FDA Approvals Roundup: Jemperli, Welireg, Xywav

RoundupsRoundups | 18 August 2021 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Jemperli cleared for dMMR advanced solid tumors
GlaxoSmithKline’s Jemperli (dostarlimab-gxly) has been granted accelerated approval for treating adults  with mismatch repair deficient (dMMR) recurrent or advanced solid tumors whose disease has progressed despite treatment.
The Ventana MMR RxDx Panel was also approved as a companion diagnostic device to select patients with dMMR solid tumors for treatment with Jemperli.
The approval for the tissue-agnostic therapy was based on efficacy findings from the nonrandomized, multicenter, open-label, multicohort GARNET trial in 209 patients from the indicated population. Overall response rate was 41.6%, with a complete response rate of 9.1% and partial response rate of 32.5%. Duration of response was 34.7 months (range, 2.6 to ≥35.8 months), with 95.4% of patients with duration 6 months or more.
The indication was approved under accelerated approval based on the tumor response rate and durability of response findings. Continued approval for the indication may depend on verification and demonstration of clinical benefit in a confirmatory trial or trials.
The review used the assessment aid, and the application was granted priority review.
Welireg okayed for cancers linked to von Hippel-Lindau disease
Merck’s Welireg (belzutifan) has been approved for treating von Hippel-Lindau (VHL) disease in adults being treated for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET) but who do not require immediate surgery.
The approval was based on findings from the ongoing Phase 2 open-label MK-6482-004 trial in 61 patients with VHL who had associated RCC, 24 with associated CNS hemangioblastomas, and 12 with associated pNET. Patients received Welireg until disease progression or unacceptable toxicity. Overall response rate (ORR) in patients with RCC was 49% (CNS hemangioblastomas, 63%; pNETs, 83%). 

Median duration of response (DoR) was not reached among patients with RCC, but 56% of responders had a DoR of 12 months or longer and a median time to response (TTR) of 8 months. Median DoR was also not reached for patients with CNS hemangioblastomas and pNET, but 73% and 50%, respectively, had response durations of 12 months or more. TTR in patients with CNS hemangioblastomas and pNETs was 3.1 months and 8.1 months, respectively.
The review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration, Health Canada, and the UK Medicines and Healthcare products Regulatory Agency. It used the Real-Time Oncology Review pilot program and the assessment and product quality assessment aids. The application was granted priority review for this indication.
New indications
Xywav use extended to adults with chronic sleep disorder
Jazz Pharmaceuticals’ Xywav (calcium, magnesium, potassium, and sodium oxybates) has received a new indication for treating adults with idiopathic hypersomnia, a chronic sleep disorder causing excessive daytime sleepiness even after a good night's sleep.
The drug was originally approved in 2020 for treating cataplexy or excessive daytime sleepiness in patients aged 7 years or older who have narcolepsy. The expanded indication marks the first time a treatment has been greenlighted for treating idiopathic hypersomnia.
The approval of Xywav was based on efficacy findings in a double-blind, placebo-controlled, randomized-withdrawal study in 154 patients aged 19 to 75 years from the indicated population. Patients who were randomized to switch from Xywav to placebo had worsening measures of sleepiness and symptoms of idiopathic hypersomnia compared with patients who continued with Xywav therapy.
Xywav carries a boxed warning for CNS depression and abuse and misuse, and its active moiety, oxybate, is a Schedule I controlled substance. The drug is therefore subject to strict safety controls on prescribing and dispensing under the Risk Evaluation and Mitigation Strategy.
The application was granted fast track and priority review designations. Xywav also received orphan drug status.
Lyumjev 100 units/mL approved for use in insulin pumps
Eli Lilly’s Lyumjev (insulin lispro-aabc injection 100 units/mL) has received a label expansion for administration by continuous subcutaneous insulin infusion with an insulin pump for glycemic control in adults with type 1 and type 2 diabetes.
The rapid-acting mealtime insulin was approved in 2020 in 100 or 200 units/mL dosages for subcutaneous administration in adults with diabetes.
Approval of the expansion was based on findings from the Phase 3 treat-to-target PRONTO-PUMP study showing the efficacy and safety of Lyumjev when used in insulin pumps in adults with type 1 diabetes. Compared with Humalog, Lyumjev demonstrated noninferior A1C reduction from baseline to week 16 and showed superior reduction in blood glucose spikes at 1 and 2 hours after meals.



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Tags: FDA, US

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