EU/3/13/1157 - orphan designation for treatment of follicular lymphoma
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
OrphanHuman
Overview
On 17 July 2013, orphan designation (EU/3/13/1157) was granted by the European Commission to Voisin Consulting SARL, France, for S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one for the treatment of follicular lymphoma.
The sponsorship was transferred to Abbvie Ltd, United Kingdom, in November 2015, then to Voisin Consulting S.A.R.L., France, in September 2016 and finally to Verastem Europe GmbH, Germany, in November 2019.
Please note that this product (marketed as Copiktra) was withdrawn from the Union Register of orphan medicinal products in March 2021 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
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 too 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 life-threatening and long-term debilitating 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 183,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).
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 bendamustine, ibritumomab tiuxetan, 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 because early studies show that it might improve the outcome of patients with advanced disease. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine blocks the effects of two enzymes called PI3K-delta and PI3K-gamma. These are members of a family of enzymes called phosphoinositide-3-kinases (PI3K) that play an important role in the growth, migration and survival of white blood cells. These enzymes are active in the abnormal B lymphocytes of follicular lymphoma patients, stimulating their growth and survival. By blocking the effects of these enzymes, the medicine is expected to reduce the growth and survival of the cancer cells.
The effects of the medicinal product have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicinal product in patients with follicular lymphoma were ongoing.
At the time of submission, the medicinal product was not authorised anywhere in the EU for follicular lymphoma 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 13 June 2013 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
- (S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
- Intended use
- Treatment of follicular lymphoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1157
- Date of designation
- Sponsor
Verastem Europe GmbH
Lange Strasse 70
Walsrode
Lower Saxony 29664
Germany
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of (S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one at the time of marketing authorisation.
The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the withdrawal assessment report – orphan maintenance.
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: