Overview

On 16 October 2017, orphan designation (EU/3/17/1923) was granted by the European Commission to Pfizer Limited, United Kingdom, for glasdegib maleate for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to Pfizer Europe MA EEIG, Belgium, in November 2018.

Glasdegib maleate for treatment of acute myeloid leukaemia has been authorised in the EU as Daurismo since 26 June 2020.

Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of abnormal, immature white blood cells. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.

AML is a long-term debilitating and life-threatening disease because these abnormal immature cells take the place of the normal blood cells, causing bleeding episodes, blood clots and a reduced ability to fight infections.

At the time of designation, AML affected approximately 1.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 57,000 people1, 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).

1Disclaimer: 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 515,700,000 (Eurostat 2017).

Treatment for AML 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. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a procedure where the patient's bone marrow is cleared of cells and replaced by stem cells to form new bone marrow that produces healthy blood cells).

The sponsor has provided sufficient information to show that glasdegib maleate might be of significant benefit for patients with AML because early studies found that the cancer had cleared when the medicine was used in combination with the cancer medicines decitabine and cytarabine. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Glasdegib maleate attaches to and blocks a protein called 'SMO'. SMO allows cancer white blood cells to become inactive and this prevents cancer medicines from working. By blocking SMO, glasdegib maleate is expected to cause the cells to become active and so leave them open to the effects of cancer medicines.

The effects of glasdegib maleate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with glasdegib maleate in patients with AML were ongoing.

Glasdegib maleate was not authorised anywhere in the EU for AML. Orphan designation of the medicine had been granted in the United States for AML.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 2017 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/3/17/1923: Public summary of opinion on orphan designation: Glasdegib maleate for the treatment of acute myeloid leukaemia

Reference Number: EMA/623187/2017

English (EN) (113.73 KB - PDF)

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Key facts

Active substance
Glasdegib maleate
Intended use
Treatment of acute myeloid leukaemia
Orphan designation status
Positive
EU designation number
EU/3/17/1923
Date of designation
Sponsor

Pfizer Europe MA EEIG
 

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Daurismo, glasdegib maleate at the time of change to marketing auhorsation, and confirmed that the orphan designation should be maintained.

More information is available in the Daurismo : Orphan maintenance assessment report (initial 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:

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

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

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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