EU/3/17/1929 - orphan designation for treatment of mastocytosis
recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in May 2024 on request of the Sponsor.
On 16 October 2017, orphan designation (EU/3/17/1929) was granted by the European Commission to Envestia Limited, United Kingdom, for recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8 (also known as AK002) for the treatment of mastocytosis.
The sponsorship was transferred to Turnkey Pharmaconsulting Ireland Limited, Ireland, in October 2019.
Mastocytosis is a group of disorders in which there are too many mast cells, a type of white blood cell, in various organs in the body. These cells release large amounts of histamine and other chemicals into the blood, causing symptoms such as a skin rash, itchy skin and hot flushes.
In children, the disorder usually only affects the skin ('cutaneous mastocytosis') and causes a red and itchy rash, which may disappear on its own. In some patients, mainly adults, the disorder progresses into 'systemic mastocytosis', in which the mast cells infiltrate organs, such as the intestine, the liver, the spleen and the bone marrow. This causes various symptoms such as palpitations and fainting, bone pain, tiredness, weight loss, diarrhoea, nausea (feeling sick), vomiting and stomach ache.
Mastocytosis is a condition that is debilitating in the long term and may be life threatening in those patients who develop the systemic form of the disorder.
At the time of designation, mastocytosis affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 155,000 people1, 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).
1Disclaimer: 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 515,700,000 (Eurostat 2017).
At the time of designation, treatments available in the EU aimed at relieving some of the symptoms of mastocytosis, including antihistamines such as cimetidine or famotidine to block the action of histamine produced by the mast cells.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with mastocytosis, with studies showing that the medicine reduces levels of mast cells and of eosinophils, another type of white blood cell found at high levels in mastocytosis.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a monoclonal antibody, a type of protein, that is designed to attach to a target on mast cells known as sialic acid-binding Ig-like lectin 8 (siglec 8). By attaching to siglec 8, the medicine blocks the growth of mast cells and their release of histamine and other chemicals. This is expected to relieve symptoms of the condition.
The effects of the medicine 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 mastocytosis were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for mastocytosis or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 2017 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
- recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8
- Intended use
- Treatment of mastocytosis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1929
- Date of designation
- Sponsor
Turnkey PharmaConsulting Ireland Limited
Ellerman House
Cratloe Wood
Cratloe V95 X925
County Clare
Ireland
Tel: +353 86 844 6816
E-mail: markturner@turnkeypharmaconsulting.com
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: