Metformin for treatment of polycystic ovary syndrome in children
Polycystic ovary syndrome (PCOS) is a disorder which affects of women of reproductive age. It is characterized by cystic ovarian morphology, androgen excess, and/or irregular periods. These symptoms emerge during or shortly after puberty and can result in distressing physical and psychosocial effects such as excess body and facial hair, and later be one of the major causes of infertility. While lifestyle changes such as diet and exercise are used for weight reduction, no approved therapeutic options are indicated for treatment of PCOS in adolescent girls.

Metformin is a medicine that is commonly used to treat people with type 2 diabetes and has also been found to help with PCOS symptoms. The off-label use of metformin for treating PCOS in children appears to have no greater risk than that in adults; drug tolerance is good with limited adverse effects. However, currently, there are no pharmaceutical treatments that are indicated for use in PCOS, and importantly there are no age-appropriate dosing forms of metformin for treatment of PCOS in adolescent girls.

Thus, clinical trials are needed to validate the safety and efficacy of this agent in PCOS. In January 2012, the European Medicines Agency (EMA) released a revised priority list of generic (off-patent) paediatric medicinal products and Metformin was identified as a requested drug for treatment of PCOS in children.

The METFIZZ consortium has been selected to conduct the required product development and clinical studies to establish efficacy and safety of administering age-appropriate innovative effervescent soluble formulations of metformin. The studies will focus attention on ethics in this psychologically sensitive condition, guided by the current legislation and by engaging patients and caregivers along with an independent ethics expert.

If clinically successful and approved, the innovative formulation will be a PUMA (Paediatric-Use Marketing Authorisation) candidate.
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METFIZZ fact sheet
METFIZZ is a clinical development project for an effervescent metformin soluble to treat PCOS in adolescent girls. METFIZZ receives funding from the Seventh Framework Programme of the European Union.
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Project start: October 2013
Project end: March 2017

About the Coordinator
EffRx Pharmaceuticals SA is an innovative specialty pharmaceutical company that exploits its proprietary technology platform to create novel therapeutic entities.
EffRx will lead the METFIZZ product commercialisation.

About the clinical study
  • Multiple study centres in 4 EU member states.
  • 150 patients to be randomized into the PCOS study.
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Project Coordinator
Ana Johansson
EffRx Pharmaceuticals SA
CH – 8807 Freienbach (SZ)
Mobile: +45 22 742 734
Project Manager
Kirsten Leufgen
Phone: +41 21 694 04 12
E-mail contact
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© 2013 METFIZZ Project - created by SCIPROM