The first Amsterdam-based European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) meeting has been held, resulting in just one new initial marketing authorisation and three label extensions.
The Committee recommended granting a conditional marketing authorisation for Bluebird Bio’s gene therapy Zynteglo. The treatment, formerly known as LentiGlobin, is for transfusion-dependent β-thalassemia (TDT).
Patients with TDT cannot produce enough beta-globin, a key component of haemoglobin, the protein that carries the oxygen in the blood from the lungs to the rest of the body. As a consequence, they have far fewer red blood cells than normal and suffer from chronic severe anaemia.
The EMA announced that since Zynteglo addresses an unmet medical need, it benefited from support within the PRIME scheme, the agency's platform for early and enhanced dialogue with developers of promising new medicines.
Due to this, the regulator gave accelerated assessment to Zynteglo in just 150 days, the fastest review time for an advanced therapy medicinal product (ATMP) to date.
Along with the new authorisation, the CHMP also recommended approving indication extensions for Celgene’s Imnovid (pomalidomide), Sanofi’s Mozobil (plerixafor injection) and another Celgene drug, Revlimid (lenalidomide).
Imnovid in combination with bortezomib and dexamethasone has been recommended as a second-line treatment option for adults with multiple myeloma who have received at least one prior treatment regimen including lenalidomide. The drug is already approved for in combination with dexamethasone for adults with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy.
The agency also recommended changing the terms of the marketing authorisation for Revlimid to include its use in combination with dexamethasone, or bortezomib and dexamethasone, or melphalan and prednisone for adults with previously untreated multiple myeloma who are not eligible for transplant.
The CHMP also supported a change to Mozobil’s label to include the treatment of paediatric patients, when used in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours.