FDA Approvals Roundup: Vtama, Dupixent, Vidaza

RoundupsRoundups | 25 May 2022 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approval
Vtama approved as topical, steroid-free treatment for plaque psoriasis in adults
Dermavant’s VTAMA (tapinarof; cream, 1%) has been approved as a steroid-free, topical treatment for adults with mild-severe plaque psoriasis.
Approval of the aryl hydrocarbon receptor agonist was based on findings from the Phase 3, multicenter, double-blind, vehicle-controlled PSOARING 1 and PSOARING 2 trials in 1,025 patients who were randomized 2:1 to daily treatment with VTAMA cream or vehicle cream for 12 weeks to any lesion. Across both trials, VTAMA cream showed statistically significant improvement at week 12 on the Physician Global Assessment 6 score (0, Clear or 1, Almost Clear), compared with vehicle. VTAMA cream also showed statistically significant improvement in all secondary endpoints, compared with the vehicle cream, including an improvement of 75% or more in the Psoriasis Area and Severity Index score from baseline.
Eligible patients who completed PSOARING 1 or PSOARING 2 could enroll in PSOARING 3, a 40-week, Phase 3, long-term extension (LTE) study. In all, 763 patients (92%) from the first two trials enrolled in the LTE study. More 40% of the LTE study patients (n = 312) achieved complete disease clearance at least once during the study period.
New indications
Dupixent gets new indication for eosinophilic esophagitis
Regeneron’s Dupixent (dupilumab; injection) has been granted a new indication for treating eosinophilic esophagitis (EoE) in adults and pediatric patients aged 12 years or older.
This is the first FDA approval of a treatment for the chronic inflammatory disorder. Dupixent, a monoclonal antibody, was originally approved in 2017 for treating moderate-to-severe atopic dermatitis in adult and pediatric patients aged 6 and older. It is also approved as an add-on maintenance therapy for adults and pediatric patients aged 6 and older with certain types of moderate-to-severe asthma and for adults with inadequately controlled chronic rhinosinusitis with nasal polyposis.
The efficacy and safety of Dupixent in EoE was studied in a randomized, double-blind, parallel-group, multicenter, placebo-controlled trial, that included two 24-week treatment periods (Part A and Part B) that were conducted independently in separate groups of patients.
In Part A, 60% of the 42 patients receiving Dupixent achieved the predetermined level of reduced eosinophils in the esophagus, compared with 5% of the 39 patients receiving placebo. Dupixent patients in Part A had an average improvement of 22 points in the patient-reported Dysphagia Symptom Questionnaire( DSQ) score, compared with 10 points in those on placebo.
In Part B, 59% of the 80 patients who received Dupixent achieved the predetermined level of reduced eosinophils in the esophagus, compared with 6% of the 79 placebo patients. Patients in Part B who received Dupixent had an average improvement of 24 points in the DSQ score, compared with 14 points in placebo patients who received placebo.
Dupixent received priority review and breakthrough therapy designations for this indication.
Vidaza gets new indication for newly diagnosed juvenile myelomonocytic leukemia
Celgene’s Vidaza (azacytidine; injection) has been granted a new indication for treating pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).
Approval of Vidaza, a nucleoside metabolic inhibitor, was based on findings from the international, open-label AZA-JMML-001 study in 18 pediatric patients with JMML. A total of 9 patients had confirmed clinical responses, of whom 3 had complete clinical remission and 6, partial remission. Median time to response was 1.2 months. The proportion of patients undergoing hematopoietic stem cell transplantation was 94%, and median time to HSCT was 4.6 months.
Vidaza was approved in 2004 for treating adults with certain myelodysplastic syndrome subtypes.
This review used the assessment aid. The application was granted priority review and breakthrough designations.
TPOXX nabs new IV formulation for smallpox
Siga’s TPOXX (tecovirimat; injection) been approved as an intravenous formulation for treating smallpox in adults and children.
The drug was originally approved in 2018 for treating smallpox in adults and children weighing at least 13 kg.
Tyvaso approved in powder form
United Therapeutics’ Tyvaso DPI (treprostinil) has been approved in powder form for pulmonary arterial
Tyvaso, a prostacyclin vasodilator, was originally approved in 2009 for treating adults with pulmonary arterial hypertension.


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Tags: FDA, US

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