PCOS, a common disorder affecting 5-10% of women of reproductive age is characterized by cystic ovarian morphology, androgen excess, and/or irregular periods and emerges during or shortly after puberty. The common 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, clinical trials are needed and requested by EMA to validate the safety and efficacy of this agent in PCOS.
In January 2012, the EMA released a revised priority list of PUMA generic medicines and Metformin was identified as a requested drug for treatment of PCOS in children. The call requests data on pharmacokinetics, efficacy and safety in this indication. A PIP was submitted to EMA in August 2012. In its final opinion of 17 May 2013 the PDCO recommend to agree to the PIP. The PDCO and the consortium have agreed that by restricting exposure to no more than 2000 mg metformin / day, no additional pharmacokinetic investigations are required or even requested by EMA.
METFIZZ will 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.
Currently, there are no age-appropriate dosing forms of metformin for treatment of PCOS in paediatric populations. This can result in over or under dosing, lack of medication compliance, dangers of iatrogenic harms. METFIZZ embraces clinical development, manufacturing scale up, and registration aspects of this pharmaceutical development programme.
Coordinator SME EffRx together with 4 SME-CROs and at least 20-30 related study centres will carry out the clinical study in 4 EU member states. The statistical analysis plan requires approximately 150 patients to be randomized into the PCOS study. EffRx will lead the METFIZZ product commercialisation to benefit children in Europe.
Call for proposal
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Funding SchemeCP-FP - Small or medium-scale focused research project