ImetelChat

29 January 2016
EMA/COMP/777525/2015
Committee for Orphan Medicinal Products

Public summary of opinion on orphan designation - Imetelstat sodium for the treatment of myelofibrosis

On 14 December 2015, orphan designation (EU/3/15/1593) was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for imetelstat sodium for the treatment of myelofibrosis.
The sponsor has provided sufficient information to show that imetelstat sodium might be of significant benefit for patients with myelofibrosis. The medicine works in a different way to existing treatments and early studies showed a reversal of bone marrow fibrosis, which is not achieved by authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

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link to the full summary: http://www.ema.europa.eu/ema/index.jsp? ... w-criteria

Some other info about the approval process in the EU based on ODD and EMA fast track treatment (the following example is about Emplicit, a drug for MM that received ODD first and now fast track treatment):

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Because multiple myeloma is rare, Empliciti received an orphan designation from the Committee for Orphan Medicinal Products (COMP) in 2012. Orphan designation is the key instrument available in the EU to encourage the development of medicines for patients with rare diseases. Orphan-designated medicines qualify for ten years’ market exclusivity. In addition orphan designation gives medicine developers access to incentives, such as fee reductions for marketing authorisation applications and scientific advice.

The applicant received scientific advice from the CHMP pertaining to quality and clinical aspects of the dossier.

The opinion adopted by the CHMP at its January 2016 meeting is an intermediary step on Empliciti’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.
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This provides some insight in how the (fast track) approval process takes place in the EU. Pricing negotians per country are part of that process. That may partly explain why approval in EU takes longer than in the US. It can happen in the EU that a drug is available in country A, but not (yet) in country B.

Link: http://www.ema.europa.eu/ema/index.jsp? ... 058004d5c1

Good info Fisher, thanks for researching this. bp