FDA Approvals Roundup: Rezurock, Fexinidazole, Bylvay

| 21 July 2021 | By Renee Matthews 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Rezurock cleared for treating chronic graft-versus-host disease
Kadmon’s Rezurock (belumosudil) has been approved for treating chronic graft-versus-host disease (GVHD) in adults and children aged 12 years or older after failure of previous systemic therapy.
Approval of the kinase inhibitor was based on efficacy findings from the randomized, open-label, multicenter dose-ranging KD025-213 trial that included 65 patients with chronic GVHD who were treated with Rezurock. Overall response rate was 75% (complete response, 6%; partial response, 69%). The median time to first response was 1.8 months; median duration of response was 1.9 months. Among those who responded to the treatment, no death or new systemic therapy initiation occurred in 62% for at least 12 months after the response.
The review was conducted under Project Orbis in collaboration with Australia’s Therapeutic Goods Administration, Health Canada, Swissmedic, and the UK’s Medicines and Healthcare products Regulatory Agency. It used the real-time oncology review pilot program and the assessment aid.
The application was granted priority review and breakthrough therapy designation.
Fexinidazole gets the goahead as first all-oral treatment for sleeping sickness
Sanofi’s fexinidazole has been approved as the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of human African trypanosomiasis, or sleeping sickness, in patients aged 6 years or older and weighing at least 20 kg. The parasitic disease is transmitted by an infected tse-tse fly and is fatal if left untreated.
The nonprofit research and development organization, Drugs for Neglected Diseases initiative (DNDi), conducted the pivotal clinical trials for this treatment, which was developed in partnership with the National Sleeping Sickness Programs of the Democratic Republic of Congo, Central African Republic, and Sanofi.
DNDi was awarded a tropical disease priority review voucher (PRV) under the FDA’s Tropical Disease PRV Program. The program was established in 2007 to incentivize development of new treatments for neglected tropical diseases.
Bylvay okayed for severe itching in patients with rare liver disease
Albireo’s Bylvay (odevixibat capsule) has been approved for treating pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC), a rare disorder causing progressive, life-threatening liver disease in children.
Pruritus is commonly associated with PFIC. Until now, there have been no approved drugs for treating PFIC; surgical techniques, including liver transplantation, have been the only available treatment options.
Approval of Bylvay was based on efficacy findings from a 24-week, randomized, double-blind, placebo-controlled study (Trial 1) in 62 patients aged 6 months to 17 years from the indicated population. They were randomized to receive a placebo or Bylvay. Primary endpoint was patient scratching, based on twice-daily observations by a caregiver. After 24 weeks, patients treated with Bylvay had a greater reduction in itching or scratching compared with placebo patients. Safety findings, as assessed in 79 patients aged 4 months to 25 years in a longer 72-week, open-label, single-arm study (Trial 2), supported the Trial 1 findings.
Bylvay received fast track review and orphan drug designations.
Octagam nabs approval for adult dermatomyositis
Octapharma’s Octagam 10% (immune globulin intravenous [human]) has been approved for treating adults with dermatomyositis, a rare immune-mediated inflammatory disease manifested by skin rashes, chronic muscle inflammation, and progressive muscle weakness.
The approval was based on efficacy and safety findings from the Phase 3 randomized, prospective, double-blind, placebo-controlled ProDERM study in 95 patients from the indicated population. The trial included an initial 16-week, double-blind, placebo-controlled phase during which patients received Octagam or placebo every 4 weeks. That was followed by a 24-week open-label extension phase. Patients were measured for response to treatment.
During the 16-week phase, 78.7% of patients receiving Octagam responded positively to treatment, compared with 43.8% of those on placebo. After switching to the study drug for the extension phase, the placebo group attained a response rate at week 40 that was similar to that of the Octagam-treated patients at week 16 (minimal improvement, 70%).
New indications
Prograf harnesses RWE to back expanded use for preventing organ rejection
Astellas’ Prograf (tacrolimus) has received a new indication for use in combination with other immunosuppressant drugs to prevent organ rejection in adults and children who undergo lung transplantation.
Approval for the new indication was based in part on a noninterventional, or observational, study that provided real-world evidence (RWE) of effectiveness. The real-world data (RWD) were obtained from the Department of Health and Human Services’ Scientific Registry of Transplant Recipients. The RWD were collected on all lung transplants in the US and were supplemented by information from the Social Security Administration’s Death Master File for mortality data. Analyses of the RWD showed a notable improvement in outcomes among lung transplant patients who received Prograf as part of their immunosuppression therapy compared with the natural history of patients who receive lung transplants with no or minimal immunosuppressive therapy.
The RWE from the noninterventional study was bolstered by confirmatory efficacy evidence from randomized controlled trials of Prograf used in other solid organ transplant settings. Additional published clinical trial evidence also provided support for Prograf’s efficacy as part of a multidrug immunosuppressive regimen.
Prograf is also approved for preventing organ rejection in adult and pediatric patients receiving liver, kidney and heart transplants.


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Tags: FDA, US

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