Pemigatinib

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.

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.

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.