FDA Approvals Roundup: StrataGraft, Rezipres, Trikafta
A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).New approvals
StrataGraft greenlighted for treating thermal burns in adults
Mallinckrodt’s StrataGraft has been approved as a topical application treatment for adults with thermal burns containing intact deep skin layers for which surgical intervention is clinically indicated.
Deep thermal burns often require the removal of damaged skin and replacement with a skin graft, usually through an autograft using healthy skin from the patient. StrataGraft is placed on the burn wound to avoid or decrease the amount of healthy skin need for the graft.
Approval of the therapy was based on findings from two randomized clinical studies in a total of 101 adults from the indicated population. In both studies, two deep partial-thickness burn wounds of comparable area and depth on each patient were identified and randomized to receive either a single topical application of StrataGraft or autograft alone. The effectiveness of StrataGraft was supported by the percentage of StrataGraft treatment sites that achieved a complete wound closure and the decreased need for autografts at the StrataGraft sites.
StrataGraft was granted regenerative medicine advanced therapy, priority review, and orphan drug designations for this indication. It was developed in conjunction with the Department of Health and Human Services’ Biomedical Advanced Research and Development Authority.
StrataGraft is produced from two kinds of human skin cells (keratinocytes and dermal fibroblasts) grown together to make a bilayered cellularized scaffold. The human keratinocyte cells were grown with mouse cells during the early stages of product development, so StrataGraft is considered a xenotransplantation product (one that uses tissues or cells belonging to different species). Mouse cells are no longer used in the final manufacturing process. StrataGraft carries a risk of transmission of infectious diseases or agents because it contains cells from human donors and animal-derived materials from the early stages of product development, although no such transmission has been reported with StrataGraft.
Rezipres approved for treating hypotension during anesthesia
Eton’s Rezipres (ephedrine hydrochloride injection) has been approved for treating patients who develop hypotension during anesthesia.
Evidence for the efficacy of Rezipres was drawn from published literature, in which 14 studies reported increases in blood pressure after administration of ephedrine in the anesthesia setting. Nine of those studies included use of ephedrine hydrochloride in pregnant women undergoing neuraxial anesthesia during Cesarean delivery; one study was conducted in patients undergoing nonobstetric surgery under neuraxial anesthesia; and four studies included patients undergoing surgery under general anesthesia.
New indications
Trikafta nabs expanded indication for cystic fibrosis in younger children with certain mutations
Vertex’s Trikafta (elexacaftor, tezacaftor, ivacaftor combination and ivacaftor alone) has received an expanded indication for the treatment of cystic fibrosis in children aged 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data.
Approval of the combination therapy for this new indication was based on findings from a Phase 3, open-label, multicenter study in 66 children from the indicated population. The twice daily regimen, administered as morning combination and evening ivacaftor-alone doses, was generally well tolerated during the 24-week study and safety data were similar with those seen in earlier studies with children aged 12 or older.
Trikafta was first approved in 2019 for treating cystic fibrosis in patients aged 12 or older with at least one F508del mutation.
Epclusa gets new formulation for treating chronic HCV infection in children
Gilead’s Epclusa (sofosbuvir and velpatasvir) has received an expanded pediatric indication for treating chronic hepatitis C virus (HCV) in children as young as 3 years, regardless of HCV genotype or liver disease severity.
The therapy is available in oral pellet formulation and in two strengths for younger children who cannot swallow tablets.
Approval of Epclusa for this younger patient population was based on data from a Phase 2, open-label clinical trial with 41 children from the indicated population. At 12 weeks after treatment completion, the sustained virologic response (SVR 12), or cure rate, was 83% (34/41) among all patients, 88% (28/32) in those with HCV genotype 1, 50% (3/6) in those with HCV genotype 2, and 100% in children with HCV genotype 3 (2/2) and genotype 4 (1/1).
Epclusa was first approved in 2016 for treating adults with all major forms of chronic HCV infection. It received subsequent expanded indications for use in patients with HIV co-infection and in children aged 6 years or older.
Mavyret granted new indication for HCV infection younger children
Abbvie’s Mavyret (glecaprevir and pibrentasvir) has been approved in oral pellet formulation for treating HCV genotype 1, 2, 3, 4, 5 or 6 infection in children aged 3 through 11 years.
The approval is based on findings from two studies, DORA Part 1 and Part 2. In Part 1, all 47 children achieved SVR-12. In Part 2, with 62 children, the cure rate was 98%.
Mavyret was first approved in 2017 for adults with HCV infection. It received an extended indication in 2019 for children aged 12 or older.