EU/3/16/1781 - orphan designation for treatment of Merkel cell carcinoma
live-attenuated non-replicative Pseudomonas aeruginosa strain expressing large T antigen of Merkel cell polyomavirus
OrphanHuman
Overview
On 18 November 2016, orphan designation (EU/3/16/1781) was granted by the European Commission to APCure SAS, France, for live-attenuated non-replicative Pseudomonas aeruginosa strain expressing large T antigen of Merkel cell polyomavirus for the treatment of Merkel cell carcinoma.
Merkel cell carcinoma (also known as cutaneous neuro-endocrine carcinoma) is a skin cancer that starts in 'neuro-endocrine' cells called Merkel cells. Neuro-endocrine cells release hormones into the blood when stimulated by the nervous system. Merkel cell carcinoma usually appears as a painless red-blue lump on the head and neck, but it can also be found on the arms and legs, and sometimes on the trunk.
Merkel cell carcinoma is a debilitating and life-threatening condition because it grows rapidly and spreads quickly to other parts of the body. Once this cancer has spread, it is associated with very poor survival.
At the time of designation, Merkel cell carcinoma affected less than 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 21,000 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 513,700,000 (Eurostat 2016).
At the time of designation, no satisfactory methods were authorised in the EU to treat Merkel cell carcinoma. Patients were treated with surgery and radiotherapy (treatment with radiation). Chemotherapy (medicines to treat cancer) was used for cancer that had spread.
The medicine works by stimulating the patient's immune system, the body's natural defences, so that it targets and destroys cancer cells. It is made of Pseudomonas aeruginosa bacteria, which have been attenuated (weakened) so that they do not cause disease and cannot multiply in the body. The bacteria have also been modified to produce the protein Large T (LT) which is found in a majority of Merkel cell carcinomas.
When the medicine is injected into the body, the patient's immune system is activated by the Pseudomonas aeruginosa and learns to treat LT as 'foreign'. This encourages the immune system to attack the cancer cells in Merkel cell carcinoma because they have the LT protein, so reducing the growth of the cancer.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
No clinical trials had been started in patients with Merkel cell carcinoma.
At the time of submission, the medicine was not authorised anywhere in the EU for Merkel cell carcinoma 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 6 October 2016 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
- live-attenuated non-replicative Pseudomonas aeruginosa strain expressing large T antigen of Merkel cell polyomavirus
- Intended use
- Treatment of Merkel cell carcinoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1781
- Date of designation
- Sponsor
APCure SAS
282 rue André Philip
69003 Lyon
France
Telephone: +33 4 26 84 69 63
E-mail: Laurent.Buffat@APCure.com
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:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
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: