EU/3/20/2273 - orphan designation for prevention of fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility
anti-(integrin beta-3) human monoclonal antibody
OrphanHuman
Overview
On 22 April 2020, orphan designation EU/3/20/2273 was granted by the European Commission to FGK Representative Service GmbH, Germany, for anti-(integrin beta-3) human monoclonal antibody (also known as RB 212) for the prevention of fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility.
Fetal and neonatal alloimmune thrombocytopenia (FNAIT) due to human platelet antigen-1a incompatibility is a rare disease in which fetuses and newborn babies have low levels of platelets (components of the blood involved in clotting).
It occurs when the mother produces antibodies that cross the placenta to attack the baby’s platelets because the platelets contain a substance (human platelet antigen-1a (HPA-1a)), which the mother’s immune system recognises as foreign.
FNAIT is chronically debilitating and life threatening since it can cause bleeding within the skull in the fetus or newborn baby, and can lead to miscarriage, stillbirth, death of the newborn baby or permanent damage to the child’s brain and nerves.
At the time of designation, the number of patients at risk of FNAIT due to human platelet antigen-1a incompatibility was estimated to be approximately 2.5 people in 10,000 in the European Union (EU). This was equivalent to a total of around 130,000 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).
* 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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, no satisfactory methods were authorised in the EU for the prevention of FNAIT due to human platelet antigen-1a incompatibility.
The medicine contains a monoclonal antibody (a type of protein) designed to attach to a specific target, in this case HPA-1a. Unlike antibodies produced by the mother, when the medicine is injected into the mother's blood, it does not pass the placenta and therefore does not affect the fetus. It is expected to work by attaching to and removing any HPA-1a platelets produced by the fetus that enter the mother’s circulation, and preventing the mother's immune system from reacting against them. In this way the immune system reaction from the mother's body against fetal platelets can be avoided, thus preventing the occurrence of the condition.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with FNAIT due to human platelet antigen-1a incompatibility had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for the prevention of FNAIT due to human platelet antigen-1a incompatibility or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 19 March 2020, 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
- anti-(integrin beta-3) human monoclonal antibody
- Intended use
- Prevention of fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2273
- Date of designation
- Sponsor
FGK Representative Service GmbH
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:
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: