European Union flag at the European Commission building
A genetic disease that slows bone growth and causes dwarfism now has an approved drug, a biological therapy developed by BioMarin Pharmaceutical.this Decision of the European Commission The drug vosoritide announced on Friday is the first drug therapy for this rare disease, and its complications are usually treated with surgery.
This disease, called achondroplasia, is caused by mutations in a gene that produces fibroblast growth factor receptor 3 (FGFR3), a protein that down-regulates bone growth. The activity of FGFR3 is usually controlled by a peptide called C-type natriuretic peptide (CNP). BioMarin’s drug, injected once a day, is an analog of this peptide. The rare disease drug developer based in San Rafael, California will market the drug under the name “Voxzogo.”
Chondroplasia is usually caused by spontaneous mutations, according to National Rare Disease OrganizationIn about 20% of patients, this condition is hereditary. This condition is the most common cause of dwarfism. People with achondroplasia have short limbs and an average height of 4 feet. Potential complications include spinal cord compression, sleep apnea, bent legs, and repeated ear infections. The main treatment is to solve the problems of the spine and limbs through surgery.
BioMarin has only tested its drug in children in the age range where the growth plate is still open. In an open-label dose exploration phase 2 study involving 35 children aged 5 to 14 years, the annual growth rate of all groups increased from baseline to 42 months.turn out Publish Published in the New England Journal of Medicine in 2019.
The company began testing the drug in a placebo-controlled phase 3 study that enrolled more than 120 children between 5 and 14 years of age. At the end of last year, BioMarin reported that the number of children in the open label long-term expansion of the Phase 3 study kept growing through the second year of continuous treatment. Among the children who received BioMarin medication, the cumulative height gain during the two-year study period was 3.52 cm higher than that of the untreated children.
The extended study that continues to follow children participating in clinical trials has not yet reached the five-year mark. However, BioMarin said that so far, the growth rate has continued and the adverse events have been minor. The most common problems are injection site reactions and hypotension. BioMarin added that the reported adverse events did not include disproportionate bone growth or other skeletal problems, and there is currently no evidence of accelerated bone age or negative changes in bone mineral density.
According to BioMarin, an estimated 11,000 children in Europe, the Middle East, and Africa suffer from achondroplasia and may be eligible for Voxzogo treatment. The drug is still under FDA review. U.S. agencies have set November 20 as the target date for regulatory decisions.
Incyte, Morphosys anticancer drug approved in Europe for the treatment of lymphoma
In other European Commission drug approval news, Incyte and Morphosys were approved Conditional marketing authorization Tafasitamab is used as a treatment for diffuse large B-cell lymphoma (DLBCL). The antibody drug decision covers adults who have relapsed or did not respond to early treatment, as well as adults who are not eligible for stem cell transplantation. The drug will be marketed in Europe under the name “Minjuvi”, and the approval includes a combination with Bristol-Myers Squibb’s anti-cancer drug lenalidomide, followed by Minjuvi as a monotherapy.
The European approval of Minjuvi was about a year later FDA granted accelerated approval for the drug. Incyte and MorphoSys share the rights of the drug; the partners jointly sell the therapy in the United States under the name “Monjuvi”.Incyte, located in Wilmington, Delaware, has exclusive commercialization rights for the drug outside the United States
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