Seven new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended seven medicines for approval at its December 2023 meeting.
The committee recommended granting a conditional marketing authorisation for Casgevy* (exagamglogene autotemcel), an advanced therapy medicinal product (ATMP) for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease, two inherited rare diseases caused by genetic mutations that affect the production or function of haemoglobin, the protein found in red blood cells that carries oxygen around the body. This is the first medicine using CRISPR/Cas9, a novel gene-editing technology. Casgevy was supported through EMA’s priority medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for medicines that have a particular potential to address patients’ unmet medical needs. See more details in the news announcement in the grid below.
Skyclarys* (omaveloxolone) received a positive opinion from the CHMP for the treatment of Friedreich’s ataxia, an inherited disease causing a range of symptoms that worsen over time, including difficulty walking, inability to co-ordinate movements, muscle weakness, speech problems, damage to the heart muscle and diabetes…
