Vosoritide

Summary

Vosoritide is a recombinant CNP analog administered subcutaneously to increase linear growth in children with achondroplasia by modulating the FGFR3 pathway.

Mechanism of Action

Agonist of natriuretic peptide receptor B (NPR-B) leading to increased intracellular cGMP and inhibition of FGFR3 signaling, thereby promoting endochondral bone growth.

Routes of Administration

Goals & Uses

• Increase annualized growth velocity in achondroplasiaSkeletal GrowthHigh
• Reduce risk of skeletal complications (e.g., spinal stenosis, foramen magnum narrowing)Disease ModificationLow
• Improve body proportionality and skeletal morphologySkeletal DevelopmentModerate
• Achondroplasia growth promotionIndicationHigh
• Improve quality of life in achondroplasia patientsPatient Reported OutcomesModerate

Contraindications

• Concomitant use of soluble guanylate cyclase (sGC) stimulators (e.g., riociguat)Drug InteractionHigh
• Hypersensitivity to vosoritide or any excipientAllergyHigh
• Hypersensitivity to vosoritide or any excipientsAllergy/hypersensitivityHigh
• Concomitant use of PDE5 inhibitorsDrug InteractionHigh

Adverse Effects

• Hypersensitivity reactionsImmunologicRare
• Injection site reactionsLocalCommon
• HeadacheNeurologicCommonPain in the head or upper neck
• HypotensionCardiovascularUncommonLow blood pressure
• Dizziness / syncopeNeurological/cardiovascularUncommon
• Nausea and vomitingGastrointestinalUncommon
• Injection site erythemaLocalCommonRedness at the injection site
• Decreased blood pressure / hypotensionCardiovascularCommon

Drug Interactions

• Soluble guanylate cyclase stimulators (e.g., riociguat)High
• Antihypertensive agentsModerate
• PDE5 inhibitors (e.g., sildenafil, tadalafil)High

Population Constraints

• PregnancyReproductive SafetyRelative
• Adults with closed growth platesEfficacy LimitationRelative
• Patients with cardiovascular disease predisposing to hypotensionCardiovascularRelative
• Children under 5 years of ageAgeAbsolute
• Patients with uncontrolled hypertensionCardiovascularRelative
• Children under 2 years (EU) or under 5 years (US)Pediatric Age RestrictionAbsolute

Regulatory Status

• European UnionApprovedApproved: Achondroplasia in children 5–18 yearsEMA approval 2021.
• United StatesApprovedApproved: Achondroplasia in children 5–18 yearsApproved by FDA in 2021 (brand name Voxzogo).
• United KingdomApprovedApproved: Achondroplasia in children 5–18 yearsApproved by MHRA.

Approved by the FDA in November 2021 for patients with achondroplasia aged 5 years and older. Approved by the EMA in August 2021 for patients aged 2 years and above. Approved by the MHRA in the UK. Breakthrough Therapy designation and Orphan Drug designation granted by FDA.

Evidence & Sources

No sources recorded yet.

Frequently Asked Questions