«Исследования рака у детей жизненно важны для открытия новых методов лечения для населения», — сказал доктор Эрик Буффе, доктор медицинских наук, FRCPC, главный исследователь клинических испытаний головастика и почетный научный сотрудник больницы для больных детей (SickKids). «Разработка целевых методов лечения, основанных на уникальных генетических особенностях опухоли пациента, — это будущее педиатрической онкологической помощи».
Это одобрение FDA комбинации Тафинлар + Мекинист основано на результатах исследования Фазы II/III TADPOLE (NCT02684058), которое показало, что у пациентов, рандомизированных для получения Тафинлара + Мекинист, наблюдалось статистически значимое улучшение общей частоты ответа (ЧОО) на 47% (ДИ: 35). -59%) по сравнению с 11% (ДИ: 3-25%) для тех, кто был рандомизирован для получения химиотерапии. При медиане наблюдения 18,9 мес медиана выживаемости без прогрессирования заболевания (ВБП) составила 20,1 мес при применении тафинлара + мекиниста (ДИ: 12,8 мес — оценка невозможна) по сравнению с 7,4 мес при химиотерапии (ДИ: 3,6–11,8 мес, отношение рисков). =0,31 [CI: 0.17-0.55] [p
“It is more important than ever to test for genetic mutations in patients living with low-grade glioma. This FDA approval may offer new hope to pediatric patients living with BRAF V600E low-grade glioma,” said Dr. Roger Packer, senior vice president of the Center for Neurosciences and Behavioral Medicine at Children’s National Hospital. “This has the potential to change the way healthcare providers treat these pediatric patients, offering a significant advancement compared to chemotherapy.”
The safety profile of Tafinlar + Mekinist observed in this study was consistent with the known safety profile in other approved indications. The most common adverse reactions (>=15%) were pyrexia (68%), rash (51%), headache (47%), vomiting (34%), musculoskeletal pain (34%), fatigue (33%), diarrhea (29%), dry skin (26%), nausea (25%), hemorrhage (25%), abdominal pain (25%), dermatitis acneiform (22%), dizziness (15%), upper respiratory tract infection (15%) and weight increased (15%). These data were highlighted as part of an official press briefing and oral presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.
“This new indication for Tafinlar + Mekinist is a potential new standard of care treatment option for young patients with this form of brain cancer with a BRAF V600E mutation, in formulations specifically designed for them,” said Reshema Kemps-Polanco, Executive Vice President, US Oncology at Novartis. “We are thankful for the families, including children and adolescents, that participated in the clinical trial that led to this approval and whose bravery has led to a new hope for children living with this serious brain cancer.”
LGG is the most common pediatric brain cancer. BRAF V600 mutations are present in 15-20% of pediatric LGGs and are associated with poor survival outcomes and less favorable response to chemotherapy(4). BRAF mutations have been identified as drivers of cancer growth across a wide range of solid tumors, and often have limited treatment options(4,5).
Full prescribing information for Tafinlar + Mekinist can be found at and
About Tafinlar + Mekinist
The combination of Tafinlar + Mekinist, the worldwide targeted therapy leader in BRAF/MEK-inhibition research and patients reached, may help to slow tumor growth by blocking signals associated with the BRAF and MEK kinases that are implicated in the growth of various types of cancer(1,2,4,5). Tafinlar + Mekinist has been studied in more than 6,000 BRAF-positive patients in more than 20 ongoing and completed trials, including in pediatric patients 1 year of age and older, and has been prescribed to more than 200,000 patients worldwide(6).
This FDA approval is the sixth for Tafinlar + Mekinist, which is indicated across multiple BRAF V600 solid tumors, including melanoma, thyroid cancer and lung cancer(1,2).
About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. We deliver high-value medicines that alleviate society’s greatest disease burdens through technology leadership in R&D and novel access approaches. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. About 106,000 people of more than 140 nationalities work together to bring Novartis products to nearly 800 million people around the world.
1. Mekinist [prescribing information]. Восточный Ганновер, Нью-Джерси: Novartis Pharmaceuticals Corp.; 2022.
2. Тафины [prescribing information]. Восточный Ганновер, Нью-Джерси: Novartis Pharmaceuticals Corp.; 2022.
3. Лассалетта А. и соавт. Дж. Клин Онкол. 2017;35:2934-2941
4. Турский М.Л. и соавт. Мол Рак Тер. 2016;15:533-547
5. Пратилас С. и соавт. Курр Топ Микробиол Иммунол. 2012;355:82-98
6. Данные в файле.
