EU/3/22/2685 - orphan designation for treatment of urea cycle disorders

allogeneic adult liver-derived stem cells

Overview

This medicine was designated as an orphan medicine for the treatment of urea cycle disorders in the European Union 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

Citrullinemia type 1 (CTLN1) is a urea-cycle disorder that arises from a deficiency in the enzyme argininosuccinate synthetase (ASS). Patients with urea-cycle disorders cannot remove ammonia from their body. Because of this, levels of ammonia in their blood can greatly increase, leading to serious problems including disability, brain damage and death.

This medicine contains stem cells (undifferentiated cells that can develop into different type of cells) derived from the liver of an adult donor ('heterologous'). When implanted in a patient, it is believed that these heterologous liver-derived stem cells will develop into mature, healthy liver cells. The new cells are expected to restore the normal urea cycle and thereby relieve the symptoms of the disease.

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 evaluation of the effects of the medicine in experimental models was ongoing.
• No clinical trials with the medicine in patients with urea cycle disorders 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 12-14 July 2022

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

Key facts

• Active substance:
  allogeneic adult liver-derived stem cells
• Intended use:
  Treatment of urea cycle disorders
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/22/2685
• Date of designation:
  10/08/2022
• Sponsor:

  Unicyte S.R.L.; Via Ernesto Lugaro 15; 10126 Turin (TO); Italy; E-mail: [e-mail]