Voxzogo

(Vosoritide)

Phase 3Peptide

BioMarin Pharmaceutical

Voxzogo is one of 19 small molecule programs targeting Hypochondroplasia. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

HypochondroplasiaActive, not recruiting

Turner SyndromeRecruiting

AchondroplasiaActive, not recruiting

Short StatureActive, not recruiting

Noonan SyndromeRecruiting

HypochondroplasiaPhase 3

Active, not recruiting

Turner SyndromePhase 2

Recruiting

AchondroplasiaPhase 2

Active, not recruiting

Short StaturePhase 2

Active, not recruiting

Noonan SyndromePhase 2

Recruiting

Competition

Competitive Landscape

9 competitors in Achondroplasia

#DrugCompanyPhaseTrials

Voxzogo

BioMarin Pharmaceutical

Phase 3

2 2

BMN 333

BioMarin Pharmaceutical

Phase 3

6 3

Truseltiq

HHelsinn Healthcare SA

Phase 2

1 4

Yuviwel

Ascendis Pharma

Phase 2

3 5

ABSK061

AAbbisko Therapeutics Co, Ltd

Phase 2

1 6

TYRA-300

TTyra Biosciences, Inc

Phase 2

1 7

Genotropin

EMD Serono

Preclinical

1 8

Transcon CNP

AAscendis Pharma Growth Disorders A/S

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2027

Phase 2Active, not recruiting

Achondroplasia

NCT04554940

Q2 2028

Phase 2Recruiting

Hypochondroplasia

NCT07126262

Q3 2029

PreclinicalRecruiting

Achondroplasia

NCT07441876

Q4 2029

Phase 1/2Active, not recruiting

Growth

NCT05845749

Clinical

Trials by Indication[1]

Hypochondroplasia3 trials

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 2·NCT07126262·60 enrolled

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Active, not recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 3·NCT06455059·80 enrolled

A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia

Enrolling by invitation· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 3·NCT07073014·140 enrolled

Achondroplasia2 trials

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Active, not recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 2·NCT04554940·20 enrolled

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Preclinical·NCT07441876·160 enrolled

Short stature2 trials

Vosoritide for Selected Genetic Causes of Short Stature

Active, not recruiting·AAndrew Dauber·Phase 2·NCT04219007·56 enrolled

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 2·NCT06382155·100 enrolled

Turner syndrome1 trial

Vosoritide for Short Stature in Turner Syndrome

Recruiting·RRoopa Kanakatti Shankar, MBBS, MS·Phase 2·NCT05849389·20 enrolled

Growth1 trial

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

Active, not recruiting·UUniversity of California, San Francisco·Phase 1/2·NCT05845749·6 enrolled

1 more indication

Noonan syndrome1 trial

A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 2·NCT06668805·30 enrolled

Regulatory

FDA Approval Timeline[4]

Nov 19, 2021

VOXZOGO

NDA NDA214938

Sources

References

[1]
ClinicalTrials.gov — 10 trial records for Vosoritide. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Vosoritide compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Vosoritide. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA214938). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Voxzogo. U.S. Food & Drug Administration.
[6]
SEC EDGAR — BioMarin Pharmaceutical company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Peptide[2]
Highest Phase: Phase 3[1]
Company: BioMarin Pharmaceutical

Patent Landscape[5]

Patent Cliff

May 20, 2030

Earliest patent expiration — generic entry possible after this date

Patent #Expires

RE48267May 20, 2030

8198242Jun 11, 2030

12514906Aug 1, 2036

9907834Aug 1, 2036

11590204Aug 1, 2036

3 more patents

11911446Aug 1, 2036

10646550Aug 1, 2036

12233106Jul 11, 2042

Voxzogo

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Q2 2028

Q3 2029

Q4 2029

Trials by Indication[1]

Hypochondroplasia3 trials

Achondroplasia2 trials

Short stature2 trials

Turner syndrome1 trial

Growth1 trial

Noonan syndrome1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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