EU/3/22/2599 - orphan designation for treatment of uveal melanoma
Belzupacap sarotalocan
OrphanHuman
Overview
This medicine was designated as an orphan medicine for the treatment of uveal melanoma in the European Union on 13 April 2022.
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:
The medicine is composed of virus-like particles conjugated (attached) to about 200 dye molecules per particle. When the medicine is injected into the eye, the particle attaches to the cancer cells. When red light is then applied to the eye, the dye is activated, leading to death of the cancer cells and release of debris from the dead cancer cells. The released cell debris causes a local immune response, that will kill additional cancer cells both immediately and in the future. Therefore, the drug works in two ways, immediate killing of the cancer cells and activating the immune system.
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 uveal melanoma were ongoing.
More information on how potential new medicines are tested during their development is available on Authorisation of medicines.
Key facts
- Active substance
- Belzupacap sarotalocan
- Intended use
- Treatment of uveal melanoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/22/2599
- Date of designation
- Sponsor
Aura Biosciences B.V.
Hogehilweg 4
1101 CC Amsterdam
Noord-Holland
Netherlands
E-mail: info@aurabiosciences.com
Update history
| Date | Update |
| September 2023 | The sponsorship was transferred from FGK Representative Service GmbH, Germany to Aura Biosciences 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: