EU/3/20/2309 - orphan designation for treatment of idiopathic pulmonary fibrosis
Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2023 on request of the Sponsor.
On 21 August 2020, orphan designation EU/3/20/2309 was granted by the European Commission to Galecto Biotech AB, Denmark, for Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane (also known as TD139) for the treatment of idiopathic pulmonary fibrosis.
Idiopathic pulmonary fibrosis is a disease in which scar tissue (collagen and fibrous tissue) gradually builds up in the lungs, making their structure thicker and less flexible. As a result, the lungs no longer work normally, reducing the amount of oxygen that gets into the blood. Patients with idiopathic pulmonary fibrosis have a persistent cough, frequent lung infections and shortness of breath that worsens over time.
Idiopathic pulmonary fibrosis is a long-term debilitating and life-threatening disease because the lungs gradually lose their ability to work properly.
At the time of designation, idiopathic pulmonary fibrosis affected approximately 3.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 166,000 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).
*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, Esbriet (pirfenidone) and Ofev (nintedanib) were authorised in the EU to treat idiopathic pulmonary fibrosis.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with idiopathic pulmonary fibrosis. Early data suggest that the medicine may be more effective than authorised medicines in patients with IPF.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine blocks the action of a protein called galectin-3, which is involved in the production of fibrous tissue in organs including the lungs, and is thought to play a role in the development of IPF. By blocking the activity of galectin-3, the medicine is expected to reduce build-up of fibrous tissue in the lungs, thereby improving their functioning.
The effects of bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane 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 idiopathic pulmonary fibrosis were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of idiopathic pulmonary fibrosis or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, 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
- Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane
- Intended use
- Treatment of idiopathic pulmonary fibrosis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/20/2309
- Date of designation
- Sponsor
Galecto Biotech AB
Cobis Science Park
Ole Maaloees Vej 3
2200 Copenhagen N Hovedstaden
Denmark
Tel: +45 70705210
E-mail: info@galecto.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: