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Servier
Receives Regulatory Filing Acceptances from FDA and
EMA
for
Vorasidenib
in the Treatment of
IDH-Mutant Diffuse Glioma
2024-02-21
·
BioSpace
临床结果
临床3期
优先审批
ASCO会议
突破性疗法
[21-February-2024] If approved,
vorasidenib
would be the first targeted therapy in
IDH-mutant diffuse glioma
, a malignant and incurable
brain tumor
In clinical studies,
vorasidenib
has demonstrated strong blood-brain barrier penetrance alongside clinically meaningful and statistically significant improvements in progression-free survival and time-to-next intervention Approval of
vorasidenib
would mark
Servier
's sixth approval for a first-in-class treatment option in
IDH-mutant cancers
BOSTON and SURESNES, France, Feb. 21, 2024 /PRNewswire/ --
Servier
, a global leader in oncology focused on delivering meaningful therapeutic progress for the patients it serves, today announced the FDA filing acceptance and priority review for a New Drug Application (NDA) for vorasidenib, as well as the
EMA
granting accelerated assessment for the vorasidenib Marketing Authorization Application (MAA). This innovative targeted therapy is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes for the treatment of
IDH-mutant diffuse glioma
. If approved,
vorasidenib
would become a first-in-class targeted therapy for patients with
IDH-mutant gliomas
and would mark
Servier
's sixth approval across
IDH-mutant cancers
. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of August 20, 2024, while the European Commission approval is anticipated in the second half of 2024. "In the realm of
glioma
treatment, innovation has been stagnant for nearly a quarter-century, posing challenges for patients who, post-surgery, may opt to defer treatment due to concerns around potential toxic side effects. As a drug specifically designed to be highly blood-brain barrier penetrant,
vorasidenib
has demonstrated clinically meaningful efficacy in patients with
IDH1/2 mutant gliomas
alongside a consistently manageable safety profile," said Susan Pandya, M.D., Head of
Cancer
Metabolism Global Development Oncology & Immuno-Oncology,
Servier
. "This promising outcome brings hope to patients grappling with IDH-
mutant diffuse gliomas
, offering a potential breakthrough for those eagerly awaiting a new therapeutic option." Adult-type
diffuse gliomas
represent approximately 81% of
primary malignant brain tumors
. Of those, approximately 20% harbor an IDH mutation, including 100% of grade 2 and grade 3 adult-type diffuse gliomas, as well as a much smaller portion of the grade 4 tumors.1,2 Testing for IDH mutations is essential for the accurate diagnosis of
adult-type diffuse gliomas
and can offer more information on the pathogenesis and prognosis of the disease.3 The 2021 WHO Classification includes disease defining histologic and molecular features, including IDH mutation status, to diagnose adult-type
diffuse gliomas.4
Additionally, the National Clinical Practice Guidelines in Oncology (NCCN Guidelines) recommend IDH mutation testing in all patients with
glioma
, noting IDH mutation status impacts diagnosis, prognosis and treatment recommendations.5 "As a dedicated pioneer in the field of mutant IDH inhibition,
Servier
has consistently spearheaded the development of cutting-edge treatment options for various
cancer
types characterized by IDH mutations. The compelling efficacy results observed with
vorasidenib
in the INDIGO study underscore its full potential to emerge as the benchmark treatment for patients grappling with
IDH-mutant diffuse glioma
harboring IDH1/2 mutations," stated Claude Bertrand, Executive Vice-President of Research & Development and Chief Scientific Officer at
Servier
. "The submission of global regulatory filings for
vorasidenib
serve as validation of
Servier
's global oncology commitment while marking a possibly significant milestone for patients who have endured more than two decades without access to new therapeutic solutions." The submissions are based on results from the pivotal Phase 3 INDIGO clinical trial, which met its primary endpoint of progression-free survival (PFS) per blinded independent review committee (BIRC) and key secondary endpoint of time to next intervention (TTNI) at the prespecified second interim analysis. The primary endpoint, PFS per BIRC, was statistically significant and clinically meaningful in favor of the
vorasidenib
arm (Hazard Ratio [HR], 0.39; 95% Confidence Interval [CI], 0.27 to 0.56; 1-sided P=0.000000067), median PFS for
vorasidenib
and placebo was 27.7 and 11.1 months, respectively. TTNI was also statistically significant (HR, 0.26; 95% CI, 0.15 to 0.43; 1-sided P=0.000000019). Median TTNI was not reached for
vorasidenib
and was 17.8 months for placebo.
Vorasidenib
was also shown to reduce the
tumor
volume by a mean of 2.5% (TGR of –2.5%; 95% CI: -4.7% to -0.2%) every 6 months, while
tumor
volume increased by a mean of 13.9% (TGR of 13.9%; 95% CI: 11.1% to 16.8%) every 6 months for patients randomized to the placebo arm, as measured by a blinded independent radiology committee. The INDIGO study showed that
vorasidenib
was well-tolerated, and its safety pro consistent with results from the Phase 1 studies. The results of INDIGO were presented at the 2023 Annual Meeting of the American Society of Clinical Oncology (ASCO) and simultaneously published in The New England Journal of Medicine. The results of additional secondary endpoints, including
vorasidenib
's impact on
tumor
growth rate (TGR) of
IDH-mutant gliomas
, were presented at the 2023 Annual Meeting of the Society for Neuro-Oncology (SNO) among other presentations including results on health-related quality of life, seizure control, neurocognition, and preliminary molecular translational analyses. Priority Review is granted to FDA applications for medicines that, if approved, would provide significant improvements in the effectiveness or safety of the treatment, diagnosis or prevention of serious conditions.6
Vorasidenib
was granted Fast Track Designation by the FDA in February 2023 and Breakthrough Therapy Designation by the FDA in August 2023. The
EMA
's accelerated assessment is granted if the Committee for Medicinal Products for Human Use decides the new medicine is expected to be of major public health interest, particularly from the viewpoint of therapeutic innovation.7
Servier
has also submitted an application for project
Orbis
member countries, including Brazil, Canada, Australia, Israel and Switzerland. In addition,
Servier
plans to submit a Marketing Application in the United Kingdom if a positive CHMP opinion is received. More information about Project
Orbis
can be found on the FDA website. In its ongoing commitment to addressing patient's needs,
Servier
recognizes that patients and their physicians may believe that a patient with a serious or immediately life-threatening disease could benefit from
Servier
's investigational drugs. An Expanded Access Program, also known as "compassionate use," is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials. More information about
Servier
's expanded access program can be found at clinicaltrials.gov. About the INDIGO Phase 3 Trial (NCT04164901) INDIGO was a registration-enabling Phase 3 global, randomized, double-blind placebo-controlled study of
vorasidenib
in patients with
residual or recurrent grade 2 glioma
with an
isocitrate dehydrogenase 1/2 (IDH1/2)
mutation who have undergone surgery as their only treatment. Results were published in The New England Journal of Medicine. About Glioma8 Adult-type
diffuse gliomas
represent approximately 81% of
primary malignant brain tumors
. Of those, approximately 20% harbor an IDH mutation, including 100% of grade 2 and grade 3 adult-type diffuse gliomas, as well as a much smaller portion of the grade 4 tumors.1,2 Establishing the IDH mutation status of these
tumors
is essential to both diagnosis and prognosis, per the 2021 WHO classification of
CNS tumors
and the NCCN treatment guidelines, respectively. As of 2021, adult-type
diffuse gliomas
are sub-divided into only three categories:
Astrocytoma
, IDH-mutant (CNS WHO grades 2-4)
Oligodendroglioma
, IDH-mutant and 1p19q-codeleted (CNS WHO grades 2-3)
Glioblastoma
, IDH-wildtype (CNS WHO grade 4) About Servier in Oncology
Servier
is a global leader in oncology focused on delivering meaningful therapeutic progress for the patients it serves. Governed by a non-profit foundation,
Servier
approaches innovation with a long-term vision, free of influence from investors and outside pressure to chase short-term monetary targets. As a leader in
oncology
,
Servier
has significantly accelerated its investment in difficult and hard-to-treat
cancers
, with more than 50% of its research and development dedicated to delivering significant advances in areas of high unmet need throughout
oncology
with the potential to change the lives of the patients it serves. Within these areas,
Servier
is the leader in mutant IDH inhibition, with the first ever
mutant IDH1 inhibitor
mutant IDH1
inhibitor approved in the U.S. and the European Union, and the company continues to drive the science behind targeted mutant IDH inhibition.
Servier
's commitment to therapeutic progress guides its collaboration strategy. While many companies across the industry are scaling back investments,
Servier
is actively building alliances, completing acquisitions, conducting licensing deals and entering new partnerships that can help to accelerate access to therapies for patients in need. With the company's commercial expertise, global reach, scientific expertise and commitment to clinical excellence,
Servier
is dedicated to bringing the promise of tomorrow to the patients it serves. Press Contacts
Servier Group
(Global) marion.breyer@servier.com
Servier Pharmaceuticals
(U.S.) Nathan Mellor nathan.mellor@servier.com Disclosures This release contains general information about the
Servier Group
and its entities (hereinafter "
Servier
and its Affiliates") and is intended for informational purposes only. The information is thought to be reliable; however,
Servier
and its Affiliates make no representation as to the accuracy or completeness of the information contained herein or otherwise provided and accept no responsibility or liability, in contract, in tort, in negligence, or otherwise, should the information be found to be inaccurate or incomplete in any respect.
Servier
and its Affiliates are not acting as an advisor to the recipient of this information, and the ultimate decision to proceed with any transaction rests solely with the recipient of this information. Therefore, prior to entering into any proposed transaction, the recipient of this information should determine, without reliance upon
Servier
or its Affiliates, the economic risks and merits, as well as the legal, tax, and accounting characterizations and consequences, of the transaction and that it is able to assume these risks. This statement also contains forward-looking statements that are subject to varying levels of uncertainty and risk. Investigational new drugs and indications are subject to further scientific and medical review and regulatory approval. They are not approved for use by the FDA. Any reliance placed on this document is done entirely at the risk of the person placing such reliance. The information contained in this document is neither an offer to sell nor the solicitation of an offer to enter into a transaction. The content of this document is a summary only, is not complete, and does not include all material information about
Servier
and its Affiliates, including potential conflicts of interest. To the maximum extent permitted by applicable laws and regulations,
Servier
and its Affiliates disclaim all representations, warranties, conditions and guarantees, whether express, implied, statutory or of other kind, nor does it accept any duty to any person, in connection with this document. Without prejudice to the generality of the foregoing,
Servier
and its Affiliates do not warrant or represent that the information or opinions contained in this document is accurate or complete. To the maximum extent permitted by applicable laws and regulations,
Servier
and its Affiliates shall not be liable for any loss, damage or expense whatsoever, whether direct or indirect, howsoever arising, whether in contract, tort (including negligence), strict liability or otherwise, for direct, indirect, incidental, consequential, punitive or special damages arising out of or in connection with this document, including (without limitation) any course of action taken on the basis of the same. The estimates, strategies, and views expressed in this document are based upon past or current data and information and are subject to change without notice. 1 Louis, D. et. al (2021). The 2021 WHO Classification of
Tumors of the Central Nervous System
: a summary, Neuro-Oncology, 23(8): 1231–1251. . Accessed February 2024. 2 Ostrom, Q. T., Price, M., Neff, C., Cioffi, G., Waite, K. A., Kruchko, C., & Barnholtz-Sloan, J. S. (2022). CBTRUS Statistical Report: Primary Brain and Other
Central Nervous System Tumors
Diagnosed in the United States in 2015-2019. Neuro-oncology, 24(Suppl 5), v1–v95. . Accessed February 2024. 3 Isocitrate Dehydrogenase. Science Direct. . Accessed February 2024. 4 Antonelli M, Poliani PL. Adult type
diffuse gliomas
in the new 2021 WHO Classification. Pathologica. 2022 Dec;114(6):397-409. doi: 10.32074/1591-951X-823. PMID: 36534419; PMCID: PMC9763975. 5 NCCN. Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for
Brain Cancer Gliomas
2021. ©
National Comprehensive Cancer Network, Inc.
Cancer
Network, Inc. 2023. Accessed February 2024. 6
U.S. Food and Drug Administration (FDA)
. Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review. . Accessed February 2024. 7
European Medicines Agency (EMA)
. Accelerated assessment. . Accessed February 2024. 8 Neuro Oncology. The 2021 WHO Classification of
Tumors of the Central Nervous System
: a summary. . Accessed February 2024.
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[+5]
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IDH1突变胶质瘤
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[+6]
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