AAV8-hFIX19

Drug Profile

AAV8-hFIX19

Alternative Names: AAV8 factor IX gene therapy; AAV8 hFIX19; Haemophilia B gene therapy - Spark Therapeutics; SPK 9001; SPK-FIX

Latest Information Update: 06 Mar 2017

Price : $50

At a glance

  • Originator The Childrens Hospital of Philadelphia
  • Developer 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Haemophilia B

Most Recent Events

  • 01 Mar 2017 AAV8-hFIX19 receives Priority Medicine (PRIME) status for Haemophilia B in European Union
  • 03 Dec 2016 Preliminary efficacy and adverse events data from a phase I/II trial in Haemophilia B presented at the 58th American Society of Hematology Annual Meeting and Exposition (ASH-2016)
  • 03 Dec 2016 Preliminary efficacy and adverse events data from a phase I/II trial in Haemophilia B released by Spark Therapeutics
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