FDA Approves Servier's VORANIGO® as First Targeted Therapy for Grade 2 IDH-mutant Glioma

Servier Pharmaceuticals has garnered approval from the U.S. Food and Drug Administration (FDA) for VORANIGO®, a groundbreaking treatment targeting isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) mutations. This medication is designated for both adult and pediatric patients aged 12 and above, who have Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery (biopsy, sub-total resection, or gross total resection). VORANIGO is presented as a convenient daily pill, offering glioma patients a more active approach to managing their condition.

Gliomas, a form of brain cancer, can significantly disrupt normal brain functions and contribute to a wide range of symptoms. Diffuse gliomas with IDH mutations are the most common malignant primary brain tumors diagnosed in adults under 50. Current therapies are unable to cure these tumors, which, if untreated, continue growing and infiltrate normal brain tissue.

Arjun H. Prasad, Chief Commercial Officer of Servier Pharmaceuticals, hailed the FDA’s approval of VORANIGO as a transformative milestone in cancer care, particularly for those afflicted with Grade 2 IDH-mutant glioma. Described as the first significant breakthrough in this disease area in nearly 25 years, VORANIGO promises substantial improvement in progression-free survival for patients. Prasad underscored Servier's commitment to delivering innovative, targeted therapies to cancer patients.

In normal human cells, the isocitrate dehydrogenase (IDH) family of genes plays a crucial role in breaking down nutrients to generate energy. Mutations in IDH1 and IDH2 are connected to various cancers, wherein they disrupt cellular differentiation, leading cells to grow uncontrollably. VORANIGO functions by mitigating the activity of these mutant IDH enzymes, aiding in disease control.

Ralph DeVitto, President & CEO of the American Brain Tumor Association, emphasized the impact of VORANIGO for patients with Grade 2 IDH-mutant gliomas, who have traditionally faced limited treatment options post-surgery. DeVitto sees this FDA approval as a monumental breakthrough, offering renewed hope for patients and their families managing this relentless disease.

The approval is bolstered by pivotal Phase 3 INDIGO trial results, published in The New England Journal of Medicine and presented during the Plenary Session at the 2023 Annual Meeting of the American Society of Clinical Oncology (ASCO). The trial demonstrated that VORANIGO significantly extended progression-free survival and time to the next intervention compared to a placebo. The study also confirmed that VORANIGO was well tolerated, with a safety profile consistent with earlier Phase 1 studies. Common adverse reactions included fatigue, COVID-19, musculoskeletal pain, diarrhea, and seizures.

David K. Lee, CEO of Servier Pharmaceuticals, highlighted the unique and devastating nature of glioma, particularly for patients in their 30s and 40s who are often in the prime of their lives. He noted that VORANIGO offers hope to these patients and their families. Lee reiterated Servier's commitment to advancing targeted therapies, focusing on identifying and understanding mutations to develop effective treatments.

The INDIGO Phase 3 trial, a global, randomized, double-blind, placebo-controlled study, validated VORANIGO’s efficacy in patients with residual or recurrent Grade 2 glioma with an IDH1/2 mutation who had undergone surgery. The trial’s significant outcomes included a median progression-free survival of 27.7 months for the VORANIGO group compared to 11.1 months for the placebo group. Additionally, the time to the next intervention was considerably extended in the VORANIGO group, emphasizing its clinical benefits.

In summary, VORANIGO presents a significant advancement in the treatment of Grade 2 IDH-mutant glioma, providing a new avenue of hope for patients and emphasizing the importance of targeted therapies in oncology.