EU/3/16/1674: Orphan designation for the treatment in haematopoietic stem cell transplantation

Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19

Table of contents

Overview

On 27 June 2016, orphan designation (EU/3/16/1674) was granted by the European Commission to QRC Consultants Ltd, United Kingdom, for allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 (also called BPX-501) for treatment in haematopoietic stem cell transplantation.

The sponsorship was transferred to Bellicum Pharma Limited, United Kingdom, in May 2017.

The sponsorship was transferred to Bellicum Pharma GmbH, Germany, in February 2019.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in April 2022 on request of the Sponsor.

Key facts

Active substance
Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19
Intended use
Treatment in haematopoietic stem cell transplantation
Orphan designation status
Withdrawn
EU designation number
EU/3/16/1674
Date of designation
27/06/2016
Sponsor

Bellicum Pharma GmbH
Josephspitalstrasse 15
Altstadt-Lehel
80331 Munich
Bavaria
Germany
Tel. +49  1721448300
E-mail: jlamport@bellicum.com

Documents related to this orphan designation evaluation

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:

How useful was this page?

Add your rating