First EMA/CHMP meeting in Amsterdam sparse on recommendations

The March 25-28 2019 meeting of the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) was held for the first time in its new post-Brexit offices in Amsterdam, Netherlands, but came back with only recommendations for one new initial marketing authorization and just three extensions of indication.

The Committee recommended granting a conditional marketing authorization for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), from US biotech firm bluebird bio (Nasdaq: BLUE), an advanced therapy medicinal product (ATMP) for beta-thalassemia, a rare inherited blood condition that causes severe anemia. 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, the 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.