EU/3/22/2606 - orphan designation for treatment of haemophilia A

adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter

Overview

This medicine was designated as an orphan medicine for the treatment of haemophilia A in the European Union on 13 April 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
• gov

How the medicine is expected to work

This medicine, also known as ASC618, is made of a virus that has been modified to contain a gene that encodes for factor VIII, the protein that is lacking in patients with haemophilia A. This protein is necessary for the formation of blood clots. When ASC618 is given to the patient, the modified virus is expected to carry the gene into the liver cells, enabling them to produce factor VIII. It is expected that a single dose of the medicine will elevate levels of factor VIII for a long period, thereby helping the blood to clot and controlling bleeding. The type of virus used in this medicine ('adeno-associated virus') does not cause disease in humans.

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 haemophilia A had been started.

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

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 haemophilia A 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

Key facts

• Active substance:
  adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter
• Intended use:
  Treatment of haemophilia A
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/22/2606
• Date of designation:
  13/04/2022
• Sponsor:

  3R Pharma Consulting GmbH;