The EHERCC and The EHE Foundation are continuing to engage with the European Medicines Agency (EMA) regarding the approval of sirolimus for the treatment of EHE. There is a growing body of clinical evidence supporting the view that sirolimus may be efficacious in the treatment of EHE. However, although the drug is widely used as an immunosuppressant post-transplant, and is now being widely prescribed off-label for EHE because it is not formally approved for EHE treatment. This means that many patients are still unable to access the drug.
Hugh Leonard explained where the process has got to with the EMA:
“In 2023 we engaged with the EMA through their formal process called Scientific Advice, where applicants can provide supporting data and pose questions relating to the data and whether it is sufficient to support the repurposing of the drug. This is not an approval, but a process designed to help applicants understand if additional data is required. This was all done under the auspices of the European Organisation for Research and Treatment of Cancer (EORTC), under the leadership of Dr Silvia Stacchiotti.
The Scientific Advice we received did clarify that some additional data may be required. It also appeared to clarify for the regulator that their standard approach with its prescriptive and rigid structure was not suitable when considering ultra-rare diseases. The first half of 2024 was therefore focused on addressing the points raised by the EMA, including the development of disease-specific criteria for evaluating whether a drug is working. This is necessary because the traditional criteria for assessing a drug are based on common cancers where tumour shrinkage is expected. As our patient community understands, a key objective with EHE is to re-establish disease stability once EHE has become progressive. We are very pleased that the EMA understand these challenges of testing the activity of a drug in such rare diseases and are actively engaging to help find solutions.”
So where is the process now? The EORTC team have recently submitted a draft of a further Scientific Advice submission, and the EMA have provided preliminary comments. Those comments have been actioned and the team formally submitted the document at the very start of July. They will now await the EMA’s response, which they expect to receive after the summer.
We look forward to updating the patient community at that time.