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
BRINEURA®, PALYNZIQ, Valoctocogene, and vosoritide. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
BRINEURA® - FDA Regulatory Timeline and 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.
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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:
- 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.
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Valoctocogene Roxaparvovec - FDA Regulatory Timeline and 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
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 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- Drug:
- vosoritide
- Announced Date:
- May 4, 2024
- Indication:
- Children with achondroplasia
Announcement
BioMarin Pharmaceutical Inc. announced that positive new data supporting the safety and efficacy of VOXZOGO® (vosoritide) in children with achondroplasia, as well as positive data for investigational uses in growth-related conditions, including idiopathic short stature (ISS) and Noonan syndrome, were presented at the 2024 Pediatric Endocrine Society (PES) Annual Meeting in Chicago, May 2-5, 2024.
AI Summary
BioMarin Pharmaceutical Inc. announced promising new data for its medicine VOXZOGO® (vosoritide) at the 2024 Pediatric Endocrine Society (PES) Annual Meeting in Chicago. The data supports the safety and efficacy of VOXZOGO® in children with achondroplasia, showing notable improvements in annualized growth velocity in patients who began treatment during adolescence. In addition, investigational studies in children with other growth-related conditions, such as idiopathic short stature (ISS) and Noonan syndrome, also showed positive results. The findings from these Phase 2 and Phase 3 studies suggest that VOXZOGO® can enhance growth outcomes, providing increased height gains compared to baseline measurements. These encouraging results underline the drug’s potential to improve health-related quality of life and support further clinical trials in a broader group of children with various skeletal dysplasias and growth-related disorders.
Read Announcement
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