EU/3/19/2246 - orphan designation for treatment of systemic sclerosis
romilkimab
Orphan
Human
Overview
On 9 January 2020, orphan designation EU/3/19/2246 was granted by the European Commission to Sanofi-Aventis Groupe, France, for romilkimab for the treatment of systemic sclerosis.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2021 on request of the Sponsor.
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.
Collagen is an important component of connective tissue (the tissue that supports the skin and internal organs). Overproduction 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 can be life threatening because of its possible effects on the heart, lungs and kidneys.
At the time of designation, systemic sclerosis affected less than 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 181,000 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 518,400,000 (Eurostat 2019).
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 was authorised in the EU 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) and patients with pulmonary arterial hypertension secondary to systemic sclerosis.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with systemic sclerosis. Preliminary data suggest that the medicine might be able to reduce thickening of the skin; when used in combination with standard treatment to reduce inflammation, the medicine was effective in the most severe form of the condition (diffuse cutaneous systemic sclerosis).
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Romilkimab is a monoclonal antibody, a type of protein, designed to attach to 2 different components of the immune system called IL-4 and IL-13. These components are involved in the inflammation and excessive production of collagen in patients with systemic sclerosis. By binding to IL?4 and IL-13, the medicine is expected to relieve the symptoms of the disease and slow down its progression.
The effects of romilkimab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with romilkimab in patients with systemic sclerosis were ongoing.
At the time of submission, romilkimab was not authorised anywhere in the EU for the treatment of systemic sclerosis. Orphan designation of romilkimab had been granted in the United States for the condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 5 December 2019, 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
- romilkimab
- Intended use
- Treatment of systemic sclerosis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/19/2246
- Date of designation
- Sponsor
Sanofi-Aventis Groupe
54 Rue La Boetie
75008 Paris
France
Tel. +33 1697 45699
E-mail: question@sanofi.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:
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: