Factors Affecting the Price of VOXZOGO

The price of VOXZOGO (vosoritide) is influenced by several key factors stemming from its nature and development.

Factors Affecting the Price of VOXZOGO

Targeting a Rare Disease: It treats achondroplasia, a rare genetic disorder, limiting the potential patient population and increasing per-patient development costs.

Complex Biologic: As a recombinant peptide analog manufactured using E. coli DNA technology, production is inherently complex and costly.

Intensive Development: The approval was based on extensive, lengthy (52-week+) randomized controlled trials and ongoing open-label extensions, representing significant R&D investment.

Specialized Use: It requires daily subcutaneous injections with precise weight-based dosing and specific reconstitution procedures using co-packaged supplies (vials, prefilled diluent syringes, needles).

Accelerated Approval Pathway: While approved based on improved growth velocity, continued approval depends on confirmatory trials, adding ongoing costs.

Monitoring Requirements: Long-term treatment necessitates regular (every 3-6 months) growth and renal function monitoring.