EU/3/22/2679 - orphan designation for treatment of graft-versus-host-disease

Allogeneic placenta-derived decidual stromal cells

Overview

This medicine was designated as an orphan medicine for the treatment of graft-versus-host-disease in the European Union (EU) on 10 August 2022.

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

This medicine belongs to the class of: Cell replacements.

Graft-versus-host disease (GvHD) occurs when transplanted cells attack the body because the transplanted tissue regards the patient’s tissue as ‘foreign’. This medicine contains decidua stromal cells (DSCs) derived from the placenta of human donors, which are then prepared and grown in a laboratory to increase their numbers. The medicine is expected to regulate the immune system, reducing the activation and growth of the graft cells responsible for attacking the patient’s tissue, thereby decreasing inflammation and damage to the affected organs caused by GvHD.

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.
• Clinical trials with the medicine in patients with Graft-versus-host-disease were ongoing.

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 12-14 July 2022

Reference Number: EMA/COMP/646322/2022 Corr.1

Key facts

• Active substance:
  Allogeneic placenta-derived decidual stromal cells
• Intended use:
  Treatment of graft-versus-host-disease
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/22/2679
• Date of designation:
  10/08/2022
• Sponsor:

  3R Pharma Consulting GmbH;

Review of designation

Date	Update
September 2023	The sponsorship was transferred from MDC RegAffairs GmbH to 3R Pharma Consulting GmbH