FDA Approvals Roundup: Ztalmy, Opdualag, Keytruda

RoundupsRoundups | 23 March 2022 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Ztalmy gets go-ahead for rare form of genetic epilepsy in children, adults
Marinus Pharmaceuticals’ Ztalmy (ganaxolone; oral suspension) has been approved for treating seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD), a rare form of genetic epilepsy, in patients aged 2 years or older.
Approval of Ztalmy, a neuroactive steroid, was based on findings from the Phase 3, double-blind, placebo-controlled MARIGOLD trial in which 101 patients from the indicated CDD population were randomized 1:1 to receive Ztalmy or placebo. Patients in the Ztalmy group had a significantly greater reduction in 28-day major motor seizure frequency, compared with those receiving placebo (30.7% and 6.9% reductions, respectively). Of 48 patients treated for at least 12 months in an open-label extension study, a median 49.6% showed reduction in major motor seizure frequency.
The application was granted priority review, orphan drug, and rare pediatric disease designations for treating CDD. The company was also granted a rare pediatric disease priority review voucher.
Opdualag combo okayed for advanced melanoma in adults, adolescents
Bristol Myers Squibb’s Opdualag (nivolumab and relatlimab-rmbw; IV injection) has received approval for its biologics license application for treating unresectable or metastatic melanoma in adults and pediatric patients aged 12 years or older.
The drug is a fixed-dose combination of nivolumab, a PD-1 blocking antibody, and relatlimab, an LAG-3-blocking antibody.
Approval of Opdualag was based on findings from the double-blinded RELATIVITY-047 trial in which 714 patients from the indicated population were randomized 1:1 to receive the Opdualag combination or nivolumab alone every 4 weeks until disease progression or unacceptable toxicity.
Patients receiving Opdualag showed a statistically significant improvement in progression-free survival (PFS) compared with nivolumab alone patients (median PFS, 10.1 months and 4.6 months, respectively). Overall survival (OS) was not statistically significant (median OS, not reached [Opdualag arm] and 34.1 months [nivolumab alone]).
The review for this application was conducted under Project Orbis, in collaboration with the Australian Therapeutic Goods Administration and Swissmedic. It also used the real-time oncology review pilot program and the assessment aid. The application granted priority review and fast track and orphan drug designations.
New indications
Keytruda nabs expanded indication for advanced endometrial carcinoma
Merck’s Keytruda (pembrolizumab; IV injection) has been granted a new indication for treating advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), as determined by an FDA-approved test, in patients with disease progression following systemic therapy and who are not candidates for curative surgery or radiation.
The Ventana MMR RxDx Panel (Ventana Medical Systems/Roche Tissue Diagnostics) received concurrent approval as a companion diagnostic for selecting patients with dMMR in solid tumors who would be eligible for treatment with Keytruda. The FoundationOne CDx (Foundation Medicine) has already been approved as a companion diagnostic for selecting patients with MSI-H who would be eligible for the treatment.
Approval for this indication for Keytruda was based on findings from the multicenter, nonrandomized, open-label, multicohort KEYNOTE-158 trial in which 90 patients from the indicated population received Keytruda every 3 weeks until unacceptable toxicity or documented disease progression. Median objective response rate was 46%. Median duration of response was not reached, with 68% having response durations of 12 months or longer, and 44% having response durations of 24 months or longer.
This review used FDA’s assessment aid program.
Keytruda, a PD-1‒blocking antibody, was originally approved in 2014 for use in previously treated metastatic melanoma and is used in numerous other cancers, including non-small cell lung cancer and triple-negative breast cancer.



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Tags: FDA, US

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