Investigational drugs for the treatment of endometriosis, an update on recent developments

ABSTRACT

Introduction: Endometriosis is a hormone-dependent benign chronic disease that requires a chronic medical therapy. Although currently available drugs are efficacious in treating endometriosis-related pain, some women experience partial or no improvement. Moreover, the recurrence of symptoms is expected after discontinuation of the therapies. Currently, new drugs are under intense clinical investigation for the treatment of endometriosis.

Areas covered: This review aims to offer the reader a complete and updated overview on new investigational drugs and early molecular targets for the treatment of endometriosis. The authors describe the pre-clinical and clinical development of these agents.

Expert opinion: Among the drugs under investigation, late clinical trials on gonadotropin-releasing hormone antagonists (GnRH-ant) showed the most promising results for the treatment of endometriosis. Aromatase inhibitors (AIs) are efficacious in treating endometriosis related pain symptoms but they cause significant adverse effects that limit their long-term use. New targets have been identified to produce drugs for the treatment of endometriosis, but the majority of these new compounds have only been investigated in laboratory studies or early clinical trials. Thus, further clinical research is required in order to elucidate their efficacy and safety in human.

KEYWORDS:

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

Article highlights

• Although currently available drugs are efficacious in treating endometriosis-related pain, some women experience partial or no improvement. Currently, new hormonal and not hormonal drugs are under intense clinical investigation for the treatment of patients with endometriosis.

• Among hormonal drugs, gonadotropin-releasing hormone antagonists (such as elegolix) obtained the most promising results for the treatment of endometriosis. Moreover, aromatase inhibitors demonstrated to be efficacious in treating endometriosis related pain symptoms even if may cause significant adverse effects that limit their long-term use.

• New targets have been identified for the treatment of endometriosis. Anyway, the majority of these new investigational compounds (anti-angiogenetic, anti-oxidant, anti-inflammatory and immunomodulatory drugs) have only been studied in pre-clinical studies or early clinical trials. Thus, further clinical research is required in order to elucidate their efficacy and safety in human.

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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.

Additional information

Funding

This paper was not funded.