EU/3/21/2464 - orphan designation for treatment of follicular lymphoma
tisagenlecleucel
OrphanHuman
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Overview
This medicine was designated as an orphan medicine for the treatment of follicular lymphoma in the European Union on 19 July 2021.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
Tisagenlecleucel contains the patient’s own T cells (a type of white blood cell) that have been modified genetically in the laboratory so that they make a protein called chimeric antigen receptor (CAR). CAR can attach to another protein on the surface of cancer cells called CD19.
When the medicine is given to the patient, the modified T cells (called CAR-T cells) attach to and kill the cancer cells, thereby helping to clear the cancer from the body.
At the time of submission of the application for orphan designation:
- The effects of the medicine had been evaluated in experimental models.
- Clinical trials with the medicine in patients with follicular lymphoma were ongoing.
Tisagenlecleucel is authorised in the EU under the name Kymriah for treating 2 types of blood cancer: B-cell acute lymphoblastic leukaemia (ALL) and diffuse large B-cell lymphoma (DLBCL).
More information on how potential new medicines are tested during their development is available on Authorisation of medicines
Medicines intended for rare diseases can be granted an orphan designation during their development.
The orphan designation allows the developer to benefit from:
- scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;
- market exclusivity once the medicine is on the market.
To qualify for orphan designation, a medicine must meet a number of criteria:
- it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
- the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
- there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or the medicine is of significant benefit to those affected by the condition.
EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.
The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
For more information, see:
Key facts
- Active substance
- tisagenlecleucel
- Medicine name
- Kymriah
- Intended use
- Treatment of follicular lymphoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/21/2464
- Date of designation
- Sponsor
Novartis Europharm Limited
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Kymriah at the time of change to the terms of the marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Kymriah : Orphan maintenance assessment report (post authorisation).
Update history
| Date | Update |
|---|---|
| April 2022 | Tisagenlecleucel for treatment of follicular lymphoma has been authorised in the EU as Kymriah since 29 April 2022. |
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: