EMA says Rocket Pharmaceuticals withdraws MA application for Fanskya

The European Medicines Agency said that Rocket Pharmaceuticals (RCKT) B.V. withdrew its application for a marketing authorisation of Fanskya for the treatment of Fanconi anaemia type A, an inherited condition that affects the bone marrow, the spongy tissue inside the large bones where blood cells are made. The company withdrew the application on 11 August 2025. Based on the review of the data, at the time of the withdrawal, the Agency had some concerns and its provisional opinion was that Fanskya could not have been authorised for the treatment of Fanconi anaemia type A. The Agency had concerns about the potential safety of the medicine, as results showing how many copies of the FANCA gene enter stem cells are only available after treatment. This is important because introducing a high number of gene copies into stem cells may impact how they function. As the possible risk of the gene changing the normal functioning of cells could not be fully evaluated before the medicine had been given to the patient, the Agency requested a comprehensive risk assessment.