Incyte (Nasdaq:INCY) today announced that the Japanese Ministry
of Health, Labour and Welfare (MHLW) has approved Pemazyre®
(pemigatinib), a selective fibroblast growth factor receptor (FGFR)
inhibitor, for the treatment of myeloid/lymphoid neoplasms (MLNs)
with FGFR1 fusion (also known as 8p11 myeloproliferative syndrome).
MLNs are a rare, aggressive group of cancers characterized by an
over-production of myeloid cells, or bone tissue, with the tendency
to rapidly progress to an acute myeloid leukemia (AML).
“The MHLW approval of Pemazyre in MLNs is an important step
toward potentially providing a therapeutic option for Japanese
patients with this rare condition,” said Lothar Finke, M.D., Ph.D.,
Group Vice President and General Manager, Incyte Asia. “Bringing
this first and only approved therapy to MLN patients in Japan
demonstrates Incyte’s commitment to finding solutions for critical
unmet medical needs regardless of the size of the patient
population – as is the case with MLNs, an extremely rare disease
that is estimated to affect fewer than 100 patients worldwide.”
The approval was based on data from the Phase 2 FIGHT-203 study,
a multicenter open-label, single-arm trial that evaluated the
safety and efficacy of Pemazyre in 41 patients in myeloid or
lymphoid neoplasms with FGFR1 fusion gene positive who received
Pemazyre 13.5 mg orally once daily continuously or intermittently.
The primary endpoint, investigator-assessed complete response rate,
was 62.5% (95% CI: 45.8 - 77.3). The complete response rate in the
continuous dosing population was 66.7% (95% CI: 46.0 - 83.5). The
most common adverse reactions observed in patients receiving
Pemazyre were hyperphosphatemia (70.7%), alopecia (56.1%), diarrhea
(43.9%) and stomatitis (43.9%).
Previously, the MHLW granted Orphan Drug Designation (ODD) for
Pemazyre – a designation granted to investigational compounds
intended to treat rare diseases that affect fewer than 50,000
people in Japan, and for which there is a high medical need1.
Designated orphan drugs are also eligible for priority review for
marketing authorizations to ensure supply to clinical settings at
the earliest opportunity1. The MLN ODD is the second such
designation granted to Pemazyre by the MHLW, having also received
ODD for cholangiocarcinoma, a type of biliary tract cancer.
MLNs with FGFR1 rearrangement are a form of very rare
hematological cancers caused by a chromosomal abnormality
(translocation) in which the chromosome breaks where the FGFR1 gene
is located (position 11 in the short arm of chromosome 8: location
8p11) and fuses with fragments (genes) of other chromosomes.
Various partner genes cause a constitutive activation of the FGFR1
tyrosine kinase, impacting cell proliferation and survival. These
cancers are largely divided into two phases according to clinical
presentation: the chronic phase, if diagnosed as myeloproliferative
disorder or myelodysplastic syndrome, or acute phase, if diagnosed
as acute leukemia. The prognosis is unfavorable, and while
allogenic hematopoietic stem cell transplant is considered to be
the only currently available treatment option that may achieve cure
or long-term remission, no standard of care has been
established.
About FIGHT-203
FIGHT-203 is a Phase 2, multicenter trial that enrolled patients
18 years and older with myeloid/lymphoid neoplasms (MLNs) with a
fibroblast growth factor receptor 1 (FGFR1) rearrangement.
Sponsored by Incyte, the study evaluated the safety and efficacy of
pemigatinib for the treatment of adults with MLNs with FGFR1
rearrangement. Patients received pemigatinib 13.5 mg once daily in
21-day cycles, either on a continuous schedule (the approved
recommended starting dosage for use in patients with MLNs with
FGFR1 rearrangement) or as an intermittent schedule (14 days on, 7
days off, an unapproved dosage regimen in MLN with FGFR1
rearrangement). Pemigatinib was administered until disease
progression or unacceptable toxicity or until patients were able to
receive allo-HSCT. For more information about the study, please
visit https://clinicaltrials.gov/ct2/show/NCT03011372.
About Pemigatinib
Pemazyre is a kinase inhibitor indicated in the United States
for the treatment of adults with previously treated, unresectable
locally advanced or metastatic cholangiocarcinoma with a fibroblast
growth factor receptor 2 (FGFR2) fusion or other rearrangement as
detected by an FDA-approved test2. This indication is approved
under accelerated approval based on overall response rate and
duration of response. Continued approval for this indication may be
contingent upon verification and description of clinical benefit in
a confirmatory trial(s).
Pemazyre is also the first targeted treatment approved for use
in the United States for treatment of adults with relapsed or
refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1
rearrangement.
In Japan, Pemazyre is approved for the treatment of patients
with unresectable biliary tract cancer (BTC) with a fibroblast
growth factor receptor 2 (FGFR2) fusion gene, worsening after
cancer chemotherapy and the treatment of myeloid/lymphoid neoplasms
(MLNs) with FGFR1 rearrangement.
In Europe, Pemazyre is approved 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.
Pemazyre is a potent, selective, oral inhibitor of FGFR isoforms
1, 2 and 3 which, in preclinical studies, has demonstrated
selective pharmacologic activity against cancer cells with FGFR
alterations.
Pemazyre is marketed by Incyte in the United States, Europe and
Japan.
Incyte has granted Innovent Biologics, Inc. rights to develop
and commercialize pemigatinib in hematology and oncology in
Mainland China, Hong Kong, Macau and Taiwan. Incyte has retained
all other rights to develop and commercialize pemigatinib outside
of the United States.
Pemazyre is a trademark of Incyte Corporation.
About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical
company focused on finding solutions for serious unmet medical
needs through the discovery, development and commercialization of
proprietary therapeutics. For additional information on Incyte,
please visit Incyte.com and follow @Incyte.
For more information on Incyte Biosciences Japan G.K., please
visit Incyte.jp.
Forward-Looking Statements
Except for the historical information set forth herein, the
matters set forth in this press release, including whether Pemazyre
might provide a successful treatment option for patients with
myeloid/lymphoid neoplasms with FGFR1 rearrangement, , contain
predictions, estimates and other forward-looking statements.
These forward-looking statements are based on Incyte’s current
expectations and subject to risks and uncertainties that may cause
actual results to differ materially, including unanticipated
developments in and risks related to: unanticipated delays; further
research and development and the results of clinical trials
possibly being unsuccessful or insufficient to meet applicable
regulatory standards or warrant continued development; the ability
to enroll sufficient numbers of subjects in clinical trials;
determinations made by the FDA, MHLW and other regulatory
authorities; Incyte’s dependence on its relationships with its
collaboration partners; the efficacy or safety of Incyte’s products
and the products of Incyte’s collaboration partners; the acceptance
of Incyte’s products and the products of Incyte’s collaboration
partners in the marketplace; market competition; sales, marketing,
manufacturing and distribution requirements; greater than expected
expenses; expenses relating to litigation or strategic activities;
and other risks detailed from time to time in Incyte’s reports
filed with the Securities and Exchange Commission, including its
annual report for the year ending December 31, 2022. Incyte
disclaims any intent or obligation to update these forward-looking
statements.
Disclaimer
The drug information contained herein is intended for the
disclosure of Incyte corporate information and is not intended to
advertise or promote any medicinal product, including those under
development.
____________________________ 1 Overview of Orphan Drug/Medical
Device Designation System. Ministry of Health, Labour and Welfare.
Available at:
https://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/orphan_drug.html
2 Pemazyre (pemigatinib) [Package Insert]. Wilmington, DE: Incyte;
2020.
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Media Catalina Loveman +1 302 498 6171
cloveman@incyte.com
Investors Christine Chiou +1 302 274 4773
cchiou@incyte.com
Cosmo PR +81 3 5561 2915 Incyte@cosmopr.co.jp
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