EU/3/21/2537 - orphan designation for prevention of bronchopulmonary dysplasia

retinol palmitate

Overview

This medicine was designated as an orphan medicine for the prevention of bronchopulmonary dysplasia in the European Union on 10 December 2021.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

• EU Clinical Trials Register

• ClinicalTrials.gov

How the medicine is expected to work

The human foetus accumulates retinol, a form of vitamin A, in the third trimester of pregnancy; however in premature babies retinol stores are lower as this process has not had time to occur. Increasing levels of retinol to normal levels in premature babies is expected to improve lung development and help the lungs absorb oxygen better, therefore reducing the need for oxygen supplementation and mechanical ventilation and helping to prevent bronchopulmonary dysplasia. This medicine contains retinol in a form that can be administered by injection or inhalation since premature babies cannot absorb vitamin A properly when given by mouth.

Based on description provided by sponsor

Stage of development at time of orphan designation

At the time of submission of the application for orphan designation:

• The effects of the medicine had been evaluated in experimental models.
• No clinical trials with the medicine in patients with bronchopulmonary dysplasia had been started.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

About orphan designation

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

• scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;

• market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;

• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;

• there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

• Orphan designation: Overview

• Rare diseases, orphan medicines - Getting the facts straight

Minutes of the COMP meeting on 3 - 5 November 2021

Adopted Reference Number: EMA/COMP/647448/2021

Key facts

• Active substance:
  retinol palmitate
• Intended use:
  Prevention of bronchopulmonary dysplasia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/21/2537
• Date of designation:
  10/12/2021
• Sponsor:

  Transcrip Ireland Limited