Pharmaceutical Grade Vosoritide Acetate API | Achondroplasia Targeted Peptide | CAS 1480724-61-5

Vosoritide Acetate More Info

Vosoritide Acetate is the acetate salt form of a recombinant C-type natriuretic peptide analog, a modified version of the human CNP37 peptide, assigned the CAS Registry Number 1480724-61-5. Its molecular structure consists of 39 amino acids, including the native 37 amino acids of CNP with two additional C-terminal amino acids designed to enhance its stability and prolong its plasma half-life. The acetate salt formation is a pharmaceutical step to optimize its solubility and stability for formulation. This peptide represents a targeted biologic therapy for a specific genetic skeletal dysplasia.

The primary mechanism of action for vosoritide involves correcting the core signaling imbalance in the growth plates of children with achondroplasia. Achondroplasia is caused by a gain-of-function mutation in the FGFR3 gene, which constitutively inhibits chondrocyte proliferation and differentiation, stunting bone growth. Vosoritide acts as a potent agonist of the natriuretic peptide receptor B on chondrocytes. This activation stimulates the production of intracellular cGMP, which counteracts the mitogen-activated protein kinase pathway signaling driven by the mutated FGFR3, thereby restoring a more normal rate of endochondral bone growth.

The clinical application of vosoritide is specifically for the treatment of achondroplasia in children aged 5 years and older whose growth plates are still open. It is indicated to increase linear growth by stimulating growth plate activity. It is not a cure for the condition but is the first approved pharmacologic therapy to directly address its underlying pathophysiology. Its development marked a significant milestone in the treatment of this genetic disorder, offering a targeted approach to modify its natural history.

This therapy is administered as a daily subcutaneous. The dose is calculated based on the child's body weight. Its pharmacokinetic profile shows a short half-life, necessitating daily administration to maintain therapeutic effect. The formulation is supplied as a powder that must be reconstituted with sterile water immediately before use. Adherence to the daily regimen is critical for sustained efficacy in promoting growth velocity.

The safety profile of vosoritide is characterized by expected pharmacologic effects and -related reactions. The most common adverse events are mild to moderate and include site reactions, transient hypotension (often asymptomatic), and a temporary drop in blood pressure shortly after, which typically resolves spontaneously. Other reported effects can include vomiting and arthralgia. Long-term safety and the sustained impact on final adult height continue to be monitored in ongoing clinical studies. Its use represents a paradigm shift from symptomatic management to targeted disease-modifying therapy for achondroplasia.

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