New medicines recommended for approval by EMA's CHMP in December

15 December 2023

The European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended a number of new and generic medicines for approval at its December 2023 meeting.

The committee recommended granting a conditional marketing authorization for USA-based Vertex Pharmaceuticals’ (Nasdaq: VRTX) 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 hemoglobin, 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.

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