EU/3/14/1352 - orphan designation for treatment of inhalation anthrax disease
Raxibacumab
OrphanHuman
Overview
On 15 October 2014, orphan designation (EU/3/14/1352) was granted by the European Commission to GlaxoSmithKline Trading Services Limited, Ireland, for raxibacumab for the treatment of inhalation anthrax disease.
The sponsorship was transferred to Emergent Countermeasures International Ltd., United Kingdom, in January 2018.
The sponsorship was transferred to Emergent Netherlands B.V., The Netherlands, in April 2019.
Anthrax is a severe disease caused by infection with bacteria called Bacillus anthracis. The bacteria produce spores that are very resistant and can lay 'dormant' (inactive) until they find an organism where they can develop and multiply. Anthrax commonly affects animals such as sheep and cows, but can spread to humans when they are exposed to spores from infected animals or contaminated animal products.
The most severe type of anthrax is inhalation anthrax, which occurs when a person has breathed in the bacteria's spores. The first symptoms of inhalation anthrax are similar to a cold. Several days after the spores have been inhaled, they grow into new bacteria and start to release toxins, which cause internal bleeding, swelling and the death of tissue.
Inhalation anthrax disease is a life-threatening disease because, if not treated early, it leads to the accumulation of fluid in the lungs, severe inflammation and bleeding of the tissues in the chest and haemorrhagic meningitis (inflammation and bleeding of the membranes that surround the brain and spine).
At the time of designation, inhalation anthrax disease affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 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 511,100,000 (Eurostat 2014).
At the time of designation, several antibiotics were authorised in the EU for the treatment of inhalation anthrax disease.
The sponsor has provided sufficient information to show that raxibacumab might be of significant benefit for patients with inhalation anthrax disease because preclinical studies showed that the medicine improves survival when used in combination with current treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Raxibacumab is a monoclonal antibody, a type of protein that has been designed to recognise and attach to the anthrax toxins (i.e. to the anthrax protective antigen). By attaching to the anthrax toxins, the medicine is expected to stop them from entering the body's cells, thereby reducing the symptoms of the disease.
The effects of raxibacumab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with raxibacumab in patients with inhalation anthrax disease were ongoing.
At the time of submission, raxibacumab was authorised in the United States for the treatment of adult and paediatric patients with inhalational anthrax due to Bacillus anthracis in combination with appropriate antibacterial medicines, and for prophylaxis of inhalational anthrax when alternative therapies are not available or are not appropriate.
At the time of submission, raxibacumab was not authorised anywhere in the EU for the treatment of inhalation anthrax disease. 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 4 September 2014 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
- Raxibacumab
- Intended use
- Treatment of inhalation anthrax disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1352
- Date of designation
- Sponsor
Emergent Netherlands B.V.
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: