The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) on Friday recommended a conditional marketing authorization for Bluebird Bio’s gene therapy Zynteglo (Autologous CD34+ cells encoding βA-T87Q-globin gene).
Zyntelgo addresses the underlying genetic cause of transfusion-dependent beta-thalassemia, a rare inherited blood condition that causes severe anemia. Zynteglo is intended for patients 12 years and older who need regular blood transfusions to manage their disease and do not have a matching donor for a stem cell transplant.
“In the two main studies to demonstrate the effects of Zynteglo it was shown that the majority of patients who do not have a β0/β0 genotype treated with Zynteglo no longer needed regular blood transfusions. The most common side effects were thrombocytopenia (low blood platelet count), abdominal pain, non-cardiac chest pain, pain in the extremities, dyspnoea and hot flush,” EMA said.
The gene therapy was granted an accelerated assessment and was reviewed in just 150 days, cutting as much as two months off the time a typical application takes to make it through the centralized procedure. EMA said it was “the fastest review time for an ATMP to date.”
The conditional aspect of the recommended authorization is because the company submitted less complete data than normally expected, EMA said, which is what happens when the agency deems a medicine’s immediate availability to patients outweighs the risk of not having more data.
And since Zynteglo addresses an unmet medical need, the agency said it benefited from the support of its PRIME scheme and Adaptive Pathways program, which provide for early and enhanced dialogue between the agency and drug developers. Zynteglo was also granted an orphan designation in 2013.
“As always at time of approval, EMA's committee for orphan medicines (COMP) will review the orphan designation to determine whether the information available to date allows maintaining Zynteglo’s orphan status and granting this medicine ten years of market exclusivity,” EMA said
Bluebird said it’s the company’s first gene therapy submitted for regulatory approval, and a final decision by the European Commission is anticipated in the second quarter of 2019.
Last September, EMA’s CHMP recommended approval
of another gene therapy, Spark Therapeutics’ Luxturna (voretigene neparvovec), for the treatment of a rare genetic disorder that causes vision loss and usually leads to blindness.
And regulators expect more gene and cell therapies are coming.
By 2020, FDA said it expects
to be receiving upward of 200 INDs for cell and gene therapies each year, adding to the 800 active INDs for such products already filed with the agency. By 2025, FDA predicts the agency will be approving between 10 and 20 cell and gene therapy products annually.
EMA Guidelines on Gene Therapy