For the second time since the entry into force of the Paediatric Regulation (Reg. EC 1901/2006), the European Medicines Agency (EMA) has granted a positive opinion for a paediatric-use marketing authorisation (PUMA).

The object of the opinion is Hemangiol, a drug for the treatment of proliferating infantile haemangioma, benign tumours involving blood vessels, that develop in the skin or internal organs. They affect around 3% to 10% of children and, although usually uncomplicated, they can be associated with painful or life-threatening complications. In most European countries, there are currently no approved treatment options, although corticosteroids are authorised for severe cases in France and Germany.

PUMA is a measure introduced by the Paediatric Regulation to incentive research of existing products in children and favour the availability of new drugs duly approved for children. It grants a 10-year period of market protection in case of paediatric development of adult’s medicines that are not protected by a patent or a supplementary protection certificate (off-patent drugs). Prerequisite for a PUMA is a paediatric investigation plan (PIP) which sets out the development of the medicine in children and has to be approved by the Agency’s Paediatric Committee.

The Gianni Benzi Foundation is involved in three research projects (DEEP, CloSed and GAPP) aimed at developing a PUMA for age appropriated new formulations of deferiprone, clonidine and gabapentin, respectively.

As it has been highlighted in the five-year report on the impact of the Paediatric Regulation, PUMA has not been sufficiently attractive for companies: more valuable benefits to protect and incentive the use of the new marketed forms/formulations and a simplified procedure to apply for a PUMA are needed.