FDA Approvals Roundup: Xaciato, cutaquig, Rituxan

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

New Approvals
Xaciato okay for treating bacterial vaginosis

Daré’s Xaciato (clindamycin phosphate vaginal gel) is approved for the treatment of bacterial vaginosis in patients 12 years of age or older.

The approval was based on findings from the Phase 3 multicenter, double-blind, placebo-controlled DARE-BVFREE trial in which 307 patients from the indicated population were randomized 2:1 to receive either Xaciato or placebo. Test-of-cure visitation data (days 21-30) showed that a significantly greater percentage of Xaciato patients in the modified intent-to-treat population experienced clinical cure, bacteriological cure, and therapeutic cure compared to those who received placebo (clinical : 70.5% and 35.6%, respectively, Bacteriological: 43.4% vs. 5.1%, Therapeutic: 36.9% vs. 5.1% Statistically significant results for the endpoints were also achieved at a mid-term review during days 7-14 (clinical: 76.2% vs. 23.7%; bacteriological: 41% vs. 3.5%; therapeutic: 35.2% vs. 0).

Xaciato received Qualified Infectious Disease Product (QIDP) and accelerated designations for this indication. Under the QIDP designation, the drug will likely be extended for another five years until the three-year market exclusivity available for the drug.

New indications
cutaquig use extended for primary humoral immunodeficiency in children

Octapharma’s cutaquig (immunoglobulin, subcutaneous) [Human]hipp solution) has been given a new indication for the treatment of children aged 2 years and older with primary humoral immunodeficiency (PI).

The current approval was based on findings from two clinical studies. One was a pivotal prospective, open-label, single-arm, multicenter study to evaluate the pharmacokinetics, efficacy, tolerability and safety of Cutaquig; the other was an extension study. In the pivotal trajectory, 75 (37 adults and 38 children) of the indicated population were randomized weekly cutaquig infusions over a 12-week wash-in/out period followed by a 12-month efficacy period. The main aim was to assess the efficacy of cutaquig in preventing serious bacterial infections. No serious bacterial infections were reported.

The drug was originally approved in 2018 for the treatment of primary humoral
immune deficiency in adults. It comes with a boxed warning for thrombosis.

Rituxan gets new indication for lymphoma, leukemia in children

Genentech’s Rituxan (rituximab injection), combined with chemotherapy, has gained a new indication for the treatment of children 6 months to less than 18 years of age who have previously untreated advanced CD20-positive diffuse large B-cell lymphoma (DLBCL), Burkitt- lymphoma (BL), Burkitt-like lymphoma (BLL), or adult B-cell acute leukemia (B-AL).

Approval for the new indication was based on findings from the global, multicenter, open-label Inter-B-NHL Ritux 2010 trial in which patients from the indicated population were randomized 1:1 to receive chemotherapy alone or in combination with Rituxan. The main efficacy outcome measure was event-free survival (EFS).

A pre-specified interim efficacy analysis was performed at 53% information fraction in 328 randomized patients after a median follow-up of 3.1 years. In total, there were 28 EFS events in the chemotherapy group and 10 in the Rituxan chemotherapy group. Also, at the time of the interim analysis, there were 20 deaths in the chemotherapy arm, compared to 8 in the study arm, with an estimated overall survival risk ratio of 0.36. There was no formal statistical test for overall survival. Randomization was discontinued after the interim analysis, with an additional 122 patients receiving combination Rituxan chemotherapy.

The assessment tool was used for the assessment and the application was assessed with priority.

Rituxan, a CD20-targeted cytolytic antibody, was originally approved in 2014. It has been used to treat a number of diseases, including non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, and rheumatoid arthritis.

Extended use of Keytruda in children with melanoma

Merck’s Keytruda (pembrolizumab injection) has gained a new indication for the adjuvant treatment of adults and children 12 years of age or older who have stage IIB or IIC melanoma after complete resection.

The approval of this new indication was based on the efficacy findings of the multicenter, double-blind, placebo-controlled KEYNOTE-716 study in which patients from the indicated population were randomized 1:1 to receive Keytruda or placebo for up to one year until disease recurrence or unacceptable toxicity. Relapse-free survival (RFS) was the main efficacy outcome measure. Findings showed a significant improvement in RFS at the first interim analysis for patients receiving the Keytruda arm compared to placebo (HR 0.65). Median RFS was not achieved in either arms.

The assessment used the real-time oncology assessment pilot program and assessment tool. The application received priority assessment and orphan drug designation.

Keytruda, a human programmed death receptor 1‒-blocking antibody, was originally approved in 2014 for use in previously treated metastatic melanoma and is used in many other cancers, including non-small cell lung cancer and triple-negative breast cancer.

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