EU/3/20/2261: Orphan designation for the treatment of chronic myeloid leukaemia

Chronic myeloid leukaemia (CML) is a cancer of the white blood cells (cells that fight against infections). In patients with CML, the bone marrow (the spongy tissue inside the large bones) produces large numbers of abnormal white blood cells, called blasts, that are not fully developed and do not work properly. Gradually these blast cells replace the normal blood cells in the bone marrow. The disease usually develops very slowly, which is why it is called ‘chronic’. However, when it progresses, CML is a severe and life-threatening disease that is associated with poor overall survival. CML is most common in adults and older people, but children may also be affected.

At the time of designation, CML affected approximately 1.2 in 10,000 people in the European Union (EU). This is equivalent to a total of around 62,000 people*, and is below the ceiling for orphan designation, which is 5 in 10,000 people. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of designation, authorised treatments included various types of cancer medicines, including tyrosine kinase inhibitors (medicines that block enzymes called tyrosine kinases) and immunotherapy (medicines that stimulate the body’s immune system to target cancer cells). Patients might also receive stem cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to restore the bone marrow and produce healthy blood cells).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with CML because early results showed that asciminib had an effect on the disease in patients who did not respond to other available therapies.

This assumption will need to be confirmed at the time of marketing authorisation in order to maintain the orphan status.

Asciminib is a tyrosine kinase inhibitor. It blocks the action of BCR-ABL, a tyrosine kinase produced by CML cells that helps them to grow and reproduce uncontrollably. By blocking BCR-ABL in this way, the medicine is expected to control the growth of the leukaemia cells. 

The effects of asciminib have been evaluated in experimental models.

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

At the time of submission, asciminib was not authorised anywhere in the EU for the treatment of chronic myeloid leukaemia. Orphan designation of asciminib had been granted in the United States for chronic myelogenous leukaemia.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 February 2020, 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.