European Medicines Agency Accepts Deciphera’s Marketing Authorization Application for Vimseltinib for Treatment of Patients with Tenosynovial Giant Cell Tumor (TGCT)
2024年7月19日 - 9:00AM
ビジネスワイヤ(英語)
- Application based on results from the MOTION
Phase 3 study, in which vimseltinib demonstrated statistically
significant and clinically meaningful improvement in objective
response rate compared to placebo -
Ono Pharmaceutical, Co., Ltd. (Headquarters: Osaka, Japan;
President: Toichi Takino; “Ono”) today announced that the European
Medicines Agency (EMA) has accepted the marketing authorization
application (MAA) for vimseltinib, a colony stimulating factor 1
receptor (CSF1R), for the treatment of patients with tenosynovial
giant cell tumor (TGCT), which is under development by Deciphera
Pharmaceuticals, Inc. (“Deciphera”), a wholly-owned subsidiary of
Ono. The review of the MAA begins under the EMA’s centralized
review process for all 27 member states of the European Union (EU),
as well as Iceland, Liechtenstein and Norway. Vimseltinib was
granted Orphan Drug Designation for the treatment of TGCT by the
EMA in December 2019.
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“Building upon positive results from the MOTION pivotal Phase 3
study, we are excited to initiate the regulatory review process in
the EU and we are one step closer in our mission to bring
vimseltinib to TGCT patients in need of an effective and
well-tolerated treatment,” said Steve Hoerter, President and Chief
Executive Officer of Deciphera Pharmaceuticals.
The submission is supported by the data from the pivotal Phase 3
MOTION study, evaluating the efficacy and safety of vimseltinib in
patients with TGCT not amenable to surgery with no prior
anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib
allowed), compared to placebo. In the study, vimseltinib
demonstrated a statistically significant and clinically meaningful
objective response rate (ORR) at Week 25 in the intent-to-treat
(ITT) population, as assessed by Blinded Independent Radiologic
Review (BIRR) per Response Evaluation Criteria in Solid Tumors
version 1.1 (RECIST v1.1), versus placebo (40% in vimseltinib arm
vs 0% in placebo arm, p <0.0001). Additionally, vimseltinib
demonstrated statistically significant and clinically meaningful
improvements versus placebo in all key secondary endpoints. The
safety profile of vimseltinib is manageable and safety data from
MOTION are consistent with data previously disclosed in the Phase
1/2 clinical trial of vimseltinib*. Data from the MOTION study was
presented at the 2024 American Society of Clinical Oncology (ASCO)
Annual Meeting.
*: Gelberblom, et at. 2024 ASCO Annual Meeting
About the MOTION Study The
MOTION study is a two-part, randomized, double-blind,
placebo-controlled Phase 3 clinical study to assess the efficacy
and safety of vimseltinib in patients with TGCT not amenable to
surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with
imatinib or nilotinib allowed). The primary endpoint of the study
is an objective response rate (ORR) at Week 25 in the
intent-to-treat (ITT) population, as assessed by Blinded
Independent Radiologic Review (BIRR) per using Response Evaluation
Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo.
The secondary endpoint includes ORR per tumor volume score (TVS),
active range of motion (ROM), physical function, stiffness, quality
of life, and pain, all assessed at Week 25. This study consists of
two Parts. In Part 1, patients were randomized to receive either
vimseltinib or placebo for 24 weeks. In Part 2, patients randomized
to placebo in Part 1 have the option to receive vimseltinib, and
all patients receive vimseltinib for a long-term period in an
open-label setting.
About Tenosynovial Giant Cell Tumor
(TGCT) TGCT is a rare disease caused by a translocation
in colony-stimulating factor 1 (CSF1) gene resulting in
overexpression of CSF1 and recruitment of colony-stimulating factor
1 receptor (CSF1R)-positive inflammatory cells into the lesion.
TGCT is a rare, non-malignant tumor that develops inside or near
joints. TGCT is caused by dysregulation of the CSF1 gene leading to
overproduction of CSF1. TGCT is also known as giant cell tumor of
the tendon sheath (GCT-TS) or pigmented villonodular synovitis
(PVNS), a diffuse-type of TGCT. Although benign, these tumors can
grow and cause damage to surrounding tissues and structures
inducing pain, swelling, and limitation of movement of the joint.
Surgery is the main treatment option; however, these tumors tend to
recur, particularly in diffuse-type TGCT. If untreated or if the
tumor continually recurs, damage and degeneration may occur in the
affected joint and surrounding tissues, which may cause significant
disability. For a subset of patients who are not amenable to
surgery, systemic treatment options are limited and a new
therapeutic option for TGCT is needed.
About Vimseltinib
Vimseltinib is an investigational, oral switch-control tyrosine
kinase inhibitor specifically designed to selectively and potently
inhibit CSF1R. Vimseltinib has been developed using Deciphera’s
proprietary switch-control kinase inhibitor platform.
About Deciphera Pharmaceuticals
Inc. (As of June 11, 2024, Deciphera is now a member
of ONO Pharma.) Deciphera is a biopharmaceutical company
focused on discovering, developing, and commercializing important
new medicines to improve the lives of people with cancer. We are
leveraging our proprietary switch-control kinase inhibitor platform
and deep expertise in kinase biology to develop a broad portfolio
of innovative medicines. In addition to advancing multiple product
candidates from our platform in clinical studies, QINLOCK® is
Deciphera’s switch-control inhibitor approved for the treatment of
fourth-line gastrointestinal stromal tumor (GIST). QINLOCK is
approved in many countries including the European Union and the
United States. For more information, visit www.deciphera.com and
follow us on LinkedIn and Twitter (@Deciphera).
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Ono Pharmaceutical Co., Ltd. Corporate Communications
public_relations@ono-pharma.com
Deciphera Pharmaceuticals, Inc. Investor Relations:
Maghan Meyers Argot Partners Deciphera@argotpartners.com
212-600-1902
Media: David Rosen Argot Partners david.rosen@argotpartners.com
646-461-6387