EU/3/19/2217 - orphan designation for treatment of retinopathy of prematurity

propranolol hydrochloride

Overview

On 17 October 2019, orphan designation EU/3/19/2217 was granted by the European Commission to Recordati Rare Diseases, France, for propranolol hydrochloride for the treatment of retinopathy of prematurity.

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 affected by the condition?

At the time of designation, retinopathy of prematurity affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 52,000 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 518,400,000 (Eurostat 2019).

What treatments are available?

At the time of the designation, no satisfactory method of treatment was authorised in the EU for 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?

Propranolol hydrochloride blocks a target called beta-adrenoreceptor which is involved in the production of VEGF, a protein involved in the growth of blood vessels. By blocking the action of this receptor, the medicine is expected to reduce the formation of new blood vessels which occurs in patients with retinopathy of prematurity. This should reduce the symptoms of the disease. The medicine is expected to be available as eye drops.

What is the stage of development of this medicine?

The effects of propranolol hydrochloride have been evaluated in experimental models.

Propranolol hydrochloride is authorised in some EU countries to treat various conditions, and is taken by mouth or injected into a vein. 

At the time of submission of the application for orphan designation, clinical trials with propranolol hydrochloride in patients with retinopathy of prematurity were ongoing.

At the time of submission, propranolol hydrochloride was not authorised anywhere in the EU for the treatment of retinopathy of prematurity 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 12 September 2019, 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/3/19/2217: Public summary of positive opinion for orphan designation of propranolol hydrochloride for the treatment of retinopathy of prematurity

Adopted Reference Number: EMADOC-628903358-1518

Key facts

• Active substance:
  propranolol hydrochloride
• Intended use:
  Treatment of retinopathy of prematurity
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2217
• Date of designation:
  17/10/2019
• Sponsor:

  Recordati Rare Diseases ;