Achondroplasia is an inherited bone growth disorder that prevents the change of cartilage into bone.
Washington:
The US Food and Drug Administration on Friday approved a drug for the first time to improve growth in children with the most common dwarfism.
Voxzogo, manufactured by the American pharmaceutical company BioMarin, is authorized in the United States to treat children over 5 years of age with bony hyperplasia whose growth plates – the cartilage areas at the ends of long bones – remain open. , which means they still have growth potential.
“With this action, children with short stature due to hyperplasia have a treatment option that targets the underlying cause of short stature,” FDA endocrinologist Theresa Kehoe said in a press release. their baby”.
Achondroplasia is an inherited bone growth disorder that prevents the change of cartilage into bone. Adults with this condition are on average about 1.2 meters tall.
According to the National Institutes of Health, it can cause health problems including breathing problems, scoliosis, obesity, and recurrent ear infections. Life expectancy is usually near normal.
Amer Haider, co-founder of the nonprofit group Growing Stronger, said in a BioMarin statement: “As a parent of a child with achondroplasia, I see the availability of treatments impacting the well-being of children. Bone development is an important step forward.
Grow Stronger is dedicated to improving the quality of medical care for the little ones through supporting research.
But growing taller isn’t necessarily the goal for everyone with hyperplasia of the eyelids.
When BioMarin shared preliminary trial results in 2015, the dwarfism advocacy organization Little People of America (LPA) emphasized that the organization’s celebration of dwarfism was a “valuable contribution to the diversity of the human condition.”
“While we encourage individuals and families to make the decisions that are best for them, we stress that emerging treatments are not necessary for people with dwarfism to be able to live a better quality of life.” attractive, healthy and productive,” the LPA statement said.
The year-long study
The FDA says the safety and effectiveness of Voxzogo, or vosoritide, have been evaluated in a one-year, double-blind study of participants 5 years of age and older with achondroplasia who present with open or raised plaques. chief.
The children who received the Voxzogo injection were on average about 0.6 inches (1.57 cm) taller than the children who received the placebo during the study period.
People with achondroplasia have an inherited mutation that causes a growth-regulating gene to become overactive, preventing normal bone growth. Voxzogo works by fighting mutations.
The most common side effects were injection site reactions, vomiting, and a severe drop in blood pressure.
The European Commission approved the drug for the treatment of children over the age of two who are still developing at the end of August this year.
The drug is expected to be available in the United States in mid to late December, according to BioMarin.
(Except for the title, this story has not been edited by NDTV staff and is published from an aggregated feed.)
