FDA approval of gonadotropin-releasing hormone (GnRH) antagonist elagolix as an oral agent for management of moderate-to-severe pain associated with endometriosis provides an important treatment option for a condition that compromises quality of life for approximately 10% of women of reproductive age. Types of pain associated with endometriosis include painful periods, painful ovulation, pain during or after sexual intercourse and chronic pelvic pain.
Clinical studies that provided a basis for FDA approval demonstrated that three out of four women given 200 mg of elagolix twice a day experienced a significant reduction in endometriosis-associated pain. A lower dose, 150 mg once daily, also provided therapeutic benefit. FDA approved the higher dose for use up to six months and the lower dose for use up to 24 months.
While the approval of elagolix provides an important new treatment option for endometriosis-associated pain, coverage determinations by healthcare payers may initially limit availability of elagolix for many patients. Furthermore, OB/GYNs and endometriosis patients could benefit from the availability of additional new therapeutic agents for this painful condition that can also impair fertility. A 2012 review of investigational drugs for endometriosis noted that most were still in the laboratory or early clinical development and that “…further clinical research is required in order to elucidate their efficacy and safety in humans.”
For those planning clinical trials of investigational endometriosis drugs, a recently published fact sheet summarizes Health Decisions’ capabilities for conducting endometriosis studies.