EU/3/18/2090 - orphan designation for treatment of haemophilia B
fidanacogene elaparvovec
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2023 on request of the Sponsor.
On 19 November 2018, orphan designation (EU/3/18/2090) was granted by the European Commission to Pfizer Europe MA EEIG, Belgium, for fidanacogene elaparvovec for the treatment of haemophilia B.
Haemophilia B is an inherited bleeding disorder caused by the lack of factor IX, a protein involved in the blood coagulation (clotting) process. Patients with haemophilia B are more prone to bleeding than normal and have poor wound healing after injury or surgery. Internal bleeding can also happen within muscles or in the joints, such as the elbows, knees and ankles. This can lead to permanent damage if it happens repeatedly.
Haemophilia B is a debilitating disease that is life long and may be life threatening because bleeding can also happen in the brain and spinal cord, the throat or the gut.
At the time of designation, haemophilia B affected approximately 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 16,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).
At the time of designation, medicines containing factor IX were authorised in the EU for the treatment of haemophilia B, to replace the missing protein. However, factor IX medicines did not work in some patients with haemophilia B because the immune system (the body’s natural defences) can produce ‘inhibitors’ (antibodies) against factor IX which stop the factor IX medicine from working. In these cases, other treatments needed to be used, such as factor VIIa (the activated form of factor VII, another protein involved in blood clotting), either alone or as part of a combination treatment.
The sponsor has provided sufficient information to show that fidanacogene elaparvovec might be of significant benefit for patients with haemophilia B. Preliminary results in patients show that a single dose can increase the amount of factor IX circulating in the blood in the long term, thus reducing bleeding and the need for treatment with factor IX medicines.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Patients with haemophilia B cannot make enough working factor IX because the gene for producing the clotting factor is damaged.
This medicine is made of a virus that contains copies of the gene responsible for producing factor IX. When injected into the patient, it is expected that the virus will be carried into the liver cells and start producing factor IX. It is expected that a single dose of the medicine will maintain raised levels of factor IX for a long time, thereby reducing bleeding.
The type of virus used in this medicine (‘adeno-associated virus’) does not cause disease in humans.
The effects of fidanacogene elaparvovec have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with haemophilia B were ongoing.
At the time of submission, fidanacogene elaparvovec was not authorised anywhere in the EU for haemophilia B. Orphan designation of the medicine had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 October 2018 recommending the granting of this designation.
- the seriousness of the condition;
- the existence of alternative methods of diagnosis, prevention or treatment;
- either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.
Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.
Key facts
- Active substance
- fidanacogene elaparvovec
- Intended use
- Treatment of haemophilia B
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/18/2090
- Date of designation
- Sponsor
Pfizer Europe MA EEIG
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
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: