EU/3/20/2247: Orphan designation for the treatment of myelofibrosis

2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate

Table of contents


On 28 February 2020, orphan designation EU/3/20/2247 was granted by the European Commission to IQVIA RDS Ireland Limited, Ireland, for 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate (also known as CPI-0610) for the treatment of myelofibrosis.


Key facts

Active substance
2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate
Intented use
Treatment of myelofibrosis
Orphan designation status
EU designation number
Date of designation

MorphoSys AG
Semmelweisstrasse 7
82152 Planegg
Tel. +49 89 89927-0


Update history

December 2021The sponsorship was transfered to MorphoSys AG, Germany. 
June 2020The sponsorship was transferred to AdRes EU B.V Netherlands.

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