EU/3/17/1895 - orphan designation for prevention of retinopathy of prematurity

retinol

Overview

On 17 July 2017, orphan designation (EU/3/17/1895) was granted by the European Commission to orphanix GmbH, Austria, for retinol for the prevention of retinopathy of prematurity.

The sponsor’s address was updated in March 2020.

What is retinopathy of prematurity?

Retinopathy of prematurity is an eye condition that occurs in babies who are born prematurely. A baby's blood supply to the eye develops mainly in the last few weeks of pregnancy. When a baby is born too early, blood vessels in the eye may not develop normally. Instead, abnormal and fragile blood vessels may develop, resulting in damage to the retina, the area at the back of the eye which detects light and is essential for normal vision.

Retinopathy of prematurity is debilitating in the long term because it can reduce sight and may cause blindness.

What is the estimated number of patients at risk of developing the condition?

At the time of designation, the number of patients at risk of retinopathy of prematurity was estimated to be approximately 1.4 people in 10,000 in the European Union (EU). This was equivalent to a total of around 72,000 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).

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*Disclaimer: For the purpose of the designation, the number of patients at risk of developing 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).

What methods of prevention are available?

At the time of the designation, no satisfactory method was authorised in the EU for the prevention of retinopathy of prematurity. Treatment once the condition began to develop consisted of laser or cryotherapy (treatment of the affected area using low temperature) to close the abnormal blood vessels that damage the retina.

How is this medicine expected to work?

Retinol is a form of vitamin A. It prevents damage to cells in the eye and is involved in maintaining the health of the retina and other parts of the eye. Retinol also controls a substance called 'vascular endothelial growth factor' which helps to develop healthy blood vessels. It is expected that giving retinol by injection will reduce damage to the retina and maintain eye health in babies at risk of retinopathy of prematurity.

What is the stage of development of this medicine?

The effects of retinol have been evaluated in experimental models. As retinol is a well-known substance, the sponsor provided data in patients from the published literature to support its application for orphan designation.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in babies at risk of retinopathy of prematurity were ongoing.

At the time of submission, retinol was not authorised anywhere in the EU for prevention of retinopathy of prematurity 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 15 June 2017 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/3/17/1895: Public summary of opinion on orphan designation: Retinol for the prevention of retinopathy of prematurity

Reference Number: EMA/402226/2017

Key facts

• Active substance:
  retinol
• Intended use:
  Prevention of retinopathy of prematurity
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1895
• Date of designation:
  17/07/2017
• Sponsor:

  orphanix GmbH; Lederergasse 18/1; 4020 Linz; Austria; 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.