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Orphan designation

On 22 May 2006, orphan designation (EU/3/06/375) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for nilotinib for the treatment of chronic myeloid leukaemia.

Nilotinib has been authorised in the EU as Tasigna since 19 November 2007.

What is chronic myeloid leukaemia?

Chronic myeloid leukemia (CML) is a disease in which cancer cells are found in the blood and in the bone marrow. The bone marrow is the spongy tissue inside the large bones in the body. Normally, the bone marrow makes cells called “blasts” that mature into several different types of blood cells that have specific functions in the body. These include red cells, white cells and platelets. Red blood cells carry oxygen and other materials to all tissues of the body. White blood cells fight infection. Platelets make the blood clot. When leukemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukemias. In myeloid leukemia blasts that are developing into white blood cells called granulocytes, are affected. The blasts do not mature and become too many. These blast cells are then found in the blood and also accumulate in the bone marrow. The disease can develop very slowly, which is why it is called “chronic” myeloid leukemia. Chronic myeloid leukemia is life-threatening.

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

At the time of designation, chronic myeloid leukaemia affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 47,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

What are the methods of treatment available?

Treatment for leukemia is complex and depends on a number of factors including the type of leukemia, the extent of the disease and whether the leukemia has been treated before. It also depends on the age, the symptoms, and the general health of the patient. At the time of submission of the application for orphan drug designation authorised treatments of chronic myeloid leukemia included chemotherapy agents (using drugs to kill cancer cells) and immunotherapy agents (using drugs that stimulate the body’s own immune system to kill cancer cells). Sometimes a combination of immunotherapy and chemotherapies were used. Another product is also used which blocks (inhibits) growth signals within cancer cells and prevents a series of chemical reactions that cause the cell to grow and divide thus stopping cancer cells to grow. Bone marrow transplantation was also used.

Nilotinib could be of potential significant benefit for the treatment of chronic myeloid leukaemia mainly because it might improve the long-term outcome of the patients. 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?

Enzymes are proteins produced by the human body that speed up the transformation of certain substances into other substances. Nilotinib blocks (inhibits) a certain class of enzymes called tyrosine kinases. These enzymes play a role in a cascade of molecular reactions that bring a certain signal from outside the cell into the cell thereby controlling the growth of cells. In chronic myeloid leukaemia, the function of these enzymes is disturbed causing uncontrolled growth and multiplication of the cancer cells. Nilotinib might, by inhibition of one or more of these enzymes activity, at certain levels in the cascade, help in slowing down or stopping the further growth of the cancer cells.

What is the stage of development of this medicine?

The effects of nilotinib were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with chronic myeloid leukaemia were ongoing.

Nilotinib was not authorised anywhere worldwide for chronic myeloid leukaemia or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 April 2006 recommending the granting of this designation.

Update: Nilotinib (Tasigna) has been authorised in the EU since <date> for the treatment of adult patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukaemia (CML) in the chronic phase.

More information on Tasigna can be found in the European public assessment report (EPAR) on the Agency’s website.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

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

Key facts

Product details for <p>Nilotinib</p>
Active substanceNilotinib
Medicine NameTasigna
Disease/conditionTreatment of chronic myeloid leukaemia
Date of decision22/05/2006
Orphan decision numberEU/3/06/375

Review of designation

Sponsor’s contact details

Novartis Europharm Limited
Frimley Business Park
Camberley GU16 7SR
United Kingdom
Tel. +41 61 324 11 11 (Switzerland)

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.