Meeting Highlights From the Paediatric Committee

October 27, 2008

Company News Release

Opinions on paediatric investigation plans.

Opinions On Paediatric Investigation Plans


The Paediatric Committee (PDCO) adopted positive opinions on paediatric investigation plans (PIPs) for the following medicines:

* Everolimus, from Novartis Europharm Ltd, in the therapeutic area of oncology;
* TGplPTH1-34 (Osteogenic Gel I-040302), from Kuros Biosurgery International AG, in the therapeutic area of endocrinology, gynaecology, fertility and metabolism;
*AEB0713-(1H-indol-3-yl)-4-(2-(4-methyl-1-piperazinyl)-4-quinazolinyl)-1H-pyrrole-2,5-dione acetate(1:1), from Novartis Europharm Ltd, in the therapeutic area of dermatology;
* Alipogene tiparvovec, from Amsterdam Molecular Therapeutics B.V., in the therapeutic area of cardiovascular diseases;
* Human Normal Immunoglobulin, from LFB Biotechnologies, in the therapeutic area of immunology and rheumatology;
* Telbivudine, from Novartis Europharm Limited, in the therapeutic area of gastroenterology;
* Maraviroc, from Pfizer, in the therapeutic area of infectious diseases;
* Tigecycline, from Wyeth Europa Limited, in the therapeutic area of infectious diseases.

The PDCO also adopted a negative opinion on a PIP for sitagliptin phosphate monohydrate- metformin hydrochloride, from Merck Sharp and Dohme Europe, in the therapeutic area of endocrinology, gynaecology, fertility and metabolism. The PDCO subsequently adopted on its own motion a positive opinion on full waiver for this medicine in all subsets of the paediatric population, on the grounds that the specific medicinal product does not represent a significant therapeutic benefit over existing treatments for paediatric patients. A PIP sets out a programme for the development of a medicine in the paediatric population. The PIP aims to generate the necessary quality, safety and efficacy data through studies to support the authorisation of the medicine for use in children of all ages. These data have to be submitted to the EMEA as part of an application for a marketing authorisation for new medicinal products or products covered by a patent. In some cases, a PIP may include a waiver to study one or more age groups of children, or a deferral when it is appropriate to conduct studies in adults prior to initiating studies in the paediatric population, or when studies in the paediatric population would take longer to conduct than studies in adults.

Opinion on PIP modification:
The PDCO adopted an opinion on the modification of an agreed PIP for Clopidogrel, from Sanofi Pharma Bristol-Myers Squibb SNC and from Bristol Myers Squibb Pharma EEIG, in the therapeutic area of cardiovascular diseases, following the adoption of the PDCO opinion on the original PIP during its April 2008 meeting.

Modifications to an agreed PIP can be requested by the applicant when the plan is no longer appropriate or there are difficulties that render the plan unworkable.

Opinions on product-specific waivers:
The PDCO adopted positive opinions for product-specific waivers recommending that the obligationto submit data obtained through clinical studies with children be waived in all subsets of the paediatric population for the following medicines:

* Lenalidomide, from Celgene Europe Limited, in the therapeutic area of oncology;
* Ibuprofen - diphenhydramine hydrochloride, from Wyeth Consumer Healthcare, in the therapeutic area of pain;
* HIN1, H3N2, and B vaccine, from MedImmune, LLC, in the therapeutic area of vaccines;
* Omega-3-acid (ethyl esters of eicosapentaenoic acid (EPA) - docosahexaenoic acid (DHA)) - simvastatin, from Sigma-Tau SpA, in the therapeutic area of cardiology, endocrinology and metabolism.

Waivers can be issued if there is evidence showing that the medicinal product concerned is likely to be ineffective or unsafe in the paediatric population, or that the disease or condition targeted occurs only in adult populations, or that the medicinal product does not represent a significant therapeutic benefit over existing treatments for paediatric patients.

Guideline on neonates:
The PDCO reviewed the comments made during the public consultation on the guideline on the investigation of medicinal products in preterm and term neonates. The guideline aims to provide guidance for the development of medicines for use in neonates. It is based on several concept papers released by the Paediatric Working Party (PEG) - the Agency's former expert working party on paediatric medicines, which addressed the impact of immaturity of different organ systems when investigating medicines in neonates.

Neonates represent a particularly vulnerable subgroup of the paediatric population. Whilst they account for a low percentage of the total use of medicines in childhood, up to 90% of medicines are used without a marketing authorisation or off-label in this population.

Interaction with experts:
The PDCO continued its interaction with academic experts with a view to bring state-of-the-art knowledge to the PDCO scientific discussions in the area of pulmonary arterial hypertension in children, paediatric rheumatology and anti-infectives.

The next meeting of the PDCO will be held on 12-14 November 2008.

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