Ema Approves First Paediatric-use Marketing Authorisation

Earlier this month the EMA (European Medicines Agency) approved its first Paediatric-Use Marketing Authorisation (PUMA).

A PUMA is a new type of Marketing Authorisation that can be granted to Medicines developed exclusively for use in Children. Aimed at encouraging research into paediatric medicines, a PUMA grants a previously authorised drug, which is now off patent, 10 years of intellectual property protection.

To gain approval the medicine must be developed by following a Paediatric Investigation Plan (PIP), which is agreed by a Paediatric committee and must discuss all paediatric subsets.

The first drug to receive approval is ViroParmas Buccolam (midazolam oromucosal solution). In adults, midazolam, as the injectable hydrochloride salt solution, had therapeutic indications for sedation and anaesthesia. In children, Buccolam is a treatment for prolonged, acute, convulsive seizures in infants, toddlers, children and adolescents, from 3 months to less than 18 years of age. The solution, which is absorbed through the lining of the mouth, offers a far more appealing delivery route than the current treatment of diazepam suppositories.

The PUMA is one of the ways that the EMA is looking to stimulate research into medicines for children. The need for this is obvious when you consider that in the EU 50% or more of the drugs prescribed to children have never been studied in this population or even for the same indication or disease. Another incentive offered is that, once a drug is authorised, if companies include the results of the PIP in the patient information then the patent life is extended by 6 months. This is true even if the tests are negative. Following the change in legislation that came into effect on 26th of January 2007, all new medicines in development must include the results from studies on children. This is also true when a company wishes to register a new indication, Pharmaceutical form or route of administration to an existing drug.

Earlier this month the EMA (European Medicines Agency) approved its first Paediatric-Use Marketing Authorisation (PUMA).

A PUMA is a new type of Marketing Authorisation that can be granted to Medicines developed exclusively for use in Children. Aimed at encouraging research into paediatric medicines, a PUMA grants a previously authorised drug, which is now off patent, 10 years of intellectual property protection.

To gain approval the medicine must be developed by following a Paediatric Investigation Plan (PIP), which is agreed by a Paediatric committee and must discuss all paediatric subsets.

The first drug to receive approval is ViroParmas Buccolam (midazolam oromucosal solution). In adults, midazolam, as the injectable hydrochloride salt solution, had therapeutic indications for sedation and anaesthesia. In children, Buccolam is a treatment for prolonged, acute, convulsive seizures in infants, toddlers, children and adolescents, from 3 months to less than 18 years of age. The solution, which is absorbed through the lining of the mouth, offers a far more appealing delivery route than the current treatment of diazepam suppositories.

The PUMA is one of the ways that the EMA is looking to stimulate research into medicines for children. The need for this is obvious when you consider that in the EU 50% or more of the drugs prescribed to children have never been studied in this population or even for the same indication or disease. Another incentive offered is that, once a drug is authorised, if companies include the results of the PIP in the patient information then the patent life is extended by 6 months. This is true even if the tests are negative. Following the change in legislation that came into effect on 26th of January 2007, all new medicines in development must include the results from studies on children. This is also true when a company wishes to register a new indication, Pharmaceutical form or route of administration to an existing drug.

In October 2010, the EMA announced that it had received its 1000th application for a Paediatric Information Plan.