Drugs / Vosoritide
Vosoritide
also known as
BMN 111 · Vosoritide Injection · VOXZOGO · Modified C-Natriuretic Peptide · ProCNP38 · Vosoritida
Regulatory milestones approvals, filings & regulatory actions · 8 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed achondroplasia 4 milestones | |||||
| Filed | EU (EMA) | VOXZOGO | achondroplasia | 2023-01-03 | “the European Medicines Agency (EMA) validated its Type II Variation application to extend the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia” biomarin.com ↗ |
| Filed | US (FDA) | — | achondroplasia | 2020-11-02 | “FDA has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia” biomarin.com ↗ “Vosoritide for children with achondroplasia: Marketing applications for vosoritide were recently validated and accepted, by EMA and FDA, respectively.” biomarin.com ↗ |
| Filed | US (FDA) | VOXZOGO | achondroplasia | 2020-08-20 | “In April, the company submitted its U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO ® for achondroplasia.” biomarin.com ↗ “today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vosoritide” biomarin.com ↗ |
| Filed | EU (EMA) | — | achondroplasia | 2020-07-23 | “Vosoritide for children with achondroplasia: Marketing applications for vosoritide were recently validated and accepted, by EMA and FDA, respectively.” biomarin.com ↗ “the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vosoritide” biomarin.com ↗ |
| Filed hypochondroplasia 1 milestone | |||||
| Filed | US (FDA) | VOXZOGO | hypochondroplasia | — | “BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of...” biomarin.com ↗ “BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of...” biomarin.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | VOXZOGO | — | 2023-10-20 | fda.gov ↗ |
| Approved | US (FDA) | VOXZOGO | — | 2021-11-19 | fda.gov ↗ |
| Approved | EU (EMA) | Voxzogo | — | 2021-08-26 | europa.eu ↗ |
Trials 15
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05845749 | Sep 2023 → Dec 2028 expected | mucopolysaccharidosis type 4A, mucopolysaccharidosis type 6 | University of California, San Francisco | Active not recruiting | No outcome recorded |
| Phase 210 trials · 1 met primary · 1 missed | ||||||
| Phase 2 | NCT07126262 | Jul 2025 → Jun 2028 expected | hypochondroplasia | BioMarin Pharmaceutical | Recruiting | No outcome recorded |
| Phase 2 | NCT06668805 | Nov 2024 → Mar 2027 expected | Noonan syndrome | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
| Phase 2 | NCT06382155 | Oct 2024 → Jun 2026 overdue | isolated short stature | BioMarin Pharmaceutical | Recruiting | No outcome recorded |
| Phase 2 | NCT05849389 | Apr 2024 → Sep 2026 overdue | Turner syndrome, isolated short stature | Roopa Kanakatti Shankar, MBBS, MS | Recruiting | No outcome recorded |
| Phase 2 | NCT04554940 | Oct 2020 → Dec 2027 expected | achondroplasia | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
| Phase 2 | NCT04219007 | Aug 2020 → Aug 2025 overdue | — | Andrew Dauber | Active not recruiting | No outcome recorded |
| Phase 2 | NCT03989947 | Jun 2019 → May 2038 expected | achondroplasia | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
| Phase 2 | NCT03583697 | Jun 2018 → Jan 2022 | achondroplasia | BioMarin Pharmaceutical | Completed | Missed primary |
| Phase 2 | NCT02724228 | Jan 2016 → Dec 2027 expected | achondroplasia | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02055157 | Jan 2014 → Oct 2017 | achondroplasia | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 34 trials · 2 met primary | ||||||
| Phase 3 | NCT07073014 | Jun 2025 → Oct 2044 expected | hypochondroplasia | BioMarin Pharmaceutical | Enrolling by invitation | No outcome recorded |
| Phase 3 | NCT06455059 | Jun 2024 → Aug 2026 overdue | hypochondroplasia | BioMarin Pharmaceutical | Active not recruiting | Met primary |
| Phase 3 | NCT03424018 | Dec 2017 → Jun 2031 expected | achondroplasia | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
| Phase 3 | NCT03197766 | Dec 2016 → Oct 2019 | achondroplasia | BioMarin Pharmaceutical | Completed | Met primary |
| Comparator or background therapy2 trials | ||||||
| Phase 1 | NCT01590446 Comparator | Feb → Jun 2012 | achondroplasia | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2/3 | NCT07441876 Comparator | Apr 2026 → Jun 2029 expected | achondroplasia | BioMarin Pharmaceutical | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 44
| Date | Issuer | Release |
|---|---|---|
| 2026-10-09 | BioMarin Pharmaceutical | Results BioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting biomarin.com ↗
The data include encouraging new imaging results from a post-hoc analysis of a randomized, double-blind, placebo-controlled trial evaluating the effect of VOXZOGO on the growth of the foramen magnum and craniocervical junction in children with achondroplasia, including infants. |
| 2026-09-09 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Results of Phase 3 VOXZOGO® (vosoritide) Study in Hypochondroplasia Published in NEJM Evidence biomarin.com ↗
The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p < 0.0001). |
| 2026-07-13 | BioMarin Pharmaceutical | Regulatory U.S. Food and Drug Administration Accepts BioMarin’s Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO ® (vosoritide) for full approval in children with achondroplasia. |
| 2026-06-16 | BioMarin Pharmaceutical | Results BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026 biomarin.com ↗
Mean height SDS improved by 0.72 SD over three years of treatment, while mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p < 0.001) and remained above baseline through years two and three. |
| 2026-05-20 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia biomarin.com ↗
Children who received VOXZOGO also showed a statistically significant increase in standing height (p < 0.0001) and height Z-score (p < 0.0001) versus placebo after one year of treatment. |
| 2026-05-02 | BioMarin Pharmaceutical | Results BioMarin Presents New Data on the Effect of Long-Term Treatment with VOXZOGO® (vosoritide) on Arm Span, Bone Health and Growth in Children With Achondroplasia at the Pediatric Endocrine Society’s 2026 Annual Meeting biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today 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. |
| 2026-03-12 | BioMarin Pharmaceutical | Results BioMarin Presents New Data on the Positive Impact of Early Treatment With VOXZOGO® (vosoritide) on Proportionality and Arm Span in Children with Achondroplasia at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO ® (vosoritide) in children with achondroplasia in ongoing clinical trials and real-world studies. |
| 2025-09-08 | BioMarin Pharmaceutical | Results BioMarin Shares New Data Reinforcing Its Leadership in Bone Health at the American Society for Bone and Mineral Research Annual Meeting biomarin.com ↗
Researchers found that children (n=40) who received VOXZOGO for 52 weeks experienced measurable improvement in interpedicular distance (IPD) and greater spinal canal width in all five vertebrae in the lower back (L1 through L5) compared to children who received placebo (n=27). |
| 2025-05-12 | BioMarin Pharmaceutical | Results BioMarin Presents New Data for VOXZOGO® (vosoritide) in Children with Achondroplasia and Other Skeletal Conditions at Two International Endocrinology Meetings biomarin.com ↗
New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function |
| 2025-03-20 | BioMarin Pharmaceutical | Results 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 College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting biomarin.com ↗
New data from a study in Japan 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. |
| 2024-11-16 | BioMarin Pharmaceutical | Results BioMarin Presents Real-World Evidence Further Supporting Safety and Efficacy of VOXZOGO® (vosoritide) in Children with Achondroplasia at the European Society for Paediatric Endocrinology (ESPE) Meeting 2024 biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive and consistent results from multiple real-world evidence studies of VOXZOGO ® (vosoritide) in children with achondroplasia. |
| 2024-09-18 | BioMarin Pharmaceutical | Results BioMarin to Present Data Underscoring Sustained Positive Impact of VOXZOGO® (vosoritide) on Health-Related Quality of Life, Growth and Maintenance of Bone Strength in Children with Achondroplasia at 2024 International Skeletal Dysplasia Society Meeting biomarin.com ↗
These results include data showing that children with achondroplasia treated with VOXZOGO experienced meaningful improvements beyond height, such as in health-related quality of life (HRQoL), and increased bone length while maintaining bone strength. |
| 2024-06-17 | BioMarin Pharmaceutical | Results BioMarin to Present Data Highlighting Significant Impact of VOXZOGO® (vosoritide) in Bone Health and Health-Related Quality of Life in Achondroplasia at 2024 International Conference on Children’s Bone Health biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today 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. |
| 2024-05-04 | BioMarin Pharmaceutical | Results New Data for BioMarin’s VOXZOGO® (vosoritide) for Multiple Growth-Related Conditions in Children Presented at Pediatric Endocrine Society (PES) Annual Meeting biomarin.com ↗
Results demonstrated marked improvement in annualized growth velocity (AGV) and height standard deviation (SD) across all the conditions studied. |
| 2024-03-12 | BioMarin Pharmaceutical | Results New Data to be Presented for BioMarin’s VOXZOGO® (vosoritide) in Children with Hypochondroplasia and Achondroplasia at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting biomarin.com ↗
The annualized growth velocity (AGV) increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period (mean difference: 1.81 cm/year, p<0.0001). |
| 2023-10-20 | BioMarin Pharmaceutical | Regulatory U.S. Food and Drug Administration Approves BioMarin’s VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia biomarin.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for VOXZOGO ® (vosoritide) to increase linear growth in pediatric patients with achondroplasia with open epiphyses (growth plates). |
| 2023-09-21 | BioMarin Pharmaceutical | Results BioMarin to Present Data Showing Long-Term Benefit of VOXZOGO® (vosoritide) on Growth in Children with Achondroplasia at 2023 European Society for Paediatric Endocrinology (ESPE) Meeting biomarin.com ↗
In an open-label, long-term Phase 2 extension study, VOXZOGO demonstrated consistent and durable growth benefits in young children with achondroplasia when initiated before the age of 5. |
| 2023-09-15 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Positive CHMP Opinion in Europe to Expand Use of VOXZOGO® (vosoritide) to Treat Children Aged 4 Months and Older with Achondroplasia biomarin.com ↗ |
| 2023-03-14 | BioMarin Pharmaceutical | Results BioMarin to Share Updated Data at 2023 American College of Medical Genetics and Genomics (ACMG) Meeting Demonstrating Commitment to Understanding Long-Term Benefit of VOXZOGO® in Children with Achondroplasia biomarin.com ↗
Mean age- and sex-specific Annualized Growth Velocities (AGVs) in children treated with VOXZOGO were greater than corresponding mean AGVs in untreated children of the same age and sex and at all ages. |
| 2023-03-07 | BioMarin Pharmaceutical | Regulatory FDA Accepts BioMarin’s Supplemental New Drug Application to Expand Use of VOXZOGO® (vosoritide) for Injection to Treat Children with Achondroplasia Under the Age of 5 biomarin.com ↗
The FDA has set a PDUFA target action date of October 21, 2023 for the sNDA. |
| 2023-01-03 | BioMarin Pharmaceutical | Regulatory European Medicines Agency Validates Application for Extension of Indication for VOXZOGO® (vosoritide) for injection to Treat Children with Achondroplasia Under the Age of 2 biomarin.com ↗
announced today that the European Medicines Agency (EMA) validated its Type II Variation application to extend the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia under the age of 2. |
| 2022-06-21 | BioMarin Pharmaceutical | Regulatory BioMarin Announces the Ministry of Health, Labor and Welfare (MHLW) in Japan Granted Approval for VOXZOGO® (vosoritide) for Injection for the Treatment of Children with Achondroplasia, Whose Growth Plates are Not Closed biomarin.com ↗
BioMarin Announces Approval of VOXZOGO in Japan for Children of all ages |
| 2022-06-13 | BioMarin Pharmaceutical | Results BioMarin Announces Favorable Results from Global Phase 2 Study of VOXZOGO™ (vosoritide) for Injection in Infants and Young Children with Achondroplasia at The Endocrine Society Annual Meeting, ENDO 2022 (June 11-14), in Atlanta biomarin.com ↗
Voxzogo (n=43), compared to placebo (n=32), increased height Z-score by 0.30 SD (95% CI 0.07, 0.54) and annualized growth velocity (AGV) by 0.92cm/year (95% CI 0.24, 1.59). |
| 2021-11-19 | BioMarin Pharmaceutical | Regulatory BioMarin Receives FDA Approval for VOXZOGO™ (vosoritide) for Injection, Indicated to Increase Linear Growth in Children with Achondroplasia Aged 5 and Up with Open Growth Plates biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to VOXZOGO™ (vosoritide) for Injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates). |
| 2021-08-27 | BioMarin Pharmaceutical | Regulatory European Commission Approves BioMarin’s VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close biomarin.com ↗
Voxzogo is the first medicine to be approved to treat children with achondroplasia in Europe. |
| 2021-06-25 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for vosoritide, a once daily injection analog of C-type Natriuretic Peptide (CNP) to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. |
| 2021-03-20 | BioMarin Pharmaceutical | Results BioMarin Announces Oral Presentation at ENDO2021, the Endocrine Society’s Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Children with Achondroplasia Treated with Vosoritide biomarin.com ↗
The data from the open-label extension presented at ENDO21 showed that children maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment with vosoritide. |
| 2020-12-21 | BioMarin Pharmaceutical | Results BioMarin Announces Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study biomarin.com ↗
The cumulative height gain over the 2-year treatment period was 3.52 cm compared to untreated children, which is the sum of the first year (1.73) and the second year (1.79). |
| 2020-11-02 | BioMarin Pharmaceutical | Regulatory Food and Drug Administration Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia biomarin.com ↗ |
| 2020-09-08 | BioMarin Pharmaceutical | Results BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondroplasia biomarin.com ↗
This study provides the first robust evidence for a precision therapy for achondroplasia that has the potential to fundamentally change the clinical management, growth trajectory, and treatment recommendations for affected children. |
| 2020-08-20 | BioMarin Pharmaceutical | Regulatory BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. |
| 2020-08-13 | BioMarin Pharmaceutical | Regulatory European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Vosoritide to Treat Children with Achondroplasia biomarin.com ↗ |
| 2020-07-23 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Marketing Authorization Application to European Medicines Agency for Vosoritide to Treat Children with Achondroplasia biomarin.com ↗ |
| 2019-12-16 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today reported positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. |
| 2019-11-14 | BioMarin Pharmaceutical | Results Poised for Significant Growth and Profitability, BioMarin Shares Company Highlights During R&D Day on November 14th in New York biomarin.com ↗
2.2 cm of this additional increase occurred in the last 12 months further informing our understanding of vosoritide's ongoing treatment impact. |
| 2019-11-14 | BioMarin Pharmaceutical | Results BioMarin Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study biomarin.com ↗
2.2 cm of this additional increase occurred in the last 12 months further informing our understanding of vosoritide's ongoing treatment impact. |
| 2019-06-18 | BioMarin Pharmaceutical | Results BioMarin Announces New England Journal of Medicine Publishes Vosoritide Phase 2 Study Showing Sustained Annualized Growth Up to 42 Months in Children with Achondroplasia biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced that the New England Journal of Medicine (NEJM) published online today results from a Phase 2 dose-finding and extension study for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia. |
| 2018-11-07 | BioMarin Pharmaceutical | Results BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on November 7th in New York biomarin.com ↗
An ongoing open-label, Phase 2 study of vosoritide for achondroplasia demonstrated sustained increase in cumulative height gained over 42 months of treatment in children in cohort 3 at a dose of 15 µg/kg/day. |
| 2017-10-18 | BioMarin Pharmaceutical | Results BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on October 18th in New York biomarin.com ↗
Vosoritide for achondroplasia demonstrates sustained increase in average growth velocity over 30 months of treatment in 10 children, who completed 30 months of daily dosing at 15 µg/kg/day. |
| 2016-10-19 | BioMarin Pharmaceutical | Results BioMarin Presents Vosoritide Data in Achondroplasia at American Society of Human Genetics (ASHG) 2016 Meeting biomarin.com ↗
Results from 8 children in cohort 4, who completed six months of daily dosing at 30 µg/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.03). |
| 2016-04-20 | BioMarin Pharmaceutical | Results BioMarin Provides Program Update on Vosoritide in Achondroplasia biomarin.com ↗
After 12 months of daily dosing at 15 µg/kg/day, the cohort 3 patients (n=10) experienced a 46% or 1.9 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.02). |
| 2015-10-12 | BioMarin Pharmaceutical | Results BioMarin’s Initial 6-Month Data from Phase 2 Study of Vosoritide (BMN 111) in Children with Achondroplasia Presented at the American Society for Bone and Mineral Research Annual 2015 Meeting biomarin.com ↗
The initial six-month data from the first three cohorts showed a 50 percent or 2.01 cm/year increase in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (p-value= 0.01). |
| 2015-06-17 | BioMarin Pharmaceutical | Results BMN 111 (vosoritide) Improves Growth Velocity in Children With Achondroplasia in Phase 2 Study biomarin.com ↗
The 10 children in Cohort 3 treated with 15 micrograms per kilogram per day had a mean increase of 50% (p-value = 0.01) in their annualized growth velocity compared to their annualized prior 6 month natural history baseline growth velocity. |
| 2013-09-17 | BioMarin Pharmaceutical | Regulatory BioMarin Provides Highlights Ahead of Research and Development Day biomarin.com ↗
BMN 111: US and European Regulatory Authorities agree that Phase 2 Study in Achondroplasia can start without additional data. |
All press releases naming this drug 52 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 18 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Vosoritide | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07441876 ↗ |
| Known as | BMN 111 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03583697 ↗ |
| Known as | Modified C-Natriuretic Peptide | “Modified C-Natriuretic Peptide, ProCNP38” NCT01590446 ↗ |
| Known as | ProCNP38 | “Modified C-Natriuretic Peptide, ProCNP38” NCT01590446 ↗ |
| Known as | Vosoritida | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707276 ↗ |
| Known as | Vosoritide Injection | “Drug: Vosoritide Injection” NCT06668805 ↗1“Drug: Vosoritide Injection” NCT06382155 ↗ |
| Known as | VOXZOGO | “Drug: Vosoritide Injection [Voxzogo]” NCT05845749 ↗1“Vosoritide Injection [Voxzogo]” NCT07441876 ↗ |
| Action | Activate | “a selective NPR-B agonist” NCT04219007 ↗ |
| Modality | Peptide | “Modified recombinant human C-type natriuretic peptide” NCT03989947 ↗ |
| Route | Subcutaneous | “SC injection, Part 1 single dose and Part 2 multiple dose.” NCT01590446 ↗ |
| Target | NPR2 | “Atrial natriuretic peptide receptor B binding agent” CHEMBL3707276 ↗ |