FDA Approvals Roundup: Cabenuva, Lupkynis, Verquvo

Regulatory NewsRegulatory News | 27 January 2021 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).

New approvals
Cabenuva  approved as first extended-release, injectable regimen for adults with HIV
ViiV Healthcare’s Cabenuva (cabotegravir and rilpivirine injection) has been approved as a long-acting, once-monthly regimen for patients HIV-1 infection.
The extended-release regimen is indicated for patients with virologic suppression who are on a stable antiretroviral regimen and have no history of treatment failure or resistance to the individual combination drugs.
The agency also approved Vocabria (cabotegravir tablets) for patients to take in combination with Edurant (oral rilpivirine) for a month before switching to Cabenuva to ensure their toleration of the individual medications.
Cabenuva’s approval was based on safety and efficacy findings in two randomized, open-label, controlled clinical trials (ATLAS and FLAIR) in 1,182 HIV-infected adults who were virologically suppressed. Patients in both trials showed continued suppression at the end of the 48-week study period and showed no relevant change from baseline in CD4+ cell counts.
FDA granted Cabenuva and Vocabria fast track and priority review designations.
Lupkynis gets the nod for adults with lupus nephritis
Aurinia’s Lupkynis (voclosporin), combined with immunosuppressive therapy, has been approved for treating active lupus nephritis in adults. It is the first approved oral therapy for the condition, which causes irreversible damage to the kidneys.
The approval was based on findings in the year-long, randomized, double-blind, placebo-controlled AURORA study of 357 patients randomized 1:1 to receive Lupkynis plus standard care or standard care alone. Patients receiving Lupkynis were more than twice as likely to achieve complete renal response by week 52, compared with patients receiving standard care alone (40.8% vs. 22.5%, respectively). Study group patients also showed a similar decline rate in urine protein-creatinine ratio over the same period, compared with the standard care patients (45.3% vs. 23.0%).
“The FDA approval of Lupkynis allows us to treat patients safely and more effectively with a rapid acting therapy which requires far less steroids,” said Brad H. Rovin, MD, professor of medicine and the director of the division of nephrology, Ohio State University Wexler Medical Center, and a clinical trial investigator in the AURORA trial.
The application was granted priority review and fast track designations.
Verquvo okayed to cut CVD, hospitalization risks in patients with heart failure
Merck’s Verquvo (vericiguat tablets) has been approved to reduce the risk of cardiovascular death (CVD) and heart failure (HF) hospitalization after hospitalization for heart failure or the need for outpatient diuretics in adults with symptomatic chronic heart failure and an ejection fraction lower than 45%. It is the first treatment for this indication.
The drug’s approval was based on based on efficacy findings in the phase 3, randomized, parallel-group, placebo-controlled, event-driven Victoria trial. More than 5,000 patients from the indicated population were randomized to receive either Verquvo or placebo. The primary endpoint was a composite of time to first event of CVD or HF hospitalization; median follow-up time for the endpoint was 11 months. A time-to-event analysis showed that Verquvo outperformed placebo in reducing CVD risk or HF hospitalization (hazard ratio, 0.90, 95% confidence interval, 0.82-0.98; P = .019), and there was an annualized absolute risk reduction of 4.2% with Verquvo compared with placebo during the study. The safety profile for Verquvo was similar to that of placebo.
The application was granted a priority regulatory review. Verquvo is being jointly developed by Merck, which holds the US commercial rights for the drug, and Bayer, which has exclusive rights for the rest of the worl

New indications
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.


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Tags: FDA, US

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