Vorasidenib

Vorasidenib (Voranigo) showed strong efficacy with tumor shrinkage, better seizure control, and reduced risk of disease progression in patients with IDH1/2-mutated diffuse glioma, according to updated phase 3 INDIGO study data presented at the 2024 SNO Annual Meeting. The median progression-free survival with vorasidenib was not estimable vs 11.4 months with placebo, indicating a 65% reduction in risk. Vorasidenib also improved time to next intervention and reduced tumor volume compared to placebo, with an acceptable safety profile.

FDA approvals in October 2024 include nivolumab for resectable NSCLC, inavolisib triplet for PIK3CA-mutated HR+/HER2– advanced breast cancer, Optune Lua for metastatic NSCLC, zolbetuximab plus chemotherapy for CLDN18.2+ gastric or GEJ adenocarcinoma, and asciminib for newly diagnosed Ph+ chronic-phase CML. Other approvals include Cologuard Plus for average-risk CRC and Ion Torrent Oncomine Dx Target Test for vorasidenib.

October 2024 episodes of OncLive On Air discussed FDA approvals for durvalumab in resectable NSCLC, tepotinib in MET+ NSCLC, vorasidenib in IDH1/2+ grade 2 astrocytoma/oligodendroglioma, denileukin diftitox in relapsed/refractory CTCL, and updates on myelofibrosis, hepatobiliary and CRC management, liquid biopsy in NSCLC, and quality of life outcomes in CRC.

October 2024 saw significant FDA actions in oncology, including priority reviews for T-DXd in HER2-low/ultra-low breast cancer, acalabrutinib for frontline mantle cell lymphoma, and approvals for nivolumab plus chemotherapy in operable NSCLC, Cologuard Plus for CRC screening, and zolbetuximab for HER2-negative gastric cancer. Other notable actions included fast track designations, orphan drug designations, and expanded approvals for various cancer treatments.

Agios Pharmaceuticals reported $9 million in net Pyrukynd revenue for Q3 2024, a 22% increase from Q3 2023, with $1.7 billion in cash and investments. The company received $1.1 billion in milestone payments following FDA approval of vorasidenib. R&D expenses decreased to $72.5 million, while SG&A expenses increased to $38.5 million. 211 patients completed Pyrukynd enrollment, with 127 net patients on therapy. Positive Phase 3 data for Mitapivat in Thalassemia and PKD were reported, and the company completed enrollment for the Phase 3 rise up study for sickle cell disease. Agios received FDA orphan drug designation for Tebapivat for MDS. The Phase 3 activate study of Mitapivat in children with PK deficiency did not meet the primary endpoint. The company faces challenges in the regulatory environment for sickle cell disease treatments and educating payers about Mitapivat for thalassemia. Agios plans to prioritize potential launches for Mitapivat in thalassemia and sickle cell disease, advancing its pipeline and expanding it internally and externally.

The FDA approved Thermo Fisher’s Ion Torrent Oncomine Dx Target Test to identify patients eligible for vorasidenib (Voranigo) treatment, following Servier Pharmaceuticals’ approval of vorasidenib for grade 2 astrocytoma or oligodendroglioma with IDH1/2 mutations. Vorasidenib is the first targeted therapy for IDH-mutant glioma, with a 61% reduction in disease progression or death risk in the INDIGO trial. The Oncomine Dx Target Test is also used for other cancers, and Thermo Fisher and Servier plan to develop more diagnostics.

The Ion Torrent Oncomine Dx Target Test is approved as a companion diagnostic for vorasidenib, targeting IDH-mutant diffuse glioma. This test provides biomarker results for multiple therapies from a single sample, aiding in timely patient treatment matching. Vorasidenib, an IDH1/IDH2 inhibitor, is FDA-approved for IDH-mutant grade 2 glioma in patients 12 and older.

The FDA approved Thermo Fisher’s Ion Torrent Oncomine Dx Target Test as a companion diagnostic for vorasidenib (Voranigo) tablets, identifying patients eligible for treatment of grade 2 astrocytoma or oligodendroglioma with IDH1/2 mutations. Vorasidenib, approved by the FDA in August 2024, is the first targeted therapy for this type of brain cancer, with the INDIGO trial showing a 61% reduction in disease progression or death risk compared to placebo. The Oncomine Dx Target Test is also used for other cancers, and Thermo Fisher and Servier Pharmaceuticals plan to develop more companion diagnostics.

The FDA approved Thermo Fisher Scientific's Ion Torrent Oncomine Dx Target Test as a companion diagnostic for vorasidenib, an IDH1/IDH2 inhibitor for grade 2 astrocytoma or oligodendroglioma. This marks the first targeted therapy for grade 2 IDH mutant glioma, addressing an unmet need in brain cancer treatment.

FDA approved Vorasidenib, an IDH inhibitor, for treating IDH mutant gliomas, showing a 27-month delay in tumor progression and fewer side effects compared to standard treatments.