical Inc. announced new data from an investigator-led analysis of the Phase 2 111-205 study. The results show that children with achondroplasia who were treated with VOXZOGO® experienced significant increases in bone length while maintaining bone strength over approximately five years of therapy. This analysis suggests that as the bones lengthen, they remain robust, which is crucial for the overall health and daily functioning of these children. The findings, to be presented at the 11th International Conference on Children’s Bone Health in Salzburg, Austria, underline the potential of VOXZOGO to address growth challenges in children with achondroplasia. Overall, the results add to the growing evidence supporting the safety and efficacy of VOXZOGO in improving bone growth and quality of life in young patients with growth-related conditions.
Read Announcement
PALYNZIQ FDA Regulatory Timeline and Events
PALYNZIQ is a drug developed by BioMarin Pharmaceutical for the following indication: In Adolescents with Phenylketonuria.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- PALYNZIQ
- Announced Date:
- October 29, 2025
- Target Action Date:
- February 28, 2026
- Indication:
- In Adolescents with Phenylketonuria
Announcement
BioMarin Pharmaceutical Inc announced that The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2026.
AI Summary
BioMarin announced that the FDA accepted its supplemental Biologics License Application for PALYNZIQ to treat adolescents with PKU for Priority Review. The decision is based on positive Phase 3 results from the PEGASUS study, which showed significant blood phenylalanine lowering compared to diet alone. The FDA has set a PDUFA target action date of Feb. 28, 2026.
In the PEGASUS trial, adolescents aged 12–17 saw reductions in blood Phe levels while eating more dietary protein from whole foods. BioMarin aims to offer treatment options that help this group achieve recommended Phe levels and greater dietary freedom.
BioMarin plans to work closely with the FDA and share data with the European Medicines Agency to expand PALYNZIQ use internationally, with the goal of making this enzyme therapy available to adolescents as soon as possible.
Read Announcement- Drug:
- PALYNZIQ
- Announced Date:
- October 29, 2025
- Indication:
- In Adolescents with Phenylketonuria
Announcement
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).
AI Summary
BioMarin announced that the U.S. Food and Drug Administration has accepted for Priority Review the company’s PALYNZIQ® supplemental application to expand treatment to adolescents aged 12–17 with phenylketonuria. The FDA set a Prescription Drug User Fee Act target action date of Feb. 28, 2026.
The application is based on Phase 3 PEGASUS study results showing that PALYNZIQ lowered blood Phe significantly compared with diet alone in 55 teens. The trial also found that treated adolescents could eat more protein from whole foods while reaching normal Phe levels. Greg Friberg, M.D., said adolescents need better options to reduce Phe and gain dietary freedom.
BioMarin plans to work closely with the FDA and share data with the European Medicines Agency to expand adolescent use in the EU. PALYNZIQ is the first enzyme therapy approved for adults with PKU and could soon benefit younger patients.
Read Announcement- Drug:
- PALYNZIQ
- Announced Date:
- September 6, 2025
- Indication:
- In Adolescents with Phenylketonuria
Announcement
BioMarin Pharmaceutical Inc. announced new data characterizing the efficacy and safety of PALYNZIQ® (pegvaliase-pqpz) for the treatment of adolescents with phenylketonuria (PKU), which were presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, Sept. 2-6, 2025.
AI Summary
BioMarin Pharmaceutical Inc. this week revealed new Phase 3 PEGASUS study data for PALYNZIQ (pegvaliase-pqpz) in adolescents aged 12–17 with phenylketonuria (PKU). Presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan (Sept. 2–6, 2025), results showed a 49.7% mean decline in blood phenylalanine (Phe) levels after 72 weeks versus a 0.3% decrease with diet alone. Nearly half (45.2%) of treated teens achieved at least a 50% Phe reduction, with many reaching guideline targets (≤600, ≤360, and ≤120 µmol/L).
Safety findings aligned with adult data, with most adverse events manageable and 5.6% experiencing serious events (anaphylaxis) leading to withdrawal. The ongoing extension phase will track long-term outcomes. BioMarin remains on schedule to submit these results to global regulators in the second half of 2025, aiming to expand PALYNZIQ’s approved use to adolescents with PKU.
Read Announcement- Drug:
- PALYNZIQ
- Announced Date:
- April 2, 2025
- Indication:
- In Adolescents with Phenylketonuria
Announcement
BioMarin Pharmaceutical Inc 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.
AI Summary
BioMarin Pharmaceutical Inc. announced that its Phase 3 PEGASUS trial of PALYNZIQ® (pegvaliase‐pqpz) in adolescents with phenylketonuria (PKU) met its primary efficacy endpoint. The study showed that PALYNZIQ significantly lowered blood phenylalanine (Phe) levels in patients aged 12-17 compared to a diet alone, marking an important development in the treatment of PKU in younger patients. This finding is particularly significant because high Phe levels can lead to severe neurological issues and other complications if not managed properly.
PALYNZIQ, which is already approved for adults with PKU, now shows potential to benefit adolescents transitioning to adult care. BioMarin plans to present the detailed study results at an upcoming medical conference and submit the data to global health authorities later this year to support a proposed label expansion.
Read Announcement
Valoctocogene Roxaparvovec FDA Regulatory Events
Valoctocogene Roxaparvovec is a drug developed by BioMarin Pharmaceutical for the following indication: severe hemophilia A.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Valoctocogene Roxaparvovec
- Announced Date:
- June 24, 2025
- Indication:
- severe hemophilia A
Announcement
BioMarin Pharmaceutical Inc. announced new data underscoring the long-term efficacy and safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) were presented at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington, D.C., June 21-25, 2025.
AI Summary
BioMarin Pharmaceutical Inc. recently presented new data at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington, D.C., highlighting the long-term efficacy and safety of its gene therapy, ROCTAVIAN® (valoctocogene roxaparvovec‐rvox). The findings come from the Phase 3 GENEr8-1 trial, which demonstrated that five years after treatment, patients maintained sustained factor VIII expression and effective bleed control. Notably, 81.3% of participants remained off prophylaxis, and no new safety signals were observed during the study period. These results confirm ROCTAVIAN's potential to reduce the burden of chronic hemophilia A management by providing durable bleeding control and stable factor VIII activity, offering significant improvements in the quality of life for individuals with severe hemophilia A.
Read Announcement- Drug:
- Valoctocogene Roxaparvovec
- Announced Date:
- June 7, 2024
- Indication:
- severe hemophilia A
Announcement
BioMarin Pharmaceutical Inc announced that new data supporting the long-term safety and efficacy of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, June 22-26, 2024..
AI Summary
BioMarin Pharmaceutical Inc. announced that new data on ROCTAVIAN® (valoctocogene roxaparvovec‐rvox) will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, from June 22-26, 2024. The data, drawn from the longest and largest Phase 3 study, show that the gene therapy maintains long-term safety and efficacy for adults with severe hemophilia A. Specifically, patients who received ROCTAVIAN demonstrated sustained bleed control and stable endogenous factor VIII levels up to four years after infusion. These results suggest that many patients remain off regular prophylaxis and could experience a reduced treatment burden, potentially improving their quality of life. BioMarin’s presentation at ISTH will provide detailed insights into the lasting benefits of ROCTAVIAN, highlighting its promise as a long-term solution for managing hemophilia A.
Read Announcement
BRINEURA® FDA Regulatory Events
BRINEURA® is a drug developed by BioMarin Pharmaceutical for the following indication: For Children Under 3 Years with CLN2 Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BRINEURA®
- Announced Date:
- July 24, 2024
- Indication:
- For Children Under 3 Years with CLN2 Disease
Announcement
BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics License Application (sBLA) for BRINEURA® (cerliponase alfa) to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
AI Summary
BioMarin Pharmaceutical Inc. announced that the FDA has approved the supplemental Biologics License Application for BRINEURA® (cerliponase alfa) to slow the loss of ambulation in children with CLN2 disease, also known as TPP1 deficiency or Batten disease. This approval expands its use to children of all ages, including those who have not yet started showing symptoms. The expanded use is based on clinical trial data showing that early treatment can maintain normal motor function and delay the progression of neurodegenerative symptoms. This decision underscores the importance of starting treatment as early as possible, which could significantly impact the disease’s long-term course. With this approval, families affected by CLN2 disease now have hope for earlier intervention to improve quality of life and slow the development of severe, debilitating symptoms.
Read Announcement
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