BioMarin Pharma gets USFDA accelerated approval for Voxzogo for Injection

Voxzogo is the first FDA approved treatment for children with achondroplasia. In patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

"Voxzogo is a medical first that is rooted in BioMarin's focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We extend our gratitude to the community, clinical investigators and the children and their families, who participated and continue to participate in our comprehensive clinical research program as we continue to investigate the full potential of vosoritide."

"Achondroplasia is a lifelong genetic condition resulting from the disordered skeletal architecture caused by impaired endochondral bone growth throughout childhood," said Lynda Polgreen, M.D., an investigator in clinical trials for Voxzogo and an Investigator at The Lundquist Institute at Harbor-UCLA Associate Professor at David Geffen School of Medicine – UCLA. "This approval is an important milestone representing the first time that physicians will be able to offer a therapy targeted at the root cause of the condition for families of children with achondroplasia aged five and older."

"We applaud the FDA for recognizing the urgent unmet medical need for this progressive condition. As a parent of a child with achondroplasia, I see the availability of treatments that impact bone growth as an important step forward," said Amer Haider Co-Founder of Growing Stronger, an organization with a mission to improve the quality of medical care for little people through supporting research. The organization raises nonprofit donations that are granted to researchers focused on dwarfism.

Mr. Haider added, "BioMarin continues to support the achondroplasia community and has a long track record of advancing the standard of care in rare genetic conditions."

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The rare pediatric disease PRV program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

Voxzogo is expected to be available in the United States by mid- to late-December, and BioMarin will begin the promotion of Voxzogo immediately.

The approval was based on the outcomes of a global randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of Voxzogo and the open-label extension of this Phase 3 study. The study enrolled 121 children aged 5 to 14.9 with achondroplasia. Baseline mean AGV in the placebo and Voxzogo groups was 4.06 cm/year and 4.26 cm/year, respectively. At week 52, the change from baseline in AGV was -0.17 cm/year for the placebo treated patients and 1.40 cm/year for the Voxzogo treated patients, resulting in a statistically significant improvement in AGV of 1.57 cm/year in favor of Voxzogo. After the 52 week double blind, placebo–controlled, phase 3 study, 58 subjects initially randomized to Voxzogo enrolled into an open–label extension. Among the subjects who had two years of follow–up since randomization, the improvement in AGV was maintained.

In August 2021, the European Commission (EC) approved Voxzogo. Marketing authorization reviews are in process in Japan, Brazil, and Australia with potential approvals in these countries in 2022.

Safety and efficacy of Voxzogo in patients with achondroplasia were assessed in one 52–week, multi–center, randomized, double–blind, placebo–controlled, Phase 3 study. Transient decreases in blood pressure have been observed with Voxzogo. In the clinical study, 8 (13%) of 60 patients treated with Voxzogo had a total of 11 events of transient decreases in blood pressure compared to 3 (5%) of 61 patients on placebo, over a 52-week treatment period. Patients with significant cardiac or vascular disease or on anti-hypertensive medicine were excluded from the trial.