EU/3/21/2481 - orphan designation for treatment of diffuse large B-cell lymphoma
Loncastuximab tesirine
Orphan
Human
Overview
This medicine was designated as an orphan medicine for the treatment of diffuse large B-cell lymphoma in the European Union on 20 August 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:
Please note that this product (marketed as Zynlonta) was withdrawn from the Union Register of orphan medicinal products by the European Commission in November 2022 at the time of the granting of a marketing authorisation.
Loncastuximab tesirine is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody, directed against human cluster of differentiation 19 (CD19) and conjugated to a pyrrolobenzodiazepine (PBD) dimer cytotoxin. Loncastuximab tesirine targets CD19-positive B-cell cancer cells. Once bound to CD19, loncastuximab tesirine is internalized and the linker is cleaved releasing free PBD dimers inside the target cell. The PBD dimers are anticancer medicines that induce cell death.
Based on description provided by sponsor
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 diffuse large B-cell lymphoma were ongoing.
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, if such a method exists, 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
- Loncastuximab tesirine
- Intended use
- Treatment of diffuse large B-cell lymphoma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/21/2481
- Date of designation
- Sponsor
ADC Therapeutics (NL) B.V
Laarderhoogtweg 25
Amsterdam
Noord-Holland
1101 EB
Netherlands
Email: info@adctherapeutics.com
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of loncastuximab tesirine (Zynlonta) at the time of marketing authorisation, and recommended that the orphan designation should not be maintained.
More information is available in the Zynlonta : EPAR - Orphan Maintenance Assessment Report (initial authorisation).
Update history
| Date | Update |
|---|---|
| November 2022 | Product withdrawn from the Union Register of orphan medicinal products by the European Commission |
| April 2022 | The sponsorship was transferred to ADC Therapeutics (NL) B.V., Netherlands. |
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: