EU/3/17/1872 - orphan designation for treatment of cutaneous T-cell lymphoma
chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p
OrphanHuman
Overview
On 22 May 2017, orphan designation (EU/3/17/1872) was granted by the European Commission to Miragen Therapeutics Europe Ltd, United Kingdom, for chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p (also known as MRG-106) for the treatment of cutaneous T-cell lymphoma.
The sponsorship was transferred to miRagen Therapeutics S.à r.l., Luxemburg in May 2019.
The sponsor's name was updated in September 2021.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in June 2022 on request of the Sponsor.
Cutaneous T-cell lymphoma (CTCL) is a cancer of the T lymphocytes (T cells), a type of white blood cell. The cancerous T cells appear in the skin, causing lesions (rashes, plaques and tumours) which can be itchy and painful.
CTCL usually happens in people aged between 40 and 60 years. In many cases, patients survive a long time with the disease; however, in some cases the disease can be serious and life threatening because it can develop into more aggressive forms of cancer and may have a large impact on quality of life, particularly because the skin lesions can cause disfigurement.
At the time of designation, CTCL affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 134,000 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 515,700,000 (Eurostat 2017).
At the time of designation, several products were authorised for the treatment of CTCL in the EU. Treatments for CTCL can be divided into topical (applied directly to the skin) and systemic (affecting the whole body):
- topical treatments included topical corticosteroids, the topical cancer medicine carmustine and ultraviolet light;
- systemic treatments included cytotoxic medicines (medicines that kill cells that are dividing, such as cancer cells) and interferon alfa (a medicine that helps the immune system to fight against the cancer cells).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with CTCL because early studies indicate that it may reduce skin tumour size when used together with other treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to work by blocking a molecule called 'microRNA-155-5p'. Studies have found that microRNA-155-5p is present in high levels inside CTCL cells, where it is thought to regulate their growth and survival. By blocking microRNA-155-5-p, the medicine is expected to reduce growth of CTCL cells, relieving symptoms of the disease.
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 including patients with CTCL were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for CTCL 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 11 April 2017 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
- chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p
- Intended use
- Treatment of cutaneous T-cell lymphoma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1872
- Date of designation
- Sponsor
Viridian Therapeutics S.à r.l.
14 Rue Edward Steichen
2540 Luxembourg
Luxembourg
Tel: +353 21 206 9222
E-mail: info@viridiantherapeutics.com
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: