Drug Profile
Dirloctocogene samoparvovec - Spark Therapeutics
Alternative Names: AAV-Spark 200-hFVIII; RG 6357; SPK-8011Latest Information Update: 13 Mar 2024
Price :
$50
*
At a glance
- Originator Spark Therapeutics
- Class Antihaemorrhagics; Gene therapies
- Mechanism of Action Factor VIII replacements; Gene transference
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase III Haemophilia A
Most Recent Events
- 29 Feb 2024 Spark Therapeutics plans a phase III KEYSTONE 1 trial for Hemophilia A (Treatment-experienced), in February 2024 (IV, Infusion) (NCT06297486, EudraCT2023-504537-46)
- 05 Dec 2023 Spark Therapeutics completes a phase I/II trial in Haemophilia A in the US, Australia, Canada, Israel and Thailand (NCT03003533)
- 12 Dec 2022 Efficacy and adverse events data from a phase I/II trial in Haemophilia A presented at the 64th American Society of Hematology annual meeting (ASH-2022)