BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse.
BioMarin HQ/courtesy of BioMarin
BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse. The U.S. Food and Drug Administration approved Voxzogo (vosoritide) to improve growth in children five years of age and older with achondroplasia, a rare genetic disorder that causes the most common form of dwarfism.
The drug, a once-daily injection, has been approved specifically for children who have open epiphyses, or growth plates, which gives them the potential for growth. For those children with achondroplasia, a mutation prevents the growth of that bone. Voxzogo, a C-type natriuretic peptide (CNP) analog, corrects that by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene. With the FDA nod, Voxzogo becomes the first drug approved in the U.S to treat the condition. Prior to the approval, there were some drastic surgical options available to patients, but no therapeutics.
Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. The average height of an adult with achondroplasia is approximately four feet.
Voxzogo was greenlit under accelerated approval based on clinical data demonstrating an improvement in annualized growth velocity (AGV). Under the framework of accelerated approval, California-based BioMarin will be required to continue to provide follow-up data to the FDA. The company said it intends to use ongoing open-label extension studies compared to available natural history to fulfill the requirement.
The European Commission approved Voxzogo in August for the same indication. That marked the first drug specifically approved in Europe for the same indication.
Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin, called Voxzogo a medical first. Bienaimé said the clinical success of the drug is rooted in the company’s focus on molecular genetics and its goal of targeting underlying causes of a condition such as achondroplasia.
“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,” Bienaimé said in a statement. “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.”
FDA approval was based on a Phase III study of 121 children between the ages of five and almost 15 who have achondroplasia. Data showed that after one year of treatment, the children who received the BioMarin treatment saw some improvements in AGV, an average of 4.26 centimeters. The results were statistically significant when compared with placebo.
Theresa Kehoe, director of the division of general endocrinology in the FDA’s Center for Drug Evaluation and Research, said the approval of Voxzogo will fulfill an unmet need for more than 10,000 children in the United States.
“With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature,” Kehoe said in a statement.
Not only did BioMarin score a first with the approval of Voxzogo, the company also snagged a Rare Pediatric Disease Priority Review Voucher. That provides the company with a means to secure priority review for a subsequent drug application that would not qualify for a rapid review. If BioMarin does not use the voucher, it could be sold to another company or used to sweeten the pot of a future deal.
For BioMarin, the approval came right after the company saw a setback in phenylketonuria when the FDA placed a clinical hold on its BMN 307 Phearless Phase I/II study. The company is working with the agency to address this clinical program.
BioMarin is also filing a lawsuit against a former employee over concerns of theft of intellectual property related to its manufacturing capabilities. The company brought a lawsuit against former Senior Engineer Gerardo Caraballo, who took a job at rival gene therapy company Sangamo Therapeutics earlier this month. BioMarin accused Caraballo of stealing more than 1,800 files before departing the company.
In the complaint, BioMarin is asking the courts to prevent Caraballo from “possessing, sharing, or using its confidential information,” Reuters reported. Additionally, BioMarin is seeking the return of the files that are related to its manufacturing process, as well as any money that Caraballo may have made from inappropriate use of the trade secrets. BioMarin said the company secrets will give Sangamo a “significant unfair advantage” for its own gene therapy manufacturing processes. The company also said that Caraballo’s taking of the files posed a “grave threat” to its business, according to the report.
In a brief statement to The San Francisco Business Times, Sangamo said it would investigate the claims and noted that it does not “use or disclose the trade secrets of third parties.”
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