U.S. Food and Drug Administration Accepts for Priority Review Deciphera’s New Drug Application for Vimseltinib for the Treatment of Patients with Tenosynovial Giant Cell Tumor (TGCT)
15 August 2024 - 11:00PM
Business Wire
Application based on results from the pivotal
Phase 3 MOTION study in which vimseltinib demonstrated
statistically significant and clinically meaningful objective
response rate in this patient population compared to placebo
The U.S. FDA has assigned a target action date
of February 17, 2025
EMA has accepted Deciphera’s a Marketing
Authorization Application for vimseltinib
Ono Pharmaceutical, Co., Ltd. (Headquarters: Osaka, Japan;
President: Toichi Takino; “Ono”) today announced that the U.S. Food
and Drug Administration (FDA) accepted a priority review for the
New Drug Application (NDA) on August 14 US time 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 FDA assigned a Prescription
Drug User Fee Act (PDUFA) goal date of February 17, 2025. In
mid-July, the European Medicines Agency (EMA) accepted a Marketing
Authorization Application (MAA) of vimseltinib and has begun the
start of the EMA's centralized review process.
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“Building upon positive results from the MOTION pivotal Phase 3
study and following our recent announcement that EMA review of the
vimseltinib MAA has begun, we are excited to initiate the
regulatory review process in the US and we look forward to working
with the FDA to deliver a new treatment option to patients with
TGCT” 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*. Results from the MOTION study
were presented at the 2024 American Society of Clinical Oncology
(ASCO) Annual Meeting and published concurrently in Lancet.
*: Gelberblom, et at. 2024 ASCO Annual Meeting
About 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 became a member of Ono
Pharmaceutical Co., Ltd.)
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 212-600-1902