AbbVie, in collaboration with Neurocrine Biosciences, has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for its lead candidate elagolix to treat heavy menstrual bleeding in women with uterine fibroids.
“This submission brings us closer to our goal of improving care for women who continue to struggle with the often disruptive effects of uterine fibroids,” Michael Severino, MD, AbbVie’s vice chairman and president, said in a press release.
Uterine fibroids, also known as leiomyomas or myomas, are noncancerous tumors that grow around the uterus. They are the most frequent type of abnormal growth in a woman’s pelvis, and can affect up to 70% of Caucasian women and up to 80% of African-American women by age 50.
Although fibroids can be asymptomatic, some can cause symptoms such as heavy menstrual bleeding, painful periods, vaginal bleeding outside of menstruation, and anemia.
Non-surgical treatments for uterine fibroids are limited, highlighting the need for more therapeutic options.
Elagolix is an oral medicine that blocks gonadotropin-releasing hormone (GnRH) signalling, by binding to GnRH receptors in the pituitary gland. This way, elagolix lowers the levels of the sex hormones estrogen and progesterone, which are linked to the development and worsening of uterine fibroids, as well as to endometriosis.
The company’s NDA submission is supported by data from the elagolix Phase 3 Uterine Fibroids Program, which included two Phase 3 trials, ELARIS UF-I (NCT02654054) and ELARIS UF-II (NCT02691494), and an extension trial, the ELARIS UF-EXTEND study (NCT02925494).
The multi-center ELARIS UF-I and ELARIS UF-II studies assessed the effectiveness and safety of elagolix alone (300 mg twice daily) and in combination with low-dose sex hormone therapy (estradiol 1.0 mg/norethindrone acetate 0.5 mg), compared with a placebo, for six months. The trials enrolled nearly 800 women with uterine fibroids.
In the extension study, women already receiving treatment were able to continue the medication for an additional six months. Those on placebo in the previous trial were randomly selected to receive elagolix alone or the combo therapy, for six months.
The studies’ primary goal was to assess the percentage of women who achieved clinical response, defined as at least a 50% reduction in menstrual bleeding. Menstrual blood loss was measured by the alkaline haematin method, which estimates the concentration of hemoglobin — the protein that carries oxygen throughout the body — in menstrual blood collected from sanitary products.
The results showed that around 70% of women receiving the combo therapy achieved clinical response, compared with approximately 10% of those on placebo. In the extension study, the percentage of women showing clinical response reached 87.9%.
The most common elagolix-related adverse effects reported were hot flush, headache, night sweats, nausea, and fatigue.
Detailed results from the Phase 3 Uterine Fibroids Program are expected to be published in a medical journal later this year.
“If approved, this combination has the potential to offer women and their healthcare providers an additional oral medical treatment option,” Severino added.