The landscape of endometriosis treatment remains remarkably limited despite the condition affecting approximately 10% of women and girls globally, highlighting persistent challenges in women's health research and drug development.
The Burden of Limited Understanding
Endometriosis, characterized by the growth of uterine tissue outside the uterus, continues to pose significant challenges for both patients and healthcare providers. The condition typically manifests as chronic pelvic pain, particularly during menstruation, though its effects can extend throughout the body. Diagnosis often requires invasive laparoscopic surgery, as conventional imaging methods like ultrasound cannot reliably detect abnormal tissue growth.
"[Endometriosis research] has been very underfunded and so, despite the prevalence of this disease, it's not very well understood at all," explains Marina R. Walther-Antonio, an assistant professor of surgery at the Mayo Clinic in Minnesota.
Current Treatment Landscape
The therapeutic options for endometriosis remain notably sparse. Most patients rely on birth control, hormonal therapies, or pain medications to manage symptoms. In the past decade, only two new drugs have received FDA approval for endometriosis-related pain: AbbVie's Orilissa (2018) and Pfizer/Myovant Sciences' Myfembree (2022).
However, these medications come with significant limitations. Both drugs carry list prices exceeding $1,000 per month and can only be used for a maximum of two years due to concerns about bone density loss. Furthermore, they address symptoms rather than the underlying disease mechanism.
"Those options are not widely discussed or celebrated because they're not actually treating the disease," notes Somer Baburek, CEO and co-founder of Hera Biotech. "They are just quieting some of the symptoms that make your life unbearable, but then you get a whole host of other symptoms that also might make your life unbearable."
Research Challenges and Data Gaps
The development of new endometriosis treatments faces multiple obstacles, including:
- Limited biobank resources compared to other conditions
- Historical exclusion of women from clinical trials
- Insufficient funding for women's health research
- Focus primarily on reproductive aspects rather than overall health impact
A 2022 study revealed that women comprised only 41% of participants in Phase 1 through Phase 3 trials for drugs and medical devices between 2016 and 2019 in the U.S., highlighting ongoing representation challenges.
Future Directions and Potential Progress
Recent developments suggest possible momentum in addressing these long-standing issues. The Biden administration's commitment of $100 million to women's health research and ARPA-H's "sprint for women's health" initiative represent significant steps forward.
Experts emphasize the critical need for non-hormonal treatment approaches and better understanding of the disease's mechanisms. "We've got to mechanistically understand [endometriosis] so we know which levers to pull and which knobs to push," Baburek states, highlighting the importance of fundamental research.
Several biotechnology companies, including Gynica, FimmCyte, and SLBST Pharma, are exploring new approaches to endometriosis treatment. However, sustained progress will require continued investment and attention to women's health research priorities.
