Search for an orphan drug

* (*) mandatory field

Other search option(s)

Orphan designation - Europe

  • Substance: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
  • ATC code : -
  • EU Number: EU/3/12/1091
  • Designation date : 24/01/2013
  • Sponsor of orphan designation : BLUEBIRD BIO FRANCE
  • Summary of Opinion


Treatment of beta-thalassaemia intermedia and major.

The documents contained in this web site are presented for information purposes only. The material is in no way intended to replace professional medical care by a qualified specialist and should not be used as a basis for diagnosis or treatment.