EU/3/17/1924 - orphan designation for treatment of follicular lymphoma
Glucopyranosyl lipid A
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in October 2019 on request of the Sponsor.
The sponsorship was transferred to Immune Design Ltd, United Kingdom, in August 2016.
On 16 October 2017, orphan designation (EU/3/17/1924) was granted by the European Commission to Immune Design Ltd, United Kingdom, for glucopyranosyl lipid A stable emulsion (also known as G100) for the treatment of follicular lymphoma.
Follicular lymphoma is a cancer of a type of white blood cell called B lymphocytes or B cells. In follicular lymphoma, the B cells multiply quickly and live for too long, so there are too many of them in the lymph nodes. The first sign of the disease is usually a lump in the neck, under the arm or in the groin area, caused by an enlarged lymph node. Patients may also have fever, weight loss, tiredness and night sweats.
Follicular lymphoma is usually diagnosed in people aged over 50 years. It is a long-term debilitating and life-threatening disease due to organ damage and the cancer coming back.
At the time of designation, follicular lymphoma affected approximately 3.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 186,000 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, the main treatments for follicular lymphoma available in the EU included chemotherapy (medicines to treat cancer) combined with immunotherapy (medicines that stimulate the body's own immune system to kill the cancer cells). The medicines ibritumomab tiuxetan, idelalisib, interferon alfa-2b and rituximab were specifically authorised for the treatment of follicular lymphoma.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with follicular lymphoma, with laboratory studies showing improved outcomes with this medicine, including when combined with radiotherapy. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is expected to work by stimulating the patient's immune (defence) system, so that it targets and destroys the cancer cells. When injected directly into a tumour, this medicine is expected to attach to and activate a protein called Toll-like receptor 4, which in turn stimulates immune cells to attack cancer cells and thereby slow progression of the disease.
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 follicular lymphoma were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for follicular lymphoma. 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 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
- Glucopyranosyl lipid A
- Intended use
- Treatment of follicular lymphoma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1924
- Date of designation
- Sponsor
Immune Design Ltd
21 Holborn Viaduct
London EC1A 2DY
United Kingdom
Tel. +44 (0) 2031 005 102
Contact us
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: