Extended indication

Myeloid/Lymphoid Neoplasms ((8p11 var)

Therapeutic value

No estimate possible yet

Total cost

67,500.00

Registration phase

Clinical trials

Product

Active substance

Pemigatinib

Domain

Hematology

Reason of inclusion

Indication extension

Main indication

Myeloproliferative disorders

Extended indication

Myeloid/Lymphoid Neoplasms ((8p11 var)

Proprietary name

Pemazyre

Manufacturer

Incyte

Portfolio holder

Incyte

Mechanism of action

Tyrosine kinase inhibitor

Route of administration

Oral

Therapeutical formulation

Tablet

Budgetting framework

Intermural (MSZ)

Additional remarks
FGFR-1 rearrangement positivity status must be known prior to initiation of Pemazyre therapy. Assessment for FGFR 1 rearrangement positivity in tumor specimen should be performed with an appropriate diagnostic test.

Registration

Registration route

Centralised (EMA)

Type of trajectory

Normal trajectory

ATMP

No

Submission date

December 2023

Expected Registration

September 2024

Orphan drug

Yes

Registration phase

Clinical trials

Additional remarks
Registration will be based on results of the FIGHT-203 study: a single arm, open-label phase 2 study evaluating the efficacy and safety of pemigatinib monotherapy in patients with MLN with FGFR1 rearrangement.

Therapeutic value

Current treatment options

MLN with FGFR1 rearrangement is a rare and progressive hematological malignancy. Incidence approx. <1 case per 100.000 persons/year. For patients with MLN with FGFR1 rearrangement in chronic phase, the National Comprehensive Cancer Network (NCCN) US-guidelines identify enrolment in clinical trials as the preferred treatment option. In the absence of a clinical trial, the current recommended approach is monotherapy with a tyrosine kinase inhibitor (TKI) with activity against FGFR1.The NCCN guidelines list pemigatinib, midostaurin, and ponatinib as TKIs with activity against FGFR1. However, none of these drugs (except pemigatinib in US) have been approved for the treatment of MLN with FGFR1 rearrangement.

Therapeutic value

No estimate possible yet

Substantiation

Not assessed, no approved treatment for MLN with FGFR-1 rearrangement available. Latest results of the FIGHT-203 study (presented at 2022 ASH) showed in treatment naïve or previously treated patients with MLN with FGFR-1 rearrangements: Of 38 evaluable patients, 28 (73.7%) achieved a clinical complete response (CR; primary endpoint). The rate of complete cytogenetic responses (CCyR) was 70.0% (n=28/40). The rates of CRs and CCyRs in previously treated, efficacy-evaluable patients were 75.8% (n=25/33) and 71.4% (n=25/35), respectively. Of 21 patients with chronic phase (CP) disease, 18 (85.7%) exhibited both clinical CRs and CCyRs. Of 17 patients with blast phase (BP) disease, 9 presented with clinical CRs (52.9%), and 8 (47.1%) with CCyRs. The median exposure to pemigatinib treatment was 6.3 months (range: 0.5-50.2).

Duration of treatment

Median 6.3 month / months

Frequency of administration

1 times a day

Dosage per administration

13,5 mg

References
ASH 2022 presentation details: Lead Author: Srdan Verstovsek, poster P1732: FIGHT-203, an ongoing phase 2 study of pemigatinib in patients with myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement (MLNFGFR1): a focus on centrally reviewed clinical and cytogenetic responses in previously treated patients. ClinicalTrials.gov. FIGHT-203. Accessed Dec 2022. https://clinicaltrials.gov/ct2/show/NCT03011372.
Additional remarks
Latest results of the FIGHT-203 study (presented at 2022 ASH) showed in treatment naïve or previously treated patients with MLN with FGFR-1 rearrangements: Of 38 evaluable patients, 28 (73.7%) achieved a clinical complete response (CR; primary endpoint). The rate of complete cytogenetic responses (CCyR) was 70.0% (n=28/40). The rates of CRs and CCyRs in previously treated, efficacy-evaluable patients were 75.8% (n=25/33) and 71.4% (n=25/35), respectively. Of 21 patients with chronic phase (CP) disease, 18 (85.7%) exhibited both clinical CRs and CCyRs. Of 17 patients with blast phase (BP) disease, 9 presented with clinical CRs (52.9%), and 8 (47.1%) with CCyRs. The median exposure to pemigatinib treatment was 6.3 months (range: 0.5-50.2).

Expected patient volume per year

Patient volume

< 1

Market share is generally not included unless otherwise stated.

Additional remarks
Very rare disease. No published estimates of incidence or prevalence Incidence estimated: <1 case per 100.000 persons/year.

Expected cost per patient per year

Cost

65,000.00 - 70,000.00

Additional remarks
Lijstprijs €7.732

Potential total cost per year

Total cost

67,500.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.