An overview of early drug development for endometriosis

ABSTRACT

Introduction: Endometriosis is an estrogen-dependent chronic disease of women of fertile age requiring chronic therapy. Although available drugs have good efficacy and safety profiles, some patients experience partial or no improvement of pain with conventional treatment and recurrence of symptoms after discontinuation of the therapies. For these reasons, many new compounds are currently under investigation for the treatment of endometriosis.

Areas covered: This review offers the reader a complete and updated overview on emerging therapies for the treatment of endometriosis. The authors describe, in detail, the laboratory and clinical studies on these therapies and highlight the potential advantages and limitations associated with the administration of these new agents.

Expert opinion: Gonadotropin-releasing hormone antagonists are the most intriguing emerging agents for the treatment of patients with endometriosis. It should be noted that while there are a number of drugs under investigation, a large majority of these new compounds have only been investigated in laboratory studies with more extensive research required to better elucidate their efficacy and safety profiles.

KEYWORDS:

• Elagolix
• endometriosis
• gonadotropin-releasing hormone antagonist
• hormonal treatment
• medical treatment

Article highlights

• Endometriosis is an estrogen-dependent disease of women of fertile age which requires long-term treatment.

• Some patients experience little/no improvement of pain with available medical therapies for endometriosis and recurrence of symptoms after discontinuation of treatment.

• Current research is investigating the development of new drugs targeting pathophysiological mechanisms.

• Phase II trials on gonadotropin releasing hormone antagonists for endometriosis have shown promising results and one of these drugs, elagolix, have the advantage of oral administration.

• Most of the emerging agents for endometriosis have only been investigated in experimental models and more extensive research is required to address their efficacy and safety profiles.

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Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Disclosure statement

No potential conflict of interest was reported by the authors.