FDA approves AbbVie endometriosis drug elagolix Orilissa

Instead of turning to surgery, drugmakers are hoping to treat diseases like uterine fibroids and endometriosis with new medications.Reuters

• The FDA just approved elagolix, a new treatment for women living with endometriosis.
• Endometriosis, a condition that affects millions of women, occurs when tissue from the uterus grows outside of it, leading to cysts, heavy bleeding, and scarring. 
• It’s the first new treatment in a pill form for the disorder in more than a decade. 

The FDA just approved a new treatment for endometriosis. 

The drug, elagolix — now marketed as Orilissa — is made by AbbVie in partnership with Neurocrine Biosciences, and it’s taken as a daily pill.  It’s the first new treatment for the disorder in a pill form in more than a decade, and will cost $844.87 a month, or $10,138.44 a year. 

An estimated 4 to 10 million women in the US are living with endometriosis, a condition in which tissue from the uterus grows outside the uterus, leading to cysts, heavy bleeding, and scarring. Worldwide, the Endometriosis Foundation of America estimates that the condition affects 200 million women. The condition has made headlines in recent years as “Girls” creator Lena Dunham has publicly documented her experience living with the condition.

Up until now, endometriosis has been treated through hormone-altering medications including birth control as well as surgery to remove the tissue. 

Endometriosis can be a painful condition, but that pain can often be misconstrued for regular period cramps.

That can mean diagnosing the condition can take about 10 years after symptoms start showing up. In the case of Piraye Beim, 39, she went undiagnosed until she almost gave birth to her second child in an Uber on the way to the hospital because she hadn’t realized just how far along into labor she was. To Beim, the pain from labor was the same as her monthly period.

“It was a very emotional realization for me that what I thought was a normal part of being a woman wasn’t normal at all,” said Beim, who is the CEO of women’s health company Celmatix. “Going through that and realizing that transitional labor is what my periods feel like is why I started seeking answers at that point.” 

For others, like Kristy Curry, 40, from New York, it took a few visits to the emergency room before she got a diagnosis. She had thought maybe she was just more sensitive to painful cramping.  

“Something bigger seems to be going on,” she eventually realized. She’s undergone surgery to remove the tissue and uses birth control to mitigate symptoms and follows a vegan diet in the hopes that it could mitigate some of the symptoms.

Michelle Kinney, an executive at a spa management company, didn’t realize she had endometriosis until she underwent surgery for what she thought was appendicitis. She’s now had surgery three times, but doesn’t take medication apart from the occasional pain drugs. Her pain – which she describes as sometimes feeling like little people in side her skating around on razor blades and other times like someone’s poured gasoline inside her — often leaves her in a position where she can’t get out of bed. 

“There’s no getting up,” she said. 

The pain can manifest itself differently. For Curry, she describes it as a twisting, stabbing pain. For Michelle Gilmore, a 34-year-old software designer, the pain comes in various forms.

“It’s almost like the pain has become an object attached to me,” she said. That could mean anything from a slight pulsing sensation in her left ovary, or at its worst like someone’s grating her insides. She recently had surgery, and “reluctantly” went on birth control after. 

Treating endometriosis involves changing hormone levels in the body, and events like pregnancy and menopause can bring relief. From a medication perspective, there have been a few ways to mimic that, including using leuprolide, a hormone suppressant that’s injected, and contraceptives. But those treatments can come with major side effects, including bone density loss and hot flashes. 

A new wave of treatments

Orilissa, taken as a pill, works by altering hormone levels in women endometriosis to reduce the pain associated with the conditions.

“It’s a drug that suppresses estrogen gently,” Dr. Sanjay Agarwal, a professor and Director of Fertility Services in the UC San Diego Department of Reproductive Medicine, who was an investigator on the Orilissa trial. “The goal is to reduce the pain of endometriosis without creating excessive problems from low estrogen levels.” Those include bone density loss and hot flashes that are side effects of other treatments. 

This is the first time there’s been new treatments for endometriosis since the 1990s, Dr. Linda Giudice, a reproductive endocrinologist and professor the University of California-San Francisco told Business Insider. For endometriosis alone, the market is estimated to be as much as $6.8 billion, according to analysts at Jefferies. To put that into perspective, a condition like rheumatoid arthritis, which impacts fewer patients in the US, has a roughly $10 billion market. 

This may be because while endometriosis affects millions of women in the US, many are misdiagnosed for years. And even if doctors believe a woman might have the disorder, diagnosis requires a complicated, full-on surgical procedure.  

Elagolix is the first in a new wave of treatments in development for both endometriosis and uterine fibroids, a separate condition characterized by benign tumors that grow in and around the uterus that can cause heavy bleeding and pain. Kevin Gorman, the CEO of Neurocrine Biosciences told Business Insider that the drug first started clinical trials as far back as 2003. Getting the drug through phase 3 trials alone took up the last seven years. Here are some other drugs in the works to treat uterine fibroids and endometriosis, some of which are looking to compete with Orilissa. 

• Myovant’s drug, relugolix, works by suppressing estrogen to low levels, and then reintroducing just enough so that it doesn’t lead to bone density loss. The US trials for Relugolix should wrap up in 2019, but a phase 3 trial conducted in Japan found that the drug worked as well as leuprolide, one of the treatments currently available for uterine fibroids.
• AbbVie is also running a trial to see how Orilissa works in treating uterine fibroids. In 2018, two of its phase 3 trials had positive results by reducing heavy menstrual bleeding in 68.5% and 76% of the women on the trial with uterine fibroids, respectively, compared to a 8.7% and 10.1% reductions in the placebo group. 
• Allergan is developing Esmya, a drug that’s being investigated for use in uterine fibroids. The drug works by modulating progesterone, a key hormone for women relating to the uterus. Allergan is aiming for a potential approval by August. 
• Bayer’s drug, vilaprisan, also works by modulating progesterone levels to treat uterine fibroids and endometriosis. In July 2017, Bayer began a phase 3 trial studying the use of Vilaprisan to treat uterine fibroids. The trial is expected to take three years. 

What’s still missing

Ideally, the approval of medications like Orilissa could spark more investment in the space so that the move from injections to pills isn’t the last step.

The National Institutes of Health plans to spend about $7 million a year on endometriosis research in 2018, down from the $10 million it spent in 2015 and 2016. Diabetes, in comparison, will get about $1.1 billion in funding in 2018.  Beim, whose company Celmatix makes a genetic test that screens for risk factors associated with female fertility including endometriosis, said she hopes one day we’ll start to see more personalized treatments for the condition, the way we do with targeted cancer treatments. 

Giudice, who advises both AbbVie and Myovant as well as Celmatix, said she’d like to see more improvements in the way endometriosis is diagnosed. Currently, the only way to diagnose the condition is through a surgical procedure. 

“It’s hard to believe,” she said. 

Overall, the women Business Insider spoke with emphasized a desire for better education about the condition, both for doctors and for the general public, so that diagnoses aren’t so delayed. 

“I just would love for it not to take seven to 10 years to get a diagnosis,” Kinney said.