Myeloma Patients Europe (MPE), in close collaboration with Myeloma UK, and along with almost 30 member and non-member associations across Europe, has submitted a letter to the Committee for Human Medicinal Products (CHMP) within the European Medicines Agency (EMA) to encourage them to reconsider the negative recommendation for ixazomib, and provide information to the appeal of their decision on ixazomib.

The appeal, filed by the pharmaceutical company Takeda, is reviewing the negative recommendation from the CHMP in May. The CHMP is the scientific committee within the EMA which provides advice on whether or not a drug is safe and effective in patients. Without a positive recommendation, it means that ixazomib is unlikely to be granted a European marketing authorisation, and doctors will be unable to routinely prescribe it for their patients.

Ixazomib is a new proteasome inhibitor for the treatment of relapsed and refractory myeloma. In their justification for this decision, the CHMP committee issued a statement which outlined that whilst the drug is safe, they “considered that the data from the main study were insufficient to demonstrate a benefit of ixazomib in the treatment of multiple myeloma”.

MPE considers ixazomib to be a critical new drug in the treatment of relapsed myeloma and one that will significantly add to the range of treatment options available to myeloma doctors and their patients. There is a strong clinical support for using ixazomib in relapsed myeloma, as evidenced by the widespread uptake of this drug through the EMA approved compassionate use programme.

In particular, MPE encourages the CHMP to further consider the following information about ixazomib (in combination with lenalidomide and dexamethasone):

• It is a safe and effective treatment option for relapsed myeloma patients and improves progression free survival (PFS) on average between 4-6 months compared to lenalidomide and dexamethasone alone. This represents a significant and prolonged period for patients and their carers/family members, specially taking into account the relapsing and remitting nature of myeloma. Because of it, is essential for patients across Europe to access a range of novel treatments and treatment combinations available at each stage for doctors to prescribe.

• The unmet need for an oral proteasome inhibitor (i.e. a drug similar to Velcade or carfilzomib but administered orally), particularly for groups of patients who are unable to regularly attend hospital appointments.

• The impact that ixazomib has on PFS is likely to be more substantial when used in the real-world, given the increased ability to provide personalised treatment (i.e. through dose modifications, supportive care etc). Doctors who have accessed ixazomib via the EMA approved compassionate use programme have seen patients respond very well to it in the real-world setting and would value the ability to use it routinely in their patients.

• Trials involving ixazomib have demonstrated that it works well in patients who have high-risk myeloma and has the potential to overcome the high-risk features of myeloma. This is a major area of unmet need, given that these patients currently have very poor outcomes

On behalf of myeloma doctors, patients and carers, MPE, along with the associations that sign the statement, very much hopes that the CHMP overturns its negative recommendation and ensures ixazomib is added to the treatment options for patients. Only through ensuring the approval of new and promising treatments for myeloma patients across Europe, can we keep survival rates in myeloma on an upward trajectory.

Over the coming months, we will continue to monitor the development of the appeal, and update our members about the outcomes and implications. In the meantime, should you have any questions or comments, please email policy@mpeurope.org.