Biomarin’s Dwarfism Drug Receives EMA ApprovalBy Miriam Bello | Fri, 08/27/2021 - 13:18
The European Commission approved the use of Biomarin Pharmaceutical Inc's treatment for one of the most common forms of dwarfism. The approval comes after the European Medicines Agency (EMA) endorsed what has now become the first achondroplasia therapy to get an approval in the region, according to Reuters. BioMarin's drug will sell under the brand name Voxzogo.
Klaus Mohnike, professor of Paediatrics at Magdeburg University Hospital, told Reuters that “this regulatory approval is based on improved height gain, one important determinant of day-to-day function for people with achondroplasia." The European Commission, following the EMA's recommendation, authorized Voxzogo for use in children from two years old until their growth plates are closed, which happens after puberty.
Achondroplasia is a disorder of bone growth that prevents the changing of cartilage to bone, particularly in the long bones of the arms and legs. Achondroplasia can cause health complications such as interruption of breathing (apnea), obesity, recurrent ear infections and an exaggerated inward curve of the lumbar spine (lordosis). More serious problems include a narrowing of the spinal canal that can compress the upper part of the spinal cord and a buildup of fluid in the brain.
In a Phase 3 study, shared BioPharma Dive, treatment with Voxzogo led to faster bone growth than did placebo over a year, a benefit that built on previous findings from a mid-stage trial. Most side effects were mild in nature and no severe adverse events related to Voxzogo were reported by BioMarin.
Increased Attention to Rare Diseases
Achondroplasia is only one of numerous rare diseases. The search for treatments has motivated the development of innovative treatments and driven many public actions, like the decision made by Mexico’s General Health Council on August 11 to prepare a census of patients with rare diseases. This decision is a vital first step for those living with a rare disease. The census will make it possible to identify what rare diseases exist in Mexico and how many people live with them. With this information, the General Health Council can begin to develop solutions that provide patients greater and better opportunities for care.
In Mexico, “there have been many advances to improve the public sector’s coverage and attention to low prevalence diseases like those we focus on,” told MBN David López, Country Manager of BioMarin Pharmaceuticals. “To date, many patients are able to get their treatments through the public sector but there is still much to cover. Patient groups, organizations and the industry have been working to increase inclusion as the prevalence of these diseases is latent.”