Children with dwarfism caused by achondroplasia have few other treatment options except for surgical treatment of spinal or limb complications. Now there is a drug that can solve some of these problems first.this FDA approved A BioMarin drug therapy that can solve the genetic mutations at the root of genetic diseases.
The regulatory approval of the drug vosoritide is for children 5 years of age and older who have achondroplasia but still have open growth plates.The decision was made three months later The European Commission granted marketing authorization for the drug, which was sold under the name “Voxzogo”,” Suitable for children from 2 years old to closed growth plates.
BioMarin, headquartered in San Rafael, California, stated in an investor introduction that as the first approved drug to treat the root cause of achondroplasia, Voxzogo may become a best-selling product. Chief Commercial Officer Jeff Ajer said that he expects the drug to exceed sales of the company’s enzyme replacement therapy Vimizan, which had sales of $544.4 million last year.
Ajer said on Friday’s conference call: “I think Voxzogo has the greatest long-term revenue potential. Compared to previously approved products, including Vimizan, this is our largest marketed product to date.”
Bone development is regulated by a variety of proteins. Chondroplasia is caused by mutations in the gene responsible for the production of fibroblast growth factor receptor 3 (FGFR3), a protein that down-regulates bone growth. This mutation causes the protein to be overactive, which in turn leads to people with short limbs and an average height of about 4 feet.Although achondroplasia usually does not impair intelligence, it may require surgery to correct spinal deformities or straighten bent legs, according to National Rare Disease Organization.
FGFR3 is counteracted in the body by a peptide called C-type natriuretic peptide (CNP). Voxzogo is an analog of this peptide. The FDA’s approval of the BioMarin drug is based on the results of a Phase 3 study of 121 children 5 years and older. These participants were randomly assigned to receive a daily injection of study drug or placebo. The goal of the study is to measure the annual growth rate, which is the rate of increase in height after one year of treatment.
The results of the study showed that children who took BioMarin drugs grew 1.57 cm on average compared to children who took placebo. Side effects include injection site reactions, vomiting, and lower blood pressure. Lower blood pressure is listed as a warning on the drug label.
BioMarin estimates that 21,000 children worldwide (of which approximately 3,000 are in the United States) suffer from achondroplasia and are eligible for Voxzogo treatment. The company sets the wholesale price at US$899 per bottle, which Ajer said is consistent with the wholesale prices in France and Germany. A bottle of Voxvogo represents one dose of Voxvogo per day. Calculated at the wholesale price, the annual treatment cost is as high as 320,000 US dollars. Through rebates and discounts, BioMarin calculated that it generates an average income of approximately US$240,000 for each American patient each year. Voxzogo is still under regulatory review in Japan, Brazil and Australia.
The goal of other companies is to challenge BioMarin in achondroplasia. A peptide drug of Ascendis Pharma is in the mid-clinical development stage.Pfizer has a mid-term biological drug candidate through it Acquired Therachon for US$340 million In 2019. Tyra Biosciences raised nearly US$173 million from its September IPO to fund the development of its FGFR3 small molecule inhibitorAlthough the focus of this biotechnology is to prevent the role of FGFR3 in cancer, it also plans to develop its main drug candidate for achondroplasia.
The FDA’s approval of Voxzogo on Friday was carried out under the agency’s accelerated approval pathway, which allows early approval of drugs for serious diseases with limited options. These faster decisions based on surrogate endpoints (indicating that the drug may be effective) require post-marketing studies to confirm patient benefits. To meet this requirement, BioMarin plans to use the ongoing open-label extension study to compare participants with the available natural history of patients with the disease. The main goal will be to assess the final adult height.
Hank Fuchs, BioMarin’s global president of R&D, said that BioMarin’s post-market research program puts the company in a good position to collect adult height data needed to convert accelerated approval to full approval, helping to defend against potential competitors. Fuchs explained that it was obtained in BioMarin After full approval, the annualized growth rate will no longer be a substitute for accelerated approval. This means that subsequent achondroplasia drugs need to meet the same adult height threshold that BioMarin must display in order to be fully approved by the FDA.
Fuchs said: “I think in the future it will take hundreds of patients to cross the finish line with their final adult height before any follower can enter this market.” “We are very satisfied with the post-marketing requirements because it sets up a considerable High threshold.”
Biomarin Said Voxzogo will be available to patients in the United States in late December (if not earlier).
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