Latest orphan designations and/or marketing authorisations

Medicinal products intended for rare diseases can receive an “orphan drug” label based on a defined number of criteria as summarised below:

• The product is intended for an indication with a prevalence not exceeding 5 in 10 000 persons in the EU.
• The disease is life-threatening, seriously debilitating or a serious and chronic condition.
• No satisfactory method of diagnosis, prevention or treatment of the condition is already authorised in the EU. If any method exists, then the medicinal product has to demonstrate that it provides a significant benefit compared to the product for which orphan designation is being requested.

Committee for Orphan Medicinal Products

A positive opinion on orphan designation is given by the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) based in London with the final decision made by the European Commission.

The COMP was created as part of the EU Regulation on Orphan Medicinal Products to review EU applications for the label "orphan drug”.

Orphan designation at any stage of development

Orphan designation is possible at any stage of drug development as long as proper scientific justification of the medical plausibility of the product in the indication requested is demonstrated.

The research may be pre-clinical (not yet tested on human subjects) or may have reached the human clinical trial phase.

Orphan designation does not indicate an endorsement for the use of the drug for the designated condition. Efficacy, safety and quality criteria first need to be satisfied for the granting of a marketing authorisation.

In addition to the position of Vice-Chair, EURORDIS has held two of the three seats assigned to patient organisation representatives on the COMP since its creation in 2000.

Detailed information on European orphan drug designation applications is available on theEMA website

A full list of designated and authorised orphan drugs in Europe available at: ec.europa.eu

6 June 2012

Treatment of meningioma

N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide

Treatment of schwannoma

N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide

Treatment of Wiskott-Aldrich syndrome

Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene

Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity

Letermovir

Treatment of pancreatic cancer

Polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine

Treatment of ovarian cancer

1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl) urea

Treatment of glioma

Adenovirus-associated vector containing human Fas-c gene

Treatment of adrenoleukodystrophy

Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA

25 May 2012

Treatment of soft tissue sarcoma

Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10

Source

EURORDIS

Online access to suspected side-effect reports

The European Medicines Agency has today begun publishing suspected side effect reports for medicines authorised in the European Economic Area (EEA) on a new public website: www.adrreports.eu. 

The reports come directly from the European Union (EU) medicines safety database EudraVigilance, and are one of the many types of data used by regulators to monitor the benefits and risks of a medicine once authorised. The launch of the new website is part of the Agency’s continuing efforts to ensure EU regulatory processes are transparent and open and is a key step in the implementation of the EudraVigilance access policy.

The information published today relates to approximately 650 medicines and active substances authorised through the centralised procedure, which is managed by the Agency. Information on the website is presented in the form of a single report per medicine or active substance. Each report pulls together the total number of individual suspected side effect reports submitted to EudraVigilance by Member States and marketing-authorisation holders. These aggregated data can be viewed by age group, sex, type of suspected side effect and by outcome. Within a year the Agency aims to additionally publish suspected side effect reports for common drug substances used in nationally authorised medicines.

A side effect (also known as an adverse drug reaction) includes side effects arising from use of a medicine within the terms of the marketing authorisation as well as from use outside the terms of the marketing authorisation, including overdose, misuse, abuse and medication errors, and those associated with occupational exposure.

All information on the website relates to suspected side effects. Suspected side effects may not be related to or caused by the medicine, and as a result, the published information cannot be used to determine the likelihood of experiencing a side effect or as an indication that a medicine is harmful. All users of the website are asked to read and accept a disclaimer explaining how to understand the information before they view a web report.

Medicines are an important part of modern healthcare, providing effective treatments for many diseases and conditions. For a medicine to be authorised for use in the EU the benefits of the medicine must always outweigh the risks.

Today’s launch also highlights the importance of side effect reporting and pharmacovigilance in safeguarding public health within the EU. Side-effect reporting is a key element in ensuring the detection of new or changing safety issues, and the Agency continues to further strengthen its work with partners and stakeholders across Europe to ensure a robust system for safety signal detection.

In June, the Agency will launch the website in the remaining 22 official EU languages.

Notes

• The European database of suspected adverse drug reaction reports:http://www.adrreports.eu
• Patients, consumers and healthcare professionals report suspected side effects to either the national medicines regulatory authority or thepharmaceutical company that holds the marketing authorisation for the medicine. These reports are then transmitted electronically toEudraVigilance. The Agency, on behalf of European Member States, is responsible for the development, maintenance and coordination of EudraVigilance.
• Pharmaceutical companies that hold the marketing authorisation for a medicine in the European Economic Area (EEA) are also legally required to submit to EudraVigilance all reports of suspected unexpected adverse reactions that are serious and that occurred in a third country (non-EEA) where they hold a marketing authorisation. The web reports present information on suspected side effects that have occurred in both the EEA and outside the EEA.  
• A web report does not include reports from studies (e.g. clinical trials and non-interventional studies) or other types of reports, i.e. they only include spontaneous reports.
• Web reports can only be viewed with Adobe Reader 10.x and Adobe FlashPlayer 10.2.
• Frequently asked questions and a short guide on how to interpret web reports can be found online.
• The Management Board of the European Medicines Agency approved the EudraVigilance access policy in December 2010. The access policy describes how stakeholders, such as national medicines regulatory authorities in EEA countries, healthcare professionals, patients and consumers, as well as marketing authorisation holders and research organisations, can access information on suspected side effects submitted electronically to EudraVigilance.
• More information on the 2010 pharmacovigilance legislation can be found on the Agency’s website.

Source

http://www.adrreports.eu/
EMA