EU/3/15/1550 - orphan designation for treatment of idiopathic pulmonary fibrosis

3-pentylbenzeneacetic acid sodium salt

Overview

On 9 October 2015, orphan designation (EU/3/15/1550) was granted by the European Commission to ProMetic BioTherapeutics Ltd, United Kingdom, for 3-pentylbenzeneacetic acid sodium salt for the treatment of idiopathic pulmonary fibrosis.

The sponsorship was transferred to ProMetic Pharma SMT Limited, United Kingdom, in June 2016.

The sponsorship was transferred to Prometic Pharma SMT B.V., The Netherlands, in May 2019.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2022 on request of the Sponsor.

What is idiopathic pulmonary fibrosis?

Idiopathic pulmonary fibrosis is a long-term disease of the lungs characterised by the progressive deposition of collagen and fibrous tissue in the lungs. This causes the lung tissue to become thick and to form scars. As a result, the lungs become unable to work normally, reducing the transfer of oxygen from the air 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.

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

At the time of designation, idiopathic pulmonary fibrosis affected not more than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 154,000 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 512,900,000 (Eurostat 2015).

What treatments are available?

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 studies showed 3-pentylbenzeneacetic acid sodium salt to have more effect in reducing fibrosis than currently authorised products. Its effects were further increased when used in combination with currently authorised products. 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 works by attaching to two receptors that are found in different types of white blood cells and are thought to be involved in inflammation and the development of fibrosis. By attaching to these receptors, the medicine is expected to help reduce inflammation and fibrosis, thereby relieving symptoms of patients with idiopathic pulmonary fibrosis.

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, no clinical trials with the medicine in patients with idiopathic pulmonary fibrosis had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for idiopathic pulmonary fibrosis. Orphan designation had been granted in the United States for the condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 September 2015 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.

Key facts

• Active substance:
  3-pentylbenzeneacetic acid sodium salt
• Intended use:
  Treatment of idiopathic pulmonary fibrosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/15/1550
• Date of designation:
  09/10/2015
• Sponsor:

  Prometic Pharma SMT B.V.; Barbara Strozzilaan 201; 1083 HN Amsterdam; Noord-Holland; Netherlands; Tel: [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.