FDA Approvals Roundup: Pyrukynd and Solosec
A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).New approvals
Pyrukynd cleared for hemolytic anemia in adults with rare genetic disorder
Agios Pharmaceuticals’ Pyrukynd (mitapivat; tablets) has been approved for treating hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare genetic disorder that causes the destruction of red blood cells, leading to anemia.
The approval was based on findings from two studies – a randomized, double-blind, placebo-controlled study of 80 adults with PK deficiency who did not receive regular blood transfusions, and a single-arm study of 27 adults with PK deficiency who received regular blood transfusions.
In the randomized study, 40% of patients receiving Pyrukynd had a hemoglobin response, defined as an increase of 1.5 g/dL or more in hemoglobin concentration from baseline and sustained at two or more assessments during the 24-week study duration. Patients receiving placebo did not have a hemoglobin response. In the single-arm study, 33% of patients receiving Pyrukynd had a reduction in transfusion burden, defined as at least a 33% reduction in the number of red blood cell units transfused during the last 24 weeks of treatment compared with the patient’s historical transfusion burden. In addition, 22% of participants did not require any transfusions during the last 24 weeks of treatment of the 40-week trial.
The application received orphan drug and fast track designations and was granted priority review.
New indications
Solosec use expanded for bacterial vaginosis, trichomoniasis in adolescents
Lupin’s Solosec (secnidazole; oral granules) has been granted an expanded indication for treating bacterial vaginosis in female patients aged 12 years or older and trichomoniasis, a sexually transmitted infection, in all patients aged 12 years or older. Sexual partners of patients with trichomoniasis should also be treated with the antimicrobial agent to prevent reinfection.
Approval of Solosec for treating bacterial vaginosis in younger patients was supported by findings from a multicenter, open-label study in 40 female patients with bacterial vaginosis as well as evidence from previous studies in adult women. The approval for trichomoniasis was based on findings from a prospective, multicenter, placebo-controlled study in 147 female patients randomized 1:1 to receive Solosec or placebo. Of the 131 patients in the modified intent-to-treat population, those receiving Solosec had a 92.2% microbial cure rate at the test-of-cure visits on days 6 and 12, compared with 1.5% of those receiving placebo.
Solosec was originally approved in 2017 for treating bacterial vaginosis in adult women.
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