Cerulean Cataract

LYTGOBI is the first irreversibly binding FGFR inhibitor in the European Union for use in the treatment of patients with cholangiocarcinoma ZUG, Switzerland, July 4, 2023 /PRNewswire/ -- Taiho Oncology Europe GmbH and Taiho Pharmaceutical Co., Ltd., announced today that the European Commission has granted conditional marketing authorization for LYTGOBI® (futibatinib) monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. CCA is an aggressive cancer of the bile ducts of the liver. While rare – in Europe, approximately 6.000-8.000 people are diagnosed with CCA1 – this disease is associated with poor outcomes and is growing in prevalence worldwide2, underscoring the need for new treatment options. "Today is an important day for current and future patients with CCA as well as the healthcare providers who treat them," said Peter Foertig, MD, Vice President, Medical Affairs, Taiho Oncology Europe. "LYTGOBI is an oral molecularly targeted medication that may provide clinically meaningful outcomes for patients undergoing treatment for CCA." Added John Bridgewater, MD, PhD, Investigator and Senior Author of the recently published FOENIX*-CCA2 pivotal trial in the New England Journal of Medicine: "FGFR2 fusions/rearrangements are one of the most frequent actionable alterations in CCA. As an irreversibly binding FGFR inhibitor, LYTGOBI targets FGFR in a unique way and offers new hope in a disease that, for me, has been among the most challenging to treat in my career." Professor Bridgewater is a Clinical Researcher and Medical Oncologist at University College London Cancer Institute and University College London Hospitals NHS Foundation Trust. The European Commission's conditional marketing authorization for LYTGOBI is based on data from the aforementioned FOENIX-CCA2 trial, a global open label trial evaluating 103 patients with unresectable, locally advanced or metastatic intrahepatic (inside the bile ducts of the liver) CCA harboring FGFR2 gene rearrangements, including fusions. In this trial, patients received LYTGOBI orally once daily at a dose of 20mg until disease progression or unacceptable toxicity. Within Europe, patients were enrolled from France, Germany, Italy, the Netherlands, Spain and the United Kingdom. Results of FOENIX-CCA2 trial showed: LYTGOBI demonstrated an objective response rate (ORR) of 42% as assessed by independent review (primary endpoint met).3 LYTGOBI demonstrated a median duration of response (DOR) – a key secondary endpoint – of 9.7 months. A total of 72% of the responses lasted >6 months.3 "I believe that LYTGOBI may be part of a new era in the treatment of CCA, one in which the power of personalised medicine may touch the lives of patients in ways we haven't seen before with traditional chemotherapy," said Helen Morement, CEO of the AAMF – The Cholangiocarcinoma Charity and the UK's only charity dedicated to this cause. "We thank the many patients and healthcare professionals who participated in the FOENIX-CCA2 trial," said Atsushi Azuma, Managing Director of Taiho Pharmaceutical and Chairman of Taiho Oncology Europe. "Patients with CCA are often diagnosed at an advanced stage when surgery is not an option. We are pleased that LYTGOBI now will be a new treatment option for patients with CCA." A conditional marketing authorization in Europe is granted for medicines that fulfill an unmet medical need to treat serious diseases, and the benefits of having them available earlier outweighs any risks associated with using the medicines while waiting for further evidence. Under the specific obligation to complete post-authorization measures for the conditional marketing authorization, Taiho has until October 2027 to provide additional clinical data on LYTGOBI. Safety Information From the EU Summary of Product Characteristics (SmPC)3 LYTGOBI may cause serious adverse reactions. The most common serious adverse reactions were intestinal obstruction and migraine. The most common adverse reactions were hyperphosphatemia, nail disorders, constipation, alopecia, diarrhoea, dry mouth, fatigue, nausea, dry skin, increased AST, abdominal pain, stomatitis, vomiting, palmar-plantar erythrodysaesthesia syndrome, arthralgia, and decreased appetite. Prolonged hyperphosphatemia may cause soft tissue mineralization, including cutaneous calcification, vascular calcification, and myocardial calcification, anaemia, hyperparathyroidism, and hypocalcemia that may cause muscle cramps, QT interval prolongation, and arrythmias. Hyperphosphatemia is a pharmacodynamic effect expected with LYTGOBI administration. Recommendations for management of hyperphosphatemia include dietary phosphate restriction, administration of phosphate-lowering therapy, and dose modification when required. LYTGOBI can cause serous retinal detachment, which may present with symptoms such as blurred vision, visual floaters or photopsia. Ophthalmological examination should be performed prior to initiation of therapy, 6 weeks thereafter, and urgently at any time for visual symptoms. For serous retinal detachment reactions, the dose modification guidelines should be followed. LYTGOBI should not be used during pregnancy unless the clinical condition of the women requires treatment with LYTGOBI. About Taiho Oncology Europe The mission of Taiho Oncology Europe is to improve the lives of patients with cancer, their families, and their caregivers. The company specializes in orally administered anti-cancer agents and has a growing pipeline of selectively targeted anti-cancer agents. Taiho Oncology Europe GmbH (Zug, Switzerland) is the European subsidiary of Taiho Pharmaceutical Co., Ltd. (Tokyo, Japan). For more information, visit About Taiho Pharmaceutical Co., Ltd. Taiho Pharmaceutical Co., Ltd., a subsidiary of Otsuka Holdings Co., Ltd., is an R&D-driven specialty pharma company with a focus on oncology. Taiho Pharmaceutical also has development programs in allergy and immunology, urology, and consumer healthcare products. Our corporate philosophy takes the form of a pledge: "We strive to improve human health and contribute to a society enriched by smiles." For more information about Taiho Pharmaceutical Co., Ltd., please visit: LYTGOBI is a registered trademark of Taiho Pharmaceutical Co., Ltd. *The FOENIX-CCA2 trial is a Phase 1 / 2 Study of TAS-120 in Patients With Advanced Solid Tumors Harboring FGF/FGFR Aberrations: FGFR Oral SElective Novel Inhibitor X [across] tumors Taiho Oncology Europe Media Contact: Judy Kay Moore +1 574-526-2369 [email protected] Taiho Pharmaceutical Media Contact: Junko Onishi +81-3-34293-2878 [email protected] LYT-PM-EU-0004 06/23 References: 1 Kirstein MM, Vogel A. Epidemiology and risk factors of cholangiocarcinoma. Visc Med. 2016;32(6):395-400. Available at: . Last accessed: May 2023. 2 Banales JM, Marin JJG., Lamarca A. et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020;17:557–588. Available at : . Last accessed: May 2023. 3 Taiho Oncology Europe GmbH. LYTGOBI® (futibatinib): Summary of Product Characteristics. 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ZUG, Switzerland, April 27, 2023 /PRNewswire/ -- Taiho Oncology Europe GmbH and Taiho Pharmaceutical Co., Ltd. announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorization (CMA) of futibatinib for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. The CHMP's opinion to recommend the use of futibatinib is now being reviewed by the full EMA. Final marketing authorizations for medical products in the European Union (EU) rest with the European Commission. If approved, futibatinib will be the first irreversibly binding FGFR-inhibitor in the EU for use in the treatment of patients with CCA and will be commercialized under the brand name LYTGOBI® (pronounced "light-GOH-bee"). Each year, approximately 6.000-8.000 individuals in Europe are diagnosed with CCA1, a rare cancer of the bile ducts of the liver, and approximately 0,3-6 people per 100.000 individuals live with CCA worldwide2. "CCA is an aggressive cancer, and the current five-year survival rate is very poor, which underscores the need for new treatment options," said Peter Foertig, MD, Vice President, Medical Affairs, Taiho Oncology Europe. "We look forward to working with the European Medicines Agency as it reviews the application for marketing authorization." The positive CHMP opinion on futibatinib is based on data from the pivotal Phase 2 FOENIX*-CCA2 trial in 103 patients with locally advanced or metastatic unresectable intrahepatic (inside the liver) CCA, harboring FGFR2 fusions or rearrangements who had received one or more prior lines of systemic therapy. Patients in the trial received futibatinib 20 mg once daily until disease progression or unacceptable toxicity. The trial's primary endpoint was an objective response rate (ORR), which was 42% as assessed by independent central review. The key secondary endpoint of duration of response (DOR) demonstrated a median of 9.7 months (72% of responses ≥6 months). The most common treatment-related adverse events in the trial were hyperphosphatemia (85%), alopecia (33%), dry mouth (30%), diarrhea (28%), dry skin (27%) and fatigue (25%). Results from the trial were published in the January 19, 2023, issue of the New England Journal of Medicine3. "As someone who treats patients with CCA, I am encouraged by these data and the positive news from the CHMP," said Professor Arndt Vogel, MD, Senior Consultant, Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Germany. "The study findings underscore the importance of molecular profiling for patients with CCA and other diseases whenever possible to potentially help improve outcomes. Indeed, the ESMO Clinical Practice Guidelines4 recommend FGFR inhibitors for the treatment of patients with CCA and FGFR2 fusions who have progressed after one or more prior lines of systemic therapy." Takeshi Sagara, PhD, Managing Director, Clinical Development and Medical Affairs, Discovery and Preclinical Research at Taiho Pharmaceutical, added, "The global Taiho group will continue its efforts to bring futibatinib as a new treatment option to patients with CCA around the world, and the positive CHMP opinion represents a big step for Taiho in Europe to deliver an innovative drug to help patients with CCA in their journey." About Futibatinib Futibatinib (TAS-120) is an oral, potent, selective, and irreversible tyrosine kinase inhibitor of FGFR1, 2, 3 and 4 being studied as a potential treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including cholangiocarcinoma, who were previously treated with chemotherapy or other therapies. Futibatinib covalently binds to the ATP binding pocket of FGFR1-4 resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation in tumors with FGFR1-4 genetic aberrations. About Taiho Oncology Europe The mission of Taiho Oncology Europe is to improve the lives of patients with cancer, their families, and their caregivers. The company specializes in orally administered anti-cancer agents and has a growing pipeline of selectively targeted anti-cancer agents. Taiho Oncology Europe GmbH (Zug, Switzerland) is the European subsidiary of Taiho Pharmaceutical Co., Ltd. (Tokyo, Japan). For more information, visit About Taiho Pharmaceutical Co., Ltd. Taiho Pharmaceutical Co., Ltd., a subsidiary of Otsuka Holdings Co., Ltd., is an R&D-driven specialty pharma company with a focus on oncology. Taiho Pharmaceutical also has development programs in allergy and immunology, urology, and consumer healthcare products. Our corporate philosophy takes the form of a pledge: "We strive to improve human health and contribute to a society enriched by smiles." For more information about Taiho Pharmaceutical Co., Ltd., please visit: *The FOENIX-CCA2 trial is a Phase 1 / 2 Study of TAS-120 in Patients With Advanced Solid Tumors Harboring FGF/FGFR Aberrations: FGFR Oral S Elective Novel Inhibitor X [across] tumors Taiho Oncology Europe Media Contact: Judy Kay Moore 574-526-2369 [email protected] Taiho Pharmaceutical Media Contact: Junko Onishi +83-3-3493-2878 [email protected] References: 1 Kirstein MM, Vogel A. Epidemiology and Risk Factors of Cholangiocarcinoma. Visc Med. 2016 Dec;32(6):395-400. Erratum in: Visc Med. 2017 Jun;33(3):226. 2 Banales, J M, Marin, J JG, Lamarca, A, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nature Reviews Gastroenterology & Hepatology. 17: 557–588 (2020). 3 Goyal L, Meric-Bernstam F, Hollebecque A, et al. FOENIX-CCA2 Study Investigators. Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma. N Engl J Med. 2023 Jan 19;388(3):228-239 4Vogel A, Bridgewater J, Edeline J, et al. ESMO Guidelines Committee. Biliary tract cancer: ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up. Ann Oncol. 2023 Feb;34(2):127-140. SOURCE Taiho Oncology Europe GmbH