EU/3/07/467 - orphan designation for treatment of idiopathic thrombocytopenic purpura

eltrombopag olamine

Overview

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in January 2012 on request of the sponsor.

On 3 August 2007, orphan designation (EU/3/07/467) was granted by the European Commission to GlaxoSmithKline Research & Development Limited, United Kingdom, for eltrombopag olamine for the treatment of idiopathic thrombocytopenic purpura (ITP). The sponsorship was transferred to GlaxoSmithKline Trading Services Limited, Ireland, in August 2008.

Eltrombopag olamine has been authorised in the EU as Revolade since 11 March 2010.

What is idiopathic thrombocytopenic purpura?

Thrombocytopenic purpura is a disease where the individual's defence system (the so-called immune system) starts to react against certain of his/her own blood cells that are involved in the blood clotting process, the so-called platelets. ITP is called “idiopathic” since the reason why the body starts reacting against its own cells is unknown. As a result, there will be fewer platelets present in the blood (thrombocytopenia), resulting in spontaneous bleeding and bruising of the skin in purple spots (this phenomenon is called purpura). ITP can be acute or chronic, and can occur in both children and adults. The severity of the bruising is determined by the degree of thrombocytopenia; it can vary from just small skin spots (petechiae) that occur after small injuries to spontaneous blood losses (haemorrhages) from the nose, in the gut or in the brain, which can be life threatening.

What is the estimated number of patients affected by the condition?

At the time of designation, idiopathic thrombocytopenic purpura affected between 1 and 4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of between 50,000 and 199,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 498,000,000 (Eurostat 2006).

What treatments are available?

Treatment of ITP depends on the form of the disease (acute or chronic) and on the age of onset. Current treatment methods include surgery, consisting of spleen removal (splenectomy), or medicines. Splenectomy is performed in order to limit the destruction of the platelets, since the spleen is the most important organ where old platelets are removed from the blood. Several types of medicines were authorised in the Community for the treatment of ITP at the time of submission of the application for orphan drug designation.

Eltrombopag olamine could be of potential significant benefit for the treatment of idiopathic thrombocytopenic purpura because it may act in a different way to other available medicines. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Platelets are cells that derive from a parent cell in the bone marrow, the so-called progenitor cells which develop into platelets through several cell divisions. Eltrombopag olamine is a medicine that might increase the number of platelets by stimulating their production from the progenitor cells.

What is the stage of development of this medicine?

The effects of eltrombopag olamine were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with idiopathic thrombocytopenic purpura were ongoing.

At the time of submission of the application, eltrombopag olamine was not authorised anywhere in the world for idiopathic thrombocytopenic purpura, nor designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 27 June 2007 recommending the granting of this designation.

Update: Eltrombopag olamine (Revolade) has been authorised in the EU since 11 March 2010 for adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Revolade may be considered as second-line treatment for adult non-splenectomised patients where surgery is contraindicated.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the Community) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/07/467: Public summary of positive opinion for orphan designation of eltrombopag olamine for the treatment of idiopathic thrombocytopenic purpura

AdoptedReference Number: EMEA/COMP/287945/2007 Rev.3

Key facts

• Active substance:
  eltrombopag olamine
• Medicine name:
  Revolade
• Intended use:
  Treatment of idiopathic thrombocytopenic purpura
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/07/467
• Date of designation:
  03/08/2007
• Sponsor:

  GlaxoSmithKline Trading Services Limited

Review of designation

Please note that this product (marketed as Revolade) was withdrawn from the Community register of designated orphan medicinal products in January 2012 on request of the sponsor, after the granting of a marketing authorisation.

During its meeting of 5-6 January 2010, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/07/467 for Revolade (eltrombopag olamine) as an orphan medicinal product for the treatment of idiopathic thrombocytopenic purpura. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition and the existence of other satisfactory methods of treatment. As other satisfactory methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained*.

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*The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.