European Medicines Agency recommends approval of 44 medicines for human use and six medicines for veterinary use in first half 2013

News 31/07/2013

The European Medicines Agency had a busy first half of the year with 44 medicines for human use being recommended for marketing authorisation by the Committee for Medicinal Products for Human Use (CHMP) compared with 33 by the same time last year.

While the number of generics is stable, the number of medicines containing new active substances is higher than last year, with 20 medicines containing active substances that had never been used in medicines before.

Almost one in every two applicants received scientific advice from the Agency's CHMP during the development phase of their medicines. Scientific advice allows companies to get advice on the appropriate studies to be conducted in the development of their medicines and is designed to facilitate the development and availability of high-quality, effective and acceptably safe medicines, for the benefit of patients.

Seeking and following scientific advice has been proven to significantly increase the probability of a positive outcome. A study has shown that the approval rate is 90% when sponsors comply with scientific advice compared with 30% when they do not*.

Eight new medicines for the treatment of cancer

During the first half of 2013, eight new cancer medicines were recommended for marketing authorisation by the CHMP. Of these, seven are oral, meaning they can be used by patients at home. Six of the new medicines are considered to be targeted therapies, which are designed to block the activity of specific proteins involved in tumour growth. The other two medicines act on the patient's immune system. One of them is an advanced-therapy medicinal product (ATMP), the cell-based immunotherapy Provenge. Both targeted therapies and immunotherapies are considered to be better tolerated than traditional cytotoxic chemotherapies.

Particularly noteworthy in the other therapeutic areas are the recommendations for marketing authorisation of three new medicines for the treatment of multiple sclerosis. Drug development in this area is particularly active. Another medicine for this indication is currently under evaluation by the CHMP.

Special regulatory pathways to facilitate market access

The Agency has mechanisms in place - conditional marketing authorisation, approval under exceptional circumstances and accelerated assessment - to facilitate or accelerate market access for medicines that fulfil unmet medical needs or that could not be made available under a standard approval because the disease they target is too rare and comprehensive data cannot be provided. Medicines that undergo any of these pathways are only recommended for marketing authorisation if they can demonstrate that their benefits outweigh their risks in the targeted patient population.

In the first half of 2013, four medicines went through these special regulatory procedures and, pending the European Commission's decision, will be available to patients. They include two anticancer agents (Bosulif and Iclusig), a smallpox vaccine (Imvanex) and a medicine intended for a very rare condition, homozygous familial hypercholesterolaemia (Lojuxta).

Negative opinions and withdrawals

Between January and June 2013, the CHMP adopted negative opinions for the marketing authorisation of four medicines.

Over the same period, seven applications for initial evaluation were withdrawn by the sponsors prior to CHMP opinion.

Number of applications received

During the first half of 2013, the Agency started the assessment of 36 applications for marketing authorisation for initial assessment, including 30 for new medicines.

Among these applications are one paediatric-use-marketing-authorisation (PUMA) application and five designated orphan medicines. For one of these orphan medicines, an ATMP consisting of heterologous human adult liver-derived progenitor cells, the Committee for Orphan Medicinal Products (COMP) recommended granting seven orphan designations at its June 2013 meeting, covering a range of rare diseases.

Veterinary medicines

In the first half of 2013, the Agency's Committee for Medicinal Products for Veterinary Use (CVMP) issued six positive opinions for new veterinary medicines, including the first vaccine for foot-and-mouth disease for authorisation at European Union level. This is also the first vaccine using the full multistrain-dossier approach, a concept that was established in 2010 to allow the swift availability of suitable vaccines in case of outbreaks of major livestock diseases.

The CVMP validated 11 marketing-authorisation applications for new veterinary medicines, including four for food-producing animals.

So far this year, the CVMP has received 19 requests from the industry for scientific advice and responded to 15. Similarly to medicines for human use, scientific advice in the veterinary area is designed to facilitate the development and availability of high-quality, effective and acceptably safe medicines.

The CVMP has also recommended minor-use-minor-species (MUMS) classification for 13 veterinary products. This is intended to stimulate development of new veterinary medicines for minor species and for rare diseases in major species that would otherwise not be developed under current market conditions. Since its introduction in October 2010, this policy has been successful in terms of increasing interest from the animal-health industry in submitting applications for MUMS products, with over 70 products classified so far.

The trend observed over the last few years towards an increase in the number of referral procedures received is confirmed for 2013, with nine referrals submitted so far this year, compared with a total of 12 in both 2012 and 2011.

The table below provides an overview of key figures for the first half of 2013:

Figures for first half of 2013

*Regnstrom J, Koenig F, Aronsson B, Reimer T, Svendsen K, Tsigkos S, Flamion B, Eichler HG, Vamvakas S. Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency. Eur J Clin Pharmacol. 2010 66(1): 39-48.

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