EU/3/18/2070 - orphan designation for treatment of dermatomyositis
(6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2023 on request of the Sponsor.
On 26 October 2018, orphan designation (EU/3/18/2070) was granted by the European Commission to Accelsiors CRO and Consultancy Services Ltd, Hungary, for (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid (also known as lenabasum) for the treatment of dermatomyositis.
The sponsorship was transferred to Pharma Gateway AB, Sweden, in July 2020.
Dermatomyositis is an inflammatory disease of the muscles and the skin which causes muscle weakness and severe skin rash. Although skeletal muscle and skin problems are the most frequent signs of the disease, inflammation may also affect the muscles of the oesophagus (the food pipe that leads from the mouth to the stomach), the lungs and the heart, leading to difficulties in eating and breathing.
Dermatomyositis is an auto-immune disease. This means that it is caused by the body’s immune (defence) system attacking its own tissues. The reason why the immune system acts in this way is not known.
Dermatomyositis is a life-threatening and long-term debilitating condition due to the severe skin problems, muscle weakness and heart problems, and also because patients with the condition are at higher risk of developing cancer.
At the time of designation, dermatomyositis affected approximately 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 67,000 people*, 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).
* Disclaimer: 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 517,400,000 (Eurostat 2018).
At the time of designation, azathioprine and corticosteroid medicines were authorised for dermatomyositis in some EU countries.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with dermatomyositis because early studies showed that it may improve the skin rash in patients who did not respond to other medicines.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine attaches to receptors (targets) called cannabinoid type 2 receptors found on immune system cells. By attaching to these receptors, it is expected to control the activity of the body’s immune system in patients with dermatomyositis, reducing inflammation and so improving symptoms of the condition.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with dermatomyositis were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for dermatomyositis 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 13 September 2018 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
- (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid
- Intended use
- Treatment of dermatomyositis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/18/2070
- Sponsor
Pharma Gateway AB
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: