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Cabometyx, Opdivo combination nabs expanded indication for advanced RCC
Bristol-Myers Sqibb’s Opdivo (nivolumab) and Exelixis’ Cabometyx (cabozantinib) have been approved as a first-line combination therapy for patients with advanced renal cell carcinoma (RCC).
The approval was based on efficacy findings from the CheckMATE 9ER randomized, open-label trial in which 323 patients with previously untreated advanced RCC received Opdivo in combination with Cabometyx and 328 patients received Sutent (sunitinib; Pfizer). Median progression-free survival in the study combination group was 16.6 months, compared with 8.3 months in the control group. Neither arm attained median overall survival, but overall response rate 55.7% and 27.1%, respectively.
The Opdivo review used the real-time oncology review program, and both the Opdivo and Cabometyx applications used the assessment aid. In addition, the Opdivo application was granted fast track review, and both applications were granted priority review.
Opdivo was first approved in 2014 and is used to treat several types of cancers as a single agent or in combination with other drugs. Cabometyx was first approved in 2012 and is used to treat RCC and hepatocellular carcinoma.
Carbaglu approved as first treatment for acute hyperammonemia in children and adults
Recordati Rare Diseases’ Carbaglu (carglumic acid tablets) has been given a new indication as an add-on to standard care for children and adults with acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA). It is the only approved therapy for this indication.
PA and MMA are rare genetic metabolic disorders resulting in the accumulation of toxic metabolites that can cause acute hyperammonemia, which is manifested by elevated levels of ammonia in the body.
Approval of the expanded indication for Carbaglu was based on efficacy findings in a randomized, double-blind, placebo-controlled trial. The reduction of ammonia was quicker in patients receiving the study drug than in those receiving placebo. In addition, in the first 3 days of treatment, more patients in the study group returned to a blood ammonia level of ˂50 micromol/L or were discharged from hospital compared with patients receiving placebo.
Carbaglu was first approved in 2010, and is also used for the treatment of acute and chronic hyperammonemia caused by N-acetylglutamate synthase deficiency.
Tags: FDA, US