EU/3/13/1234 - orphan designation for treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma

N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2015 on request of the sponsor.

On 16 March 2014, orphan designation (EU/3/13/1234) was granted by the European Commission to Celgene Europe Limited, United Kingdom, for N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt for the treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is chronic lymphocytic leukaemia / small lymphocytic lymphoma?

Chronic lymphocytic leukaemia (CLL) is cancer of a type of white blood cells called B lymphocytes. In this disease, the lymphocytes multiply too quickly and live for too long, so that there are too many of them circulating in the blood. The cancerous lymphocytes look normal, but they are not fully developed and do not work properly. Over a period of time, the abnormal cells replace the normal white cells, red cells and platelets (components that help the blood to clot) in the bone marrow (the spongy tissue inside the large bones in the body, where blood cells are produced). The disease known as 'small lymphocytic lymphoma' (SLL) is essentially the same disease as CLL. The name SLL is normally used when the cancer cells are located mainly in the lymph nodes.

CLL/SLL mainly affects older people and is rare in people under the age of 40 years. It is a long-term debilitating and life-threatening disease because it involves the weakening of the immune system (the body's natural defences), which can leave patients vulnerable to severe infections.

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

At the time of designation, CLL/SLL affected approximately 3.8 to 4.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 195,000 to 241,000 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).

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* 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.
At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).

What treatments are available?

Treatment for CLL/SLL is complex and depends on a number of factors, including the extent of the disease, whether it has been treated before, and the patient's age, symptoms and general state of health. Patients whose CLL/SLL is not causing any symptoms or is only getting worse very slowly may not need treatment. Treatment for CLL/SLL is only started if symptoms become troublesome. At the time of designation, the main treatment for CLL/SLL was chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with CLL/SLL because early studies showed that the medicine was beneficial for patients whose disease did not respond or had come back after treatment with currently authorised medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine is expected to work in patients with CLL/SLL by blocking the action of an enzyme called Bruton's tyrosine kinase (BTK), which is mostly found in B lymphocytes. BTK is important for the growth and survival of B lymphocytes and their migration to the organs where these cells normally divide. By blocking BTK, the medicine is expected to slow down the migration of B lymphocytes in CLL/SLL and to induce cell death, thereby slowing the progression of the disease.

The medicine is expected to be taken by mouth.

What is the stage of development of this medicine?

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

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with CLL/SLL were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for CLL/SLL 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 12 December 2013 recommending the granting of this designation.

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 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/3/13/1234: Public summary of opinion on orphan designation: N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt for the treatment of chronic lymphocytic leu...

Adopted Reference Number: EMA/COMP/9979/2014 Rev.1

Key facts

• Active substance:
  N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt
• Intended use:
  Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/13/1234
• Date of designation:
  16/03/2014
• Sponsor:

  Celgene Europe Limited; 1 Longwalk Road; Stockley Park; Uxbridge; Middlesex UB11 1DB; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]

Review of designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2015 on request of the sponsor, before marketing authorisation had been granted.