FDA Approves BioMarin’s Voxzogo to treat Dwarfism in Children

BioMarin Pharmaceutical’s Voxzogo (vosoritide) injection received approval by the FDA (Food and Drug Administration) on November 19, 2021, to manage Dwarfism in children. It can now be used to improve growth in children aged above 5 years who are suffering from open epiphyses (growth plates) and achondroplasia (short-limbed dwarfism). This means that such patients can still manage the condition and grow. Earlier in August 2021, Voxzogo (vosoritide) injection received approval for use in Europe. 

The approval of VOXZOGO injection was based on the results of a double-blind, global randomized, placebo-controlled Phase 3 research study that was conducted to determine and evaluate Voxzogo's efficacy and its safety. A total of 121 children aged 5 to 14.9 years suffering from achondroplasia were initially enrolled in the study. The placebo had a baseline AGV of 4.06 cm per year and the Voxzogo groups had a baseline AGV of 4.26 cm per year. 

At 52nd week, the change in AGV from baseline for placebo-treated patients was about -0.17 cm per year when compared to 1.40 cm per year for Voxzogo-treated patients which leads to a statistically relevant improvement in AGV of 1.57 cm per year in favor of Voxzogo. 58 patients were initially randomized to Voxzogo joined in the open-label extension after the 52-week double-blind, placebo-controlled phase 3 investigation. 

The most common side effects include injection site reactions like rash, hives, skin redness, swelling, bruising, itching, pain in the arms), diarrhea, dizziness, seasonal allergy, vomiting, ear pain, influenza, fatigue, joint pain, gastroenteritis, decreased blood pressure, and dry skin.

About Achondroplasia

Achondroplasia is a very common form of short-limbed dwarfism that occurs in children. This disease is mostly caused by a genetic mutation. It’s evident because most cases of Achondroplasia are diagnosed in children who have parents of average height. This is a bone growth disorder. Mainly, it prevents the process in which long bones of the legs and arms change to bones. Besides dwarfism, the condition is characterized by a limited range of motion and very small fingers with larger (than usual) head sizes. In 80% of Achondroplasia patients, the disease is a result of a spontaneous mutation. The remaining 20% might get it from certain inherited genes. 

About VOXZOGO (vosoritide) injection

Voxzogo (vosoritide) injection is an analog of C-type natriuretic peptide which is used in the treatment of patients diagnosed with Achondroplasia. Specifically, it promotes bone growth in pediatric patients (aged above 5 years) who are suffering from short-limbed dwarfism caused by some gene mutations. This medicine is a potential regimen for the promotion of linear growth in children.


FDA Approves | BioMarin’s Voxzogo drug | Dwarfism in Children