Refusal of orphan designation for the treatment of retinopathy of prematurity

melatonin

Overview
Key facts

Overview

Melatonin has not been designated as an orphan medicine for the treatment of retinopathy of prematurity in the European Union, despite an application from the sponsor.

Absence of orphan designation does not preclude the development of this medicine, including its use in clinical trials. The medicine can be authorised if its quality, safety and efficacy are demonstrated.

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

: The sponsor applied for orphan designation on the basis of the seriousness and the rarity of the condition, as well as an assumption of potential benefit of this medicine over currently available methods of treatment.
The Committee for Orphan Medicinal Products (COMP) recommended against granting the orphan designation because the data provided by the sponsor were not sufficient to establish that the medicine will provide a significant benefit over the authorised methods for treating retinopathy of prematurity.
The COMP adopted its negative opinion on 14 July 2022. Following the appeal withdrawal by the sponsor, a negative decision was issued by the European Commission on 28 November 2022.

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

Key facts

Active substance	melatonin
Intended use	Treatment of retinopathy of prematurity
Orphan designation status	Negative
EU designation number	-
Date of refusal of designation	28/11/2022
Sponsor	Industria Farmaceutica Galenica Senese S.r.l. Via Cassia Nord 351 53014 Monteroni D'arbia (SI) Italy E-mail: f.rosi@galenicasenese.it

Documents related to this orphan designation evaluation

Minutes of the COMP meeting 4-6 October 2022 (PDF/467.61 KB)

Adopted

First published: 17/11/2022
EMA/COMP/835868/2022

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

European Commission: Community Register of orphan medicinal products

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