Drug Profile


Alternative Names: AAV8 factor IX gene therapy; AAV8 hFIX19; Haemophilia B gene therapy - Spark Therapeutics; PF-06838435; PF-6838435; rAAV-Spark100-hFIX-Padua; SPK 9001; SPK-FIX

Latest Information Update: 14 Nov 2017

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At a glance

  • Originator The Childrens Hospital of Philadelphia
  • Developer Pfizer; Spark Therapeutics
  • Class Antihaemorrhagics; Gene therapies
  • Mechanism of Action Blood coagulation factor replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Haemophilia B
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Haemophilia B

Most Recent Events

  • 07 Nov 2017 Spark Therapeutics and Pfizer amend license agreement for AAV8-hFIX19 for Haemophilia B
  • 10 Jul 2017 Interim efficacy data from a phase I/II trial in Haemophilia B released by Spark Therapeutics
  • 22 Jun 2017 Spark Therapeutics and Pfizer initiate the observational FIX-LONG trial for Haemophilia B in USA (NCT03307980)
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