A treatment aimed at improving the growth of children with dwarfism has just been authorized in the United States.
- Voxzogo, a treatment to improve the growth of children with dwarfism, has just been authorized in the United States.
- In the European Union, it has been authorized since last August. Marketing authorization applications are in progress in Japan, Brazil and Australia.
According to Departmental house for people with disabilities, individuals with bone dwarfism or short people represent 8,000 to 10,000 people in France and nearly 130,000 worldwide. In the United States, for the first time, the Food and Drug Administration (FDA) has just authorized a treatment to improve the growth of children with dwarfism. The US Medicines Agency has limited its approval to those at least five years old with achondroplasia. This should be available across the Atlantic by mid-December.
Achondroplasia affects one birth in 15,000
Achondroplasia is the most common form of dwarfism. According to association of short people (APPT), this can be defined as a genetic disease that mainly affects the limbs. The growth of the facial bones of people with achondroplasia may also be disturbed, but the trunk remains of normal size. The average height of an adult reaches four feet and never exceeds one meter thirty-five. According to’National Institute of Health and Medical Research (Inserm), achondroplasia concerns approximately one in 15,000 births. In the most severe cases, patients may suffer from deformities of the skull and/or vertebrae. This can lead to both neurological and orthopedic complications.
More than 10,000 children affected by this treatment in the United States
Voxzogo is the name of the treatment now authorized in the United States. This is marketed by the BioMarin laboratory. It is taken daily, once a day, by subcutaneous injection. “This clearance meets a medical need for more than 10,000 children in the United States and underscores the FDA’s commitment to making new therapies available for rare diseases, says Theresa Kehoe, director of the Division of General Endocrinology at the FDA’s Center for Drug Evaluation and Research in the communicated. Children with short stature and achondroplasia thus have a treatment option that targets the underlying cause of their short stature.“.
Voxzogo stimulates growth in patients with achondroplasia
Achondroplasia comes from a mutation in a gene called FGFR3. This produces a protein that is involved in the regulation of bone growth. The normal process by which an individual grows is as follows: the growth factor FGF attaches to and detaches from the FGFR3 receptor. This mechanism is therefore disrupted in people withachondroplasia due to the mutation of FGFR3, which therefore prevents their growth. The Voxzogo treatment works by reducing the activity of the growth regulation gene and stimulates bone growth.
Some observed side effects
Before being marketed, the treatment has of course validated all phases of clinical trials up to the third, which included 121 participants aged 5 and 14 years. And the conclusions are convincing: those who took Voxzogo grew 1.57 centimeters more than those who had a placebo. A few side effects have nevertheless been observed, such as vomiting, joint pain, stomach aches, a decrease in blood pressure or mainly skin reactions in the area where the product is injected.