EU/3/18/2056 - orphan designation for treatment of idiopathic pulmonary fibrosis

(S)-(−)-3-(4-aminophenyl)-2-methoxypropanoic acid

Overview

On 24 August 2018, orphan designation (EU/3/18/2056) was granted by the European Commission to Nogra Pharma Limited, Ireland, for (S)-(−)-3-(4-aminophenyl)-2-methoxypropanoic acid (also known as GED-0507-34-Levo) for the treatment of idiopathic pulmonary fibrosis.

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 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.

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

At the time of designation, idiopathic pulmonary fibrosis affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 155,000 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 517,400,000 (Eurostat 2018).

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 because laboratory studies suggested that it might work better than authorised medicines. 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?

The medicine is expected to activate targets inside the cell called PPAR-gamma receptors. These receptors control various processes in the body that reduce inflammation and production of fibrous tissue. By activating PPAR-gamma receptors in the airways, the medicine is expected to reduce inflammation and the development of fibrosis in the lungs and so control the symptoms of the disease.

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, this medicine was not authorised anywhere in the EU for idiopathic pulmonary fibrosis 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 19 July 2018 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/3/18/2056: Public summary of opinion on orphan designation: (S)-(−)ypropanoic acid for the treatment of idiopathic pulmonary fibro-3-(4-aminophenyl)-2-methoxsis

AdoptedReference Number: EMA/521616/2018

Key facts

• Active substance:
  (S)-(−)-3-(4-aminophenyl)-2-methoxypropanoic acid
• Intended use:
  Treatment of idiopathic pulmonary fibrosis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2056
• Date of designation:
  24/08/2018
• Sponsor:

  Nogra Pharma Limited; 33 Sir John Rogerson's Quay; Dublin 2; Ireland; 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.