EU/3/16/1808 - orphan designation for treatment of systemic sclerosis

(6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid

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Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2023 on request of the Sponsor.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 12 January 2017, orphan designation (EU/3/16/1808) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid (also known as JBT-101 or resunab) for the treatment of systemic sclerosis.

The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in July 2019.

This medicine is now known as lenabasum.

The sponsorship was transferred to Pharma Gateway AB in July 2020.

What is systemic sclerosis?

Systemic sclerosis, also known as scleroderma, is a complex disease in which the immune system (the body's natural defences) is overactive, causing inflammation and excessive production of some proteins, particularly collagen. The reason why the immune system is overactive is not known. Collagen is an important component of connective tissue (the tissue that supports the skin and internal organs).

Overproduction of collagen leads to abnormal growth of connective tissue, causing the skin to become thick and hard. Initial symptoms include swelling of fingers and hands, followed by a thickening of the skin over the arms, legs, face and trunk. The disease can also damage the walls of blood vessels of internal organs such as the heart, lungs and kidneys. This makes it more difficult for the blood to flow, causing tissue damage and circulation problems.

Systemic sclerosis is a long-lasting, debilitating disease and may be life threatening because of its possible effects on the gut, heart, lungs and kidneys.

What is the estimated number of patients affected by the condition?

At the time of designation, systemic sclerosis affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 180,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*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 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of designation, there were no treatments for systemic sclerosis that could stop the build-up of collagen and abnormal growth of connective tissue. Treatments authorised in the EU were aimed at relieving the symptoms of the disease and limiting the damage it causes. Several medicines were used to reduce inflammation and circulation problems. Bosentan has been authorised in the EU specifically to treat patients with systemic sclerosis in whom poor blood circulation caused by the disease has led to the development of digital ulcers (sores on the fingers and toes).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with systemic sclerosis because early laboratory studies show that it may reduce the abnormal growth of connective tissue, an effect not seen with the currently authorised treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine attaches to receptors called cannabinoid type 2 receptors (CB2) found on immune cells. By attaching to these receptors, it is expected to control the body's immune system better in patients with systemic sclerosis, reducing inflammation and abnormal growth of connective tissue, and so improving symptoms of the condition.

What is the stage of development of this medicine?

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 systemic sclerosis were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for systemic sclerosis. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 December 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

EU/3/16/1808: Public summary of opinion on orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid for the treatment of systemic sclerosis

AdoptedReference Number: EMA/14165/2017

Key facts

• Active substance:
  (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid
• Medicine name:
  -
• Intended use:
  Treatment of systemic sclerosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/16/1808
• Date of designation:
  12/01/2017
• 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.