EMA’s human medicines committee (CHMP) adopted recommendations for one new initial marketing authorisation and three extensions of indication at its first meeting in Amsterdam in March 2019.

The Committee recommended granting a conditional marketing authorisation for Zynteglo (Autologous CD34+ cells encoding βA-T87Q-globin gene), an advanced therapy medicinal product (ATMP) for beta-thalassaemia, a rare inherited blood condition that causes severe anaemia. Zynteglo is intended for adult and adolescent patients 12 years and older who need regular blood transfusions to manage their disease and have no matching donor for a stem cell transplant.

Since Zynteglo addresses an unmet medical need, it benefited from support within the PRIME scheme, EMA’s platform for early and enhanced dialogue with developers of promising new medicines. This interaction led to a more robust application package which allowed accelerated assessment of Zynteglo in 150 days, the fastest review time for an ATMP to date. Zynteglo was designated as an orphan medicine during its development.

Three recommendations on extensions of therapeutic indication

The Committee recommended extensions of indication for ImnovidMozobil and Revlimid.

Outcome of re-examination of recommendation on omega-3 fatty acid medicines

The CHMP confirmed that omega-3 fatty acid medicines containing a combination of an ethyl ester of eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA) are not effective in preventing further problems with the heart and blood vessels in patients who have had a heart attack. This is the outcome of a re-examination requested by some of the companies that market the medicines concerned, following the Committee’s original recommendation in December 2018.

Posted on the EMA website on 29 March 2019