EU/3/14/1391 - orphan designation for treatment of malignant mesothelioma

5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine
OrphanHuman

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in January 2016 on request of the sponsor.

On 16 December 2014, orphan designation (EU/3/14/1391) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine for the treatment of malignant mesothelioma.

Malignant mesothelioma is a cancer that affects the mesothelial cells (found on the inner linings of the organs), mainly in the pleura (lining the lungs) and in the peritoneum (lining the abdominal cavity). It is usually caused by exposure to asbestos. Mesothelioma of the pleura causes difficulty breathing and chest pain, and mesothelioma of the peritoneum causes ascites (a build-up of fluid in the abdomen) and abdominal pain.

Malignant mesothelioma is life-threatening because it may lead to bowel obstruction, heart or breathing problems and lung infections. Survival is poor, with patients only living, on average, for a year after diagnosis.

At the time of designation, malignant mesothelioma affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 26,000 people*, and is below the ceiling 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

At the time of designation, the main treatment for malignant mesothelioma was surgery followed by chemotherapy (medicines to treat cancer) or radiotherapy (treatment with radiation). If the disease was too advanced for surgery, chemotherapy alone was used. Only one medicine, pemetrexed, was specifically authorised in the EU for the treatment of malignant mesothelioma.

The sponsor has provided sufficient information to show that 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine might be of significant benefit for patients with the condition. Studies in experimental models have shown improved effects of the medicine compared with a combination of pemetrexed and cisplatin (another cancer medicine). This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The medicine works by blocking the activity of 'mammalian target of rapamycin' (mTOR) and 'phosphoinositide-3-kinases' (PI3K), two types of enzymes involved in controlling cell growth. In many cancers including mesothelioma, the biological pathway involving mTOR and PI3K is overactive and enables the uncontrolled growth and spread of cancer cells. By blocking the actions of these enzymes, the medicine is expected to help slow down the growth and spread of the cancer.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with malignant mesothelioma had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for malignant mesothelioma or 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 13 November 2014 recommending the granting of this designation.

  • 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 EU) 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/3/14/1391: Public summary of positive opinion for orphan designation: 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine for the treatment of malignant mesothelioma

Adopted Reference Number: EMA/COMP/744278/2014

English (EN) (164.91 KB - PDF)

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Key facts

Active substance
5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine
Intended use
Treatment of malignant mesothelioma
Orphan designation status
Withdrawn
EU designation number
EU/3/14/1391
Date of designation
Sponsor

TMC Pharma Services Ltd
 

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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