Pemazyre - opinion on variation to marketing authorisation
Opinion
EMA has issued an opinion on a change to this medicine's authorisation
pemigatinib
Post-authorisation
Human
Pemazyre is a cancer medicine used to treat adults with cholangiocarcinoma (biliary tract cancer or cancer of the bile ducts) when the cancer cells have an abnormal form of a receptor called FGFR2 on their surface. Pemazyre is used when the cancer has spread to other parts of the body or cannot be removed by surgery and has worsened after treatment with at least one cancer medicine.
Pemazyre has been authorised in the EU since March 2021 under a conditional marketing authorisation. It contains the active substance pemigatinib and is available as tablets to be taken by mouth.
Further information on Pemazyre’s uses can be found on the Agency’s website: ema.europa.eu/en/medicines/human/EPAR/pemazyre
The company applied to extend the use of Pemazyre to the treatment of adults with myeloid/lymphoid neoplasms when the cancer cells have an abnormal form of a receptor called FGFR1 on their surface.
Pemazyre was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 October 2019 for the treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangements or fusion in the genes for some proteins called receptor tyrosine kinases (PDGFRA, PDGFRB, FGFR1 and PCM1-JAK2). Further information on the orphan designation can be found on the Agency’s website: ema.europa.eu/medicines/human/orphan-designations/EU3192216.
The active substance in Pemazyre, pemigatinib, belongs to a group of medicines called protein kinase inhibitors. It works by blocking the activity of tyrosine kinase receptors, such as FGFR receptors. Abnormal FGFR receptors are found on the surface of cancer cells and are involved in the growth and spread of the cancer. By blocking their activity, Pemazyre slows the progression of the cancer.
The company submitted data from a study in 45 adults with myeloid/lymphoid neoplasms with FGFR1 rearrangement. Patients received Pemazyre for 6 months and the main measure of effectiveness was the number of patients whose disease responded to treatment. The study did not compare Pemazyre with another medicine or placebo (a dummy treatment).
EMA considered that the data were not sufficiently comprehensive, meaning that there were remaining uncertainties about the benefits and risks of the medicine. The study did not compare Pemazyre with another treatment or placebo and included a small number of patients, reflecting a limited range of the different forms of the disease. This, in addition to changes that were made in the design of study while it was carried out could have affected the validity of the results.
The company was requested to generate additional data after authorisation on the benefits and risks of Pemazyre in myeloid/lymphoid neoplasms with FGFR1 rearrangement to address the remaining uncertainties. The agency asked the company to set up a registry-based study to provide further data, but the company considered that it would not be possible to provide the required data.
Therefore, although there is a need for better treatment options in patients with myeloid/lymphoid neoplasms, the results provided by the company so far are not considered sufficient to support an authorisation without a commitment to generate new data and further inform the benefit-risk profile of Pemazyre in this new use. The fact that the company would not be able to generate additional data to address the remaining uncertainties on the benefits and risks of Pemazyre meant that a conditional marketing authorisation could not be granted for Pemazyre in the treatment of myeloid/lymphoid neoplasms with FGFR1 rearrangement. The Agency therefore recommended refusing the conditional marketing authorisation for this use.
The company informed the Agency that there are no consequences for patients who are receiving Pemazyre for myeloid/lymphoid neoplasms with FGFR1 rearrangement in clinical trials or in compassionate use programmes.
If you are in a clinical trial or compassionate use programme and need more information about your treatment, speak with your clinical trial doctor.
There are no consequences for Pemazyre in its authorised use.