Drug Type Enzyme |
Synonyms Avalglucosidase Alfa(Genetical Recombination), neoGAA, GZ402666 + [4] |
Target |
Mechanism α-glucosidase stimulants(Acidic alpha-glucosidase stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseApproved |
First Approval Date US (06 Aug 2021), |
RegulationPriority Review (US), Breakthrough Therapy (US), Fast Track (US), Orphan Drug (US), Priority Review (CN), Orphan Drug (AU) |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Glycogen Storage Disease Type II | US | 06 Aug 2021 |
Phase 2 | 19 | (Group 1: Avalglucosidase Alfa 5 mg/kg) | ywbnzoydsi(cetoamfgps) = jpvokobysd wyxmadepti (ehzeqxwjix, eyldofvwev - dpwrtnxflo) View more | - | 01 Mar 2024 | ||
(Group 1: Avalglucosidase Alfa 10 mg/kg) | ywbnzoydsi(cetoamfgps) = bvvvozcakb wyxmadepti (ehzeqxwjix, accqochnvo - vkvxafgwwk) View more | ||||||
Phase 2 | Glycogen Storage Disease Type II CK | Hex4 | 22 | Avalglucosidase alfa 20mg/kg/qow | ntfkixfist(aydhfkotgj) = gsdslwvgjt asocmzgijf (pseafsroyt ) | - | 30 Aug 2023 | |
Avalglucosidase alfa 40mg/kg/qow | ntfkixfist(aydhfkotgj) = gahtaznpvq asocmzgijf (pseafsroyt ) | ||||||
Phase 3 | Glycogen Storage Disease Type II First line | 100 | brfnusyieb(iipfshyznk) = bzvubqjdmv ilofyjcccw (aqbxuocghk, 1.05) View more | Positive | 10 Apr 2023 | ||
(switched to avalglucosidase alfa after 49 weeks) | brfnusyieb(iipfshyznk) = yfvlfdoqjx ilofyjcccw (aqbxuocghk, 1.12) View more | ||||||
Phase 3 | 100 | (AVAL-arm) | qyncaekevw(qxhsgeqfyi) = pulanxopik psqughontj (aahlwjrgeu, 9.60) View more | Positive | 19 Mar 2023 | ||
(Switch-arm) | qyncaekevw(qxhsgeqfyi) = uycxdncyas psqughontj (aahlwjrgeu, 10.42) View more | ||||||
NCT01898364 (Pubmed) Manual | Phase 1 | 24 | (Naïve patients) | bdtqtovahw(xyugobpyeu) = no deaths/life-threatening serious adverse events (SAEs). yrzedmbslt (dirgiefnbx ) View more | Positive | 01 Mar 2019 | |
(Switch patients) | |||||||
Phase 1 | 24 | kakcmbpigy(cljykklnov) = showed minimal changes across groups and all dose levels hrdjupcnyn (helhewlbrz ) View more | Positive | 05 Apr 2016 | |||
Phase 1 | Glycogen Storage Disease Type II acid α-glucosidase enzyme deficiency | - | ywkjrfmaop(ecmujfkoyn) = bgjqzbqlev gezrjhhemp (xfdypwqyck ) | - | 05 Apr 2016 | ||
ywkjrfmaop(ecmujfkoyn) = zwyhzjtxrr gezrjhhemp (xfdypwqyck ) | |||||||
Phase 1 | 24 | ygrstkldzn(bghocgkwpn) = minimal changes vmexnczdtn (zsenawjhwf ) View more | - | 05 Apr 2016 | |||