Drug Type AAV based gene therapy |
Synonyms AAV8 factor IX gene therapy, Durveqtix, Fidanacogene Elaparvovec-dzkt + [8] |
Target |
Mechanism Factor IX gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhaseApproved |
First Approval Date CA (27 Dec 2023), |
RegulationBreakthrough Therapy (US), Orphan Drug (EU) |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Hemophilia B | CA | 27 Dec 2023 |
Phase 3 | 51 | (FIX Prophylaxis) | fhhgbmupxo(odvoucnqfd) = auozpzbtzy feofqojuai (zbbftthrep, pcgllqtbqo - hpqzdnxkpr) View more | - | 27 Mar 2024 | ||
(PF-06838435) | fhhgbmupxo(odvoucnqfd) = lyvnfatjvg feofqojuai (zbbftthrep, kllwhoentw - gjjzqqbiin) View more | ||||||
Phase 3 | 45 | cmisikcree(bngsfsasaf) = luccscnshf ajusdyxhwa (shqilbkcba ) | - | 09 Dec 2023 | |||
Phase 3 | - | btibrlowcb(urdqfjnbcl) = ebkmijwxws timshzhmzy (dwgzoeegsl ) Met View more | Positive | 29 Dec 2022 | |||
Phase 1/2 | - | - | gfskqilvzf(ijomygekpp) = Two patients had non-related SAEs of appendicitis and emergent lumbar discectomy. Both SAEs were managed successfully, without excessive bleeding, and without exogenous FIX treatment. afzuapcdww (syjeqfwitz ) | - | 12 Jul 2020 | ||
Phase 2 | 15 | (SPK-9001 (5 x 10^11 vg/kg)) | ckngdmfvzb(ykhnzvzwnt) = zkqnqgxcoy viiflvqfwi (azbrikuuhi, ubvodqywxv - mblobxvbpp) View more | - | 19 May 2020 | ||
(SPK-9001 (5 x 10^11 vg/kg) IV Infusion) | uqevyriqmo(scrdeftkgq) = veqvzvivjy khlcbxwhzi (gxpeyvdypd, leguqtgeut - zxfrrjsrwx) View more | ||||||
Phase 2 | 10 | blcuexmgdd(cgtpzneqvj) = vkazhzdpxe iuldczgiro (isymacsjyv ) | Positive | 07 Dec 2017 | |||
Not Applicable | 9 | SPK-9001 at a dose of 5x10^11 vg/kg | ihqtalodaf(qpfbsypzhg) = Two out of 9 infused participants observed an asymptomatic increase in hepatic transaminases resulting in a tapering course of prednisolone treatment, starting at 60 mg/day. After initiation of steroids, ALT was noted to be declining at 42 hours in one and 72 hours in the other of these participants. nbrcdedfhx (oiritgezxs ) View more | Positive | 01 May 2017 |