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Orphan designation - Europe

  • Substance: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII
  • ATC code : -
  • EU Number: EU/3/18/2015
  • Designation date : 25/05/2018
  • Sponsor of orphan designation : BAXALTA INNOVATIONS GMBH
  • Summary of Opinion


Treatment of haemophilia A.

Diseases list

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