Esmya - Ulipristal acetate

What is Esmya - Ulipristal acetate?

Esmya is a medicine that contains the active substance ulipristal acetate, available as tablets (5 mg).

What is Esmya - Ulipristal acetate used for?

Esmya is indicated in the pre-operative treatment of moderate to severe symptoms of uterine fibroids, non-cancerous (benign) tumors of the uterus. Esmya is used in adult women who have not yet reached menopause.

The medicine can only be obtained with a prescription.

How is Esmya - Ulipristal acetate used?

Esmya is taken by mouth and the recommended dose is one tablet a day. The treatment can last up to three months and must start during the first week of the menstrual cycle (menstruation).

How does Esmya - Ulipristal acetate work?

The active ingredient of Esmya, ulipristal acetate, is a "selective progesterone receptor modulator". It works by blocking the receptor of a hormone in the body called progesterone, which is responsible for controlling the growth of the uterine mucosa. In some women progesterone can promote the growth of fibroids, which can cause symptoms such as abundant uterine bleeding (bleeding from the uterus at or less than the menstrual period), anemia (decrease in the number of red blood cells in the blood, due to hemorrhage) and abdominal pain (menstrual pain or stomach ache). If progesterone activity is interrupted, fibroid cells break up the division and die. In this way the size of the fibroids is reduced and the related symptoms diminish.

What studies have been carried out on Esmya - Ulipristal acetate?

The effects of Esmya were first tested in experimental models before being studied in humans.

Two main studies were conducted with Esmya, involving 549 women with symptomatic uterine fibroids. In both studies the treatment lasted three months.

One study looked at the effects of Esmya compared to placebo (a dummy treatment) in adult women with abundant uterine bleeding and anemia, who would have had to undergo surgery to remove the fibroids. All patients were also subjected to an iron supplement treatment for anemia. The main indicators of effectiveness were the decrease in the haemorrhagic flow and the associated anemia and the reduction in the size of the fibroids.

The second study investigated the effects of Esmya compared to another medicine used in the treatment of fibroids, leuprorelin. The main measure of effectiveness was the ability of the treatment to reduce the abundant uterine bleeding.

What benefits has Esmya shown during the studies?

Esmya has been shown to improve symptoms in patients with uterine fibroids.

In the first study, 91.5% of women treated with Esmya reported a reduction in menstrual bleeding compared to 18.8% of women treated with placebo. The size of the fibroids also decreased more with Esmya than with placebo.

In the second study, Esmya demonstrated an equivalent efficacy to that of leuprorelin in reducing hemorrhagic flow, because the bleeding had decreased in 90.3% of women treated with Esmya compared to 89.1% of patients treated with leuprorelin.

What is the risk associated with Esmya?

The most common side effects with Esmya (seen in more than 1 patient in 10) are amenorrhea (absence of menstruation), thickening of the endometrium (thickening of the mucosa covering the internal cavity of the uterus) and flushing. For the full list of all side effects reported with Esmya, see the package leaflet.

Esmya should not be used in women who are hypersensitive (allergic) to ulipristal acetate or any of the other ingredients. It must not be used during pregnancy and lactation, in case of vaginal bleeding of unknown origin or for reasons other than uterine fibroid, in the presence of cancer of the uterus, cervix (cervix), ovarian or mammary. The duration of treatment with Esmya should not exceed three months. For more details, see the package leaflet.

Why has Esmya - Ulipristal acetate been approved?

The CHMP concluded that the effectiveness of Esmya in reducing bleeding and anemia as well as the size of fibroids has been demonstrated. No critical safety elements emerged. The increase in endometrial thickness seen in some patients usually resolves at the end of treatment. The CHMP therefore decided that Esmya's benefits are greater than its risks and recommended that it be given marketing authorization. The committee limited the duration of treatment to three months, due to the absence of long-term safety data.

What measures are being taken to ensure the safe use of Esmya?

The company that markets Esmya must ensure that all doctors who could prescribe the medicine are given informational material containing important information on the safety of Esmya, including recommendations for monitoring and managing endometrial changes caused by treatment with Esmya.