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PEMAZYRE (pémigatinib)

Opinions on drugs - Posted on Sep 30 2021

Reason for request

First assessment

Key points

Favourable opinion for reimbursement only in the subgroup of patients with locally advanced or metastatic intrahepatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement who have progressed after at least one prior line of systemic therapy and are not eligible for chemotherapy with FOLFOX.

Unfavourable opinion for reimbursement in other situations.

 What therapeutic improvement?

No clinical added value in the therapeutic strategy on the basis of currently available data.

Role in the care pathway?

According to the national guidelines of the TNCD (Thésaurus National de Cancérologie Digestive - French National Thesaurus of Gastrointestinal Oncology), the first-line treatment in metastatic intrahepatic cholangiocarcinoma is chemotherapy with gemcitabine and cisplatin. In France the GEMOX protocol is also used in this situation. In patients requiring second-line systemic therapy, the FOLFOX protocol is a treatment option.

Role of PEMAZYRE (pemigatinib) in the care pathway:

Considering:

  • the limited efficacy data available based on the results of a non-comparative phase 2 study, although a comparison with FOLFOX chemotherapy was feasible for second-line treatment,
  • the common, severe and, in some cases unusual adverse events (phosphorous metabolism disturbances, retinal detachments),

The Transparency Committee considers that, on the basis of currently available data, PEMAZYRE is a treatment option only for patients with intrahepatic cholangiocarcinoma with an FGFR2 fusion or rearrangement from the second line of treatment and who are not eligible for chemotherapy with FOLFOX. In this context, a simplified chemotherapy regimen, such as the LV5FU2 protocol also remains another treatment option.


Clinical Benefit
Low

The Committee deems that the clinical benefit of PEMAZYRE (pemigatinib) is LOW in the subgroup of patients with locally advanced or metastatic intrahepatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement who have progressed after at least one prior line of systemic therapy and are not eligible for chemotherapy with FOLFOX.

 
Insufficient

The Committee deems that the clinical benefit of PEMAZYRE (pemigatinib) is INSUFFICIENT to justify public funding cover in view of the available alternatives in the rest of the MA population.

Clinical Added Value
no clinical added value
  • Within the reimbursement scope retained by the Committee:

Considering:

  • data from one of three cohorts in a non-comparative phase 2 study, although a comparison with FOLFOX chemotherapy was feasible at the time the study was conducted,
  • the medical need partially met within the scope of the indication but to a lesser degree in the event of non-eligibility for the FOLFOX protocol,
  • the additional toxicity, in particular the incidence of grade ≥ 3 adverse events (AE) of 59.8% and that of serious AEs of 40.2%. In addition, the development of serous retinal detachment and hyperphosphataemia were identified as important risks in the Risk Management Plan,

The Transparency Committee considers that PEMAZYRE (pemigatinib) provides no clinical added value (CAV V) in the treatment of the subgroup of patients with locally advanced or metastatic intrahepatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement who have progressed after at least one prior line of systemic therapy and are not eligible for chemotherapy with FOLFOX.

  • Within the scope included in the MA but not retained for reimbursement: not applicable.
Not applicable

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