EU/3/17/1954 - orphan designation for treatment of mantle cell lymphoma
venetoclax
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2025 on request of the Sponsor.
On 12 December 2017, orphan designation (EU/3/17/1954) was granted by the European Commission to Abbvie Ltd., United Kingdom, for venetoclax for the treatment of mantle cell lymphoma.
The sponsorship was transferred to AbbVie Deutschland GmbH & Co. KG, Germany, in May 2018.
Mantle cell lymphoma is an aggressive cancer of a type of white blood cell called B lymphocytes, or B cells. In mantle cell lymphoma, the B cells multiply quickly and live for too long, so they build up in the lymph nodes. The first sign of the disease is usually a lump in the neck, under the arm or in the groin, caused by an enlarged lymph node. Patients may also have fever, weight loss, tiredness and night sweats. Mantle cell lymphoma is usually diagnosed in people aged over 50 years. It is more common in men than in women.
Mantle cell lymphoma is a long-term debilitating and life-threatening disease associated with poor survival.
At the time of designation, mantle cell lymphoma affected less than 0.6 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 31,000 people1/, 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).
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).
At the time of designation, the main treatments for mantle cell lymphoma included chemotherapy (medicines to treat cancer), immunotherapy (medicines that act on the body's immune system) and radiotherapy (treatment with radiation). Bortezomib, ibrutinib, lenalidomide and temsirolimus were specifically authorised in the EU for the treatment of mantle cell lymphoma that has come back after previous treatment or has not responded to other treatments. Haematopoietic (blood) stem-cell transplantation was also used. This is a procedure where cells in the patient's bone marrow are replaced by stem cells to form new bone marrow that produces healthy blood cells.
The sponsor has provided sufficient information to show that venetoclax might be of significant benefit for patients with mantle cell lymphoma. This is because early studies show that venetoclax might improve the outcome of patients whose disease has come back or not improved with previous treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Venetoclax is expected to work by blocking proteins called Bcl-2. These proteins prevent the natural process that leads to cell death (apoptosis). Bcl-2 proteins can be found in high levels in cancer cells. By blocking the action of these proteins, the medicine is expected to make cancer cells more responsive to the natural process that causes their death, and this can slow down the growth 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, clinical trials with venetoclax in patients with mantle cell lymphoma were ongoing.
At the time of submission, the medicine was authorised in the EU as Venclyxto for chronic lymphocytic leukaemia.
Venetoclax was not authorised anywhere in the EU for mantle cell lymphoma 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 31 October 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.
Key facts
- Active substance
- venetoclax
- Intended use
- Treatment of mantle cell lymphoma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1954
- Date of designation
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:
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: