FDA Approvals Roundup: Ponvory, Zegalogue, Exparel

RoundupsRoundups | 24 March 2021 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Ponvory okayed for use in adults with relapsing multiple sclerosis
Janssen’s Ponvory (ponesimod) has been approved as a once-daily oral therapy for adults with relapsing multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.
Approval of Ponvory was based on findings in the phase 3 prospective, multicenter, randomized, double-blind OPTIMUM study in 1,133 patients from the indicated population. Patients were randomized 1:1 to receive Ponvory or Aubagio (teriflunomide) and followed for 108 weeks.
The annualized relapse rate from baseline through Week 108 was significantly reduced by 30.5% in the study group compared with Aubagio (P = .0003). There were no confirmed relapses in 70.7% of patients receiving Ponvory, compared with 60.6% in the Aubagio group, and the proportion of patients with confirmed disability progression 3 months from baseline was 10.8% and 13.2%, respectively. In addition, the number of new gadolinium-enhancing T1 lesions (indicators of disease activity and burden) per year for the two groups were 1.46 and 3.16, respectively (relative reduction: 55.7%).
Zegalogue approved for treating severe hypoglycemia in people with diabetes
Zealand Pharma’s Zegalogue (dasiglucagon) has been approved for the treatment of severe hypoglycemia in patients with type 1 diabetes aged 6 years or older.
Approval was based on efficacy results from three phase 3 randomized, double-blind, placebo-controlled, multicenter studies in 20 children aged 6-17 years and 316 adults with type 1 diabetes. In all three studies, the primary endpoint of time to plasma glucose recovery, or treatment success, was achieved. Median time to blood glucose recovery was 10 minutes after treatment with Zegalogue, compared with 30-45 minutes with placebo. In one of the three trials, 99% of patients recovered within 15 minutes. 
New indications
Exparel nabs new indication as postsurgical, local analgesia for children
Pacira’s Exparel (bupivacaine liposome injectable suspension) has received an expanded indication for use as single-dose, long-acting postsurgical local analgesia in patients aged 6 years or older. The approval provides a nonopioid option for pain control in this patient population.
The supplemental new drug application was approved based on safety and pharmacokinetic findings from the phase 3 randomized, multicenter PLAY study of 98 children aged between 6 and 17 years undergoing spinal or cardiac surgeries. The findings matched those from similar studies for adult patients.
Exparel was first approved in 2011 for use as a postsurgical local analgesia in adults.
Arcalyst gets expanded indication for pediatric recurrent pericarditis
Kiniksa’s Arcalyst (rilonacept injection) has been approved for treating recurrent pericarditis and reducing the risk of recurrence in adults and children aged 12 years or older. 
Approval was based on efficacy and safety findings in the double-blind, randomized RHAPSODY study with 86 patients from the indicated population. All  patients received Arcalyst for a 12-week run-in period, after which they were randomized 1:1 to receive either Arcalyst or placebo. Primary endpoint was the time to first pericarditis recurrence. In all, 2 patients (7%) in the study drug arm had a recurrence, compared with 23 (74%) patients in the placebo arm. Patients receiving Arcalyst had a median time-to-treatment response of 5 days and a treatment response rate of 97%. In addition, they also had a 96% reduction in risk for recurrence of a pericarditis event and were pain free or had minimal pain for 92% of trial days, compared with 40% of trial days for those on placebo.
The drug received orphan drug designation for the treatment of recurrent pericarditis.
Arcalyst was discovered by Regeneron and was first approved in 2008 for the treatment of Cryopyrin-associated periodic syndromes. In 2020, it received expanded approval for maintaining remission of deficiency of interleukin-1 receptor antagonist.
In 2017, Kiniksa licensed Arcalyst from Regeneron. Following the latest approval, for recurrent pericarditis, Kiniksa will be responsible for sales and distribution of the drug for all approved indications in the US. It will split the profits 50:50 with Regeneron under a license agreement.
Keytruda handed extended indication for esophageal or GEJ carcinoma
Merck’s Keytruda (pembrolizumab), in combination with platinum and fluoropyrimidine-based chemotherapy, has received an extended indication for treating metastatic or locally advanced esophageal or gastroesophageal junction (GEJ) carcinoma in patients who do not qualify for surgery or chemoradiation.
The approval was based on efficacy findings in the multicenter, randomized, placebo-controlled KEYNOTE-590 study with 749 patients from the indicated population. They were randomized 1:1 to receive Keytruda or placebo, both in combination with cisplatin and fluorouracil, until unacceptable toxicity or disease progression. Median overall survival was 12.4 months for the Keytruda arm and 9.8 months chemotherapy-alone arm. Median progression-free survival was 6.3 and 5.8 months, respectively.
The review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration, Health Canada, and Swissmedic. It used the real-time oncology review pilot program and the assessment aid. The application was granted priority review.
Keytruda was first approved in 2014 and is used to treat several types of cancers, including melanoma, head and neck squamous cell cancer, and triple-negative breast cancer.



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Tags: FDA, US

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