The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for relugolix combination tablet (relugolix, estradiol, norethindrone acetate; Myovant Sciences) for the treatment of women with heavy menstrual bleeding associated with uterine fibroids.

Relugolix is an investigational oral gonadotropin-releasing hormone (GnRH) antagonist. The NDA submission is supported by results from the phase 3 LIBERTY program, which includes 2 multinational, replicate phase 3 studies (LIBERTY 1 and 2) and an open-label extension study. LIBERTY 1 and 2 evaluated the efficacy and safety of relugolix combination therapy in women with heavy menstrual bleeding with uterine fibroids. Patients were randomized to receive either relugolix combination therapy once daily for 24 weeks, relugolix 40mg once daily monotherapy for 12 weeks followed by relugolix combination therapy once daily for an additional 12 weeks, or placebo once daily for 24 weeks. 

The primary endpoint of the studies was the proportion of women who achieved menstrual blood loss volume of <80mL and a ≥50% reduction from baseline in menstrual blood loss volume during the last 35 days of the 24-week treatment period (responder criteria).

Results from LIBERTY 1 and 2 showed that 73.4% and 71.2% of women treated with relugolix combination therapy achieved responder criteria vs 18.9% and 14.7% of women who received placebo, respectively (P <.0001). Additionally, an 84.3% reduction in menstrual blood loss from baseline was observed in both studies among women treated with relugolix combination therapy (P <.0001).

Patients who completed the LIBERTY 1 or 2 studies were eligible to enroll in the open-label extension study of relugolix combination therapy for an additional 28 weeks. Findings from the open-label extension study showed that treatment with the combination therapy was associated with an 87.7% response rate at 52 weeks, as well as an average reduction in menstrual blood loss of 89.9% from baseline.

As for safety, relugolix combination therapy and placebo were found to be comparable with regard to overall incidence of adverse events.

A Prescription Drug User Fee Act (PDUFA) target action date of June 1, 2021 has been set for this application.

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“An estimated five million women in the US suffer from symptomatic uterine fibroids, which may include heavy menstrual bleeding, pain, and anemia,” said Lynn Seely, MD, CEO of Myovant Sciences. “Noninvasive treatment options are limited, and we believe women deserve an alternative to surgery that delivers a predictable and clinically-meaningful reduction in menstrual blood loss while maintaining bone health as demonstrated by relugolix combination therapy in the LIBERTY program.”

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Myovant Sciences announces FDA acceptance of New Drug Application for once-daily Relugolix combination tablet for uterine fibroids. Accessed August 17, 2020.