Overview
Achondroplasia is an autosomal dominant genetic disease and the most common cause of dwarfism in humans. It results from a gain-of-function missense mutation in FGFR3 that results in a dramatic suppression of bone growth, both in volume and in length. Treatment for achondroplasia includes both surgical and pharmacological interventions, the latter of which includes C-type natriuretic peptide (CNP) analogs. Endogenous CNP, first described in 1998, is primarily responsible for the stimulation of chondrocytes and long bone growth via activity at the NPR-B receptor, making it an attractive target in the treatment of a condition like achondroplasia. While the remarkably short half-life of endogenous CNP - 2 to 3 minutes due to its rapid degradation by endopeptidases - makes it ineffective as a therapeutic intervention, the development of a peptidase-resistant formulation has allowed for its use as a viable treatment option in achondroplasia. Vosoritide is an analog of CNP with proline-glycine on its N-terminus to convey resistance to neutral endopeptidase. It was approved for use under the brand name Voxzogo (BioMarin Pharmaceutical Inc.) in the EU in August 2021 and the US in November 2021, becoming the first pharmacological intervention approved for the treatment of achondroplasia in both regions.
Indication
Vosoritide is indicated for the promotion of linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
Associated Conditions
No associated conditions information available.
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2024/10/31 | Phase 2 | Recruiting | |||
2024/06/12 | Phase 3 | ENROLLING_BY_INVITATION | |||
2025/06/24 | Phase 2 | Not yet recruiting | |||
2023/05/08 | Phase 2 | Recruiting | Roopa Kanakatti Shankar, MBBS, MS | ||
2023/05/06 | Phase 1 | Active, not recruiting | |||
2020/09/18 | Phase 2 | Active, not recruiting | |||
2020/01/06 | Phase 2 | Active, not recruiting | Andrew Dauber | ||
2018/07/11 | Phase 2 | Completed | |||
2018/02/06 | Phase 3 | Active, not recruiting | |||
2017/06/23 | Phase 3 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| No FDA approvals found for this drug. | |||||
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 8/26/2021 | ||
Authorised | 8/26/2021 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| VOXZOGO vosoritide 0.4 mg powder for injection vial and diluent pre-filled syringe | 376616 | Medicine | A | 7/6/2022 | |
| VOXZOGO vosoritide 1.2 mg powder for injection vial and diluent pre-filled syringe | 376618 | Medicine | A | 7/6/2022 | |
| VOXZOGO vosoritide 0.56 mg powder for injection vial and diluent pre-filled syringe | 376617 | Medicine | A | 7/6/2022 |
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