The first oral treatment for women with the common condition of uterine fibroids could be available as soon as summer 2018, with two other medications in the pipeline.
The drugs, experts say, would provide a new treatment option for a condition most commonly treated with a hysterectomy, or removal of the uterus.
“For the longest time we have not really had good options from an oral medication standpoint to treat fibroids,” says Arnold Advincula, vice chairman of women’s health and chief of gynecology at Sloane Hospital for Women at NewYork-Presbysterian/Columbia University Medical Center. “It’s nice to have some additional tools in the toolbox.”
Earlier in October the U.S. Food and Drug Administration accepted Allergan ’s new drug application for ulipristal acetate (UA) to treat abnormal uterine bleeding caused by uterine fibroids. The company expects a decision by May.
The drug has been available in Europe and Canada for several years.
Two other oral medications indicated to treat uterine fibroids are also in development. Bayer launched a phase 3 clinical trial—usually the final trial used by regulators to decide whether to approve a drug—for a similar drug, vilaprisan, in the summer. And AbbeVie is in the midst of phase 3 trials for elagolix to treat uterine fibroids and endometriosis, a disorder in which the tissue that lines the inside of the uterus grows outside of it, causing pelvic pain. The company hopes to have a product available by 2020.
Uterine fibroids are benign tumors that grow in and around the uterus. It’s estimated that as many as 26 million women in the U.S. have one or more such tumors, though less than half experience symptoms. The most common symptom is long and excessive bleeding during menstrual periods, which can lead to anemia and the need for blood transfusions. Other symptoms can include pelvic pain or pressure, a swollen abdomen and frequent urination. Fibroids can also cause fertility and pregnancy problems, depending on their size and location.
The cause of fibroids remains unknown, though those with a family history have an increased risk of developing them. Dietary factors may also play a role. African-American women have a higher rate of uterine fibroids. If a woman has no symptoms, treatment of fibroids isn’t necessary, experts say.
While a hysterectomy is the most common way to treat fibroid tumors, women who still want to have children can have a myomectomy. That surgery removes fibroids while keeping the uterus intact. But fibroids can grow back after the procedure. A number of other minimally invasive procedures are less commonly used and usually not recommended for women who want to have children. There’s some chance of recurrence with most of them.
UA belongs to a group of drugs called selective progesterone receptor modulators (SPRM). They block the progesterone from feeding the fibroids, which causes them to shrink.
“This particular drug also works directly on the lining of the uterus—the endometrium—so it also quite fast and dramatically decreases and stops the vaginal bleeding,” says Ayman Al-Hendy, director of interdisciplinary translational research at Augusta University in Georgia and a member of Allergan’s advisory board. Bleeding is reduced within five to six days, he says. Headaches and hot flashes were the most common side effects in the trials with UA, affecting less than 10% of women.
Patients take the once-a-day pill for three months and then stop for one menstrual cycle to allow the endometrium to shed, since the medication makes it grow thicker. Dr. Al-Hendy says the changes to the endometrium don’t raise the risk of endometrial cancer, which some have raised as a concern.
Patients with the condition say it’s time for better treatments.
Tanika Gray Valbrun, a 39-year-old network news producer in Atlanta, says she welcomes less invasive treatments that don’t affect fertility. She started the White Dress Project, a nonprofit patient advocacy group for women with uterine fibroids, in 2014.
She was diagnosed with fibroids at 19. She has suffered from pelvic pain, abdominal bloating that has resulted in people mistakenly thinking she’s pregnant and extremely heavy bleeding that has left her anemic.
“I’ve had five blood transfusions because of the amount of blood I’ve lost,” she says.
She has tried different types of birth control, but none helped with her symptoms. In 2013 she had a myomectomy and doctors removed 27 fibroids, keeping her out of work for nine weeks. Four years later, the fibroids are back.
Vanessa Jacoby, an associate professor in the department of obstetrics, gynecology and reproductive sciences at University of California, San Francisco, conducts research in new fibroid treatments.
She says UA appears to have fewer side effects than Lupron, the only medication, an injectable, available in the U.S. that shrinks fibroids. Lupron effectively puts women into early menopause, which can result in hot flashes and bone loss. Women often must take it with hormone replacement therapy. It is usually used in preparation for surgery to shrink fibroids and maintain blood reserves.
While the company has done studies of women taking the drug up to a year, some experts believe the long-term health profile of a drug needs to be evaluated up to seven years, Dr. Jacoby says.
Shao-Lee Lin, vice president of therapeutic areas and international development at AbbVie , says the company is testing a twice daily dosing of elagolix. It is also studying including low-dose hormone therapy within elagolix to help maintain bone health.
Like Lupron, elagolix decreases sex hormones that cause fibroids to grow. What makes it different is it’s fast-acting and easier to reverse the effects if you stop taking it.
Studies have found it significantly reduces heavy menstrual bleeding and decreases the thickness of the endometrium by six months. It also decreases the size of fibroids.