Pemazyre

Authorised

This medicine is authorised for use in the European Union

pemigatinib

Overview

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 (target) 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 previous treatment with at least one cancer medicine.

Cholangiocarcinoma is rare, and Pemazyre was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 24 August 2018.

Pemazyre contains the active substance pemigatinib.

How is Pemazyre used?

Pemazyre is available as tablets to be taken by mouth. The medicine can only be obtained with a prescription and treatment must be started by a doctor experienced in diagnosis and treatment of the disease. It is taken in three-week cycles consisting of two weeks where Pemazyre is taken daily followed by a week without the medicine. Treatment can continue for as long as the patient benefits from it and side effects are manageable.

For more information about using Pemazyre, see the package leaflet or contact your doctor or pharmacist.

How does Pemazyre work?

The active substance in Pemazyre, pemigatinib, belongs to a group of medicines called protein kinase inhibitors. It works by blocking the activity of receptors called fibroblast growth factor receptors (FGFRs). Abnormal FGFRs are found on the surface of cancer cells and are involved in the growth and spread of the cancer. By blocking their activity, Pemazyre reduces the growth and spread of the cancer.

What benefits of Pemazyre have been shown in studies?

Pemazyre was effective in reducing the size of the cancer lesions in one main study involving 108 patients with biliary tract cancer with abnormal FGFR2. Around 37% of patients had a shrinkage of their cancer that lasted on average for 8 months.

What are the risks associated with Pemazyre?

The most common side effects with Pemazyre (which may affect more than 1 in 10 people) are high or low levels of phosphate in the blood, alopecia (hair loss), diarrhoea, problems with the nails, tiredness, nausea (feeling sick), dysgeusia (taste disturbances), stomatitis (inflammation of the lining of the mouth), constipation, joint pain, dry mouth, eyes and skin, rash and numbness on the palms and soles, low levels of sodium in the blood and high levels of creatinine in the blood, which could indicate kidney problems.

Pemazyre must not be taken with the herbal medicine St John’s wort. For the full list of side effects and restrictions with Pemazyre, see the package leaflet.

Why is Pemazyre authorised in the EU?

Pemazyre is considered effective in patients with biliary tract cancer that progressed after at least one prior treatment and for whom there are no other authorised treatments. Patients can tolerate the side effects of the medicine when they are closely monitored and treated if needed. The European Medicines Agency therefore decided that Pemazyre’s benefits are greater than its risks and it can be authorised for use in the EU.

Pemazyre has been given ‘conditional authorisation’. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.

What information is still awaited for Pemazyre?

Since Pemazyre has been given conditional authorisation, the company that markets the medicine will provide the final results of the main study on the safety and effectiveness of Pemazyre and the results of a study comparing Pemazyre with gemcitabine and cisplatin (other cancer medicines) in patients with newly diagnosed biliary tract cancer.

What measures are being taken to ensure the safe and effective use of Pemazyre?

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Pemazyre have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Pemazyre are continuously monitored. Side effects reported with Pemazyre are carefully evaluated and any necessary action taken to protect patients.

Other information about Pemazyre

Pemazyre received a conditional marketing authorisation valid throughout the EU on 26 March 2021.

Pemazyre : EPAR - Medicine overview

Reference Number: EMA/95344/2021

Pemazyre : EPAR - Risk-management-plan summary

Product information

Pemazyre : EPAR - Product information

Pemazyre : EPAR - All authorised presentations

Product details

• Name of medicine:
  Pemazyre
• Active substance:
  pemigatinib
• International non-proprietary name (INN) or common name:
  pemigatinib
• Therapeutic area (MeSH):
  Cholangiocarcinoma
• Anatomical therapeutic chemical (ATC) code:
  L01EN02

Pharmacotherapeutic group

Antineoplastic agents

Therapeutic indication

Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed  after at least one prior line of systemic therapy.

Authorisation details

• EMA product number:
  EMEA/H/C/005266
• Additional monitoring:

  This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

• Conditional approval:

  This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.

• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation holder:
  Incyte Biosciences Distribution B.V.

  Paasheuvelweg 25; 1105 BP Amsterdam; The Netherlands

• Opinion adopted:
  28/01/2021
• Marketing authorisation issued:
  26/03/2021
• Revision:
  6

Assessment history

Changes since initial authorisation of medicine

Pemazyre : EPAR - Procedural steps taken and scientific information after authorisation

Pemazyre-H-C-PSUSA-00010923-202210 : EPAR - Scientific conclusions and grounds for the variation to the terms of the marketing authorisation

Reference Number: EMA/366120/2023

Pemazyre-H-C-PSUSA-00010923-202204 : EPAR - Scientific conclusions and grounds recommending the variation to the terms of the marketing authorisation new

AdoptedReference Number: EMA/93779/2023

Initial marketing authorisation documents

Pemazyre : Orphan maintenance assessment report (initial authorisation)

AdoptedReference Number: EMADOC-1700519818-628240

Pemazyre : EPAR - Public assessment report

AdoptedReference Number: EMA/CHMP/105411/2021

CHMP summary of positive opinion for Pemazyre

AdoptedReference Number: EMA/35501/2021 Rev.1