EU/3/22/2731 - orphan designation for treatment of small cell lung cancer
serplulimab
Orphan
Human
This medicine was designated as an orphan medicine for the treatment of small cell lung cancer in the European Union on 9 December 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:
Serplulimab belongs to the class of: Antibody-dependent cell cytotoxicity; Programmed cell death-1 receptor antagonists; T lymphocyte stimulants
This medicine is a fully humanized IgG4 monoclonal antibody, a type of protein that has been designed to attach to programmed cell death protein 1 (PD-1), a receptor found on T cells (a type of cell in the immune system).
Cancer cells can produce certain proteins, PD-L1 and PD-L2, on their surface. These also attach to the PD-1 receptor and, by doing so, switch off the activity of the T cells and prevent them from attacking the tumour. Serplulimab binds to the PD-1 receptors on T cells without switching off their activity. This blocks the PD-1 receptor so PD-L1 and PD-L2 on the surface of the cancer cells can no longer attach and switch off the T cells. This may help to restore immune function through the activation of T cells against tumour cells.
Based on description provided by sponsor
At the time of submission of the application for orphan designation:
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:
To qualify for orphan designation, a medicine must meet a number of criteria:
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:
Accord Healthcare S.L.U.
Date | Update |
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February 2025 | The sponsorship was transferred to Accord Healthcare SLU from Henlius Europe GmbH. |
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: