EU/3/17/1908 - orphan designation for treatment of retinitis pigmentosa

Recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor

Overview

On 23 August 2017, orphan designation (EU/3/17/1908) was granted by the European Commission to Dorian Regulatory Affairs BV, the Netherlands, for recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor for the treatment of retinitis pigmentosa.

What is retinitis pigmentosa?

Retinitis pigmentosa is a group of hereditary diseases of the eye that lead to progressive loss of sight. In patients with retinitis pigmentosa, cells in the retina (the light-sensitive surface at the back of the eye) become damaged and eventually die.

Retinitis pigmentosa is a long-term debilitating disease because it causes the patient's sight to get worse, eventually leading to blindness.

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

At the time of designation, retinitis pigmentosa affected approximately 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 191,000 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 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).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for treating retinitis pigmentosa. Patients with the condition were given sunglasses to slow down damage to the retina, genetic counselling (discussion of the risks of passing the condition on to children) and general support.

How is this medicine expected to work?

The way this medicine works is still being investigated, but laboratory studies have shown that injecting it into the eye can reduce damage to the retina. It is believed to reduce damage to retinal cells by encouraging the cells of the retina to produce protective proteins that help them resist damage from toxic substances or improperly formed proteins.

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 retinitis pigmentosa had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for retinitis pigmentosa. 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 13 July 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.

Key facts

• Active substance:
  Recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor
• Medicine name:
  -
• Intended use:
  Treatment of retinitis pigmentosa
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/17/1908
• Date of designation:
  23/08/2017
• Sponsor:

  Dorian Regulatory Affairs BV; Soestdijkerstraatweg 7; 1213 VP Hilversum; The Netherlands; Tel. [phone]; Fax [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.