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Atlas/Gene therapy/Fayuvi/Efficacy evidence

Fayuvi: animal efficacy model or human cells?

rebisufligene etisparvovec · SGSH (AAV9)
Check my plan vs. these
Efficacy evidence
MPS IIIA mouse pharmacology study (survival, brain GAG, Morris water maze, GFAP, neuropathology); SGSH knock-out cells in vitro
“The minimum effective dose level of 5.0x1012 vg/kg was identified in the definitive pharmacology study conducted in mice based on survival, brain glycosaminoglycan (GAG) concentration, Morris water maze performance, reduced brain glial fibrillary acidic protein (GFAP) , and improved neuropathology.”
✓ VerifiedFDA summary basis for approval, p. 11Source ↗
Why that model
Not stated in public reviews
Not reported in the public reviews. Reviewers summarise selectively, so this usually means it wasn't discussed publicly, not that it wasn't studied.
Reviewer's view of the efficacy data
Not stated in public reviews
Not reported in the public reviews. Reviewers summarise selectively, so this usually means it wasn't discussed publicly, not that it wasn't studied.

Same question, other gene therapies

Adstiladrin

nadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical)
Orthotopic mouse model of human bladder cancer: anti-tumor activity after intravesical rAd-IFN/Syn3

Beqvez

fidanacogene elaparvovec · Factor IX
Hemophilia B mice given BEQVEZ: liver hFIX-R338L expression and hemostatic FIX activity

Casgevy

exagamglogene autotemcel · BCL11A enhancer (CRISPR-Cas9, ex vivo HSC)
Edited human CD34+ cells engrafted in NSG mice retained >90% on-target editing at 16 weeks

Elevidys

delandistrogene moxeparvovec · Micro-dystrophin
Rodent DMD models: mdx mice and Dmdmdx rats (force, muscle pathology, micro-dystrophin expression)

Genglycos

pariglasgene brecaparvovec · G6PC (AAV8)
G6PC-deficient GSDIa mouse model: dose-dependent increases in hepatic G6Pase activity and fasting glucose

Glybera

alipogene tiparvovec · LPL (AAV1)
LPL-/- mice and LPL-deficient cats: triglyceride-lowering pharmacodynamic activity

Hemgenix

etranacogene dezaparvovec · Factor IX
Hemophilia B mouse (B6.129P2-F9tm1Dws) with predecessor vector AMT-060: dose-dependent liver transduction, hFIX protein and activity

Imlygic

talimogene laherparepvec · GM-CSF (oncolytic HSV-1)
Syngeneic mouse melanoma (B16F10-muNectin1) with clinical virus: significant anti-tumour effect after one or three intratumoral doses

Itvisma

onasemnogene abeparvovec (intrathecal) · SMN1 (AAV9, intrathecal)
SMA piglet model, IT research-grade vector (scAAV9.CBA.SMN): prevented hindlimb weakness and slowed disease progression

Kebilidi

eladocagene exuparvovec · AADC
In vitro studies in human cells (AADC expression and biological activity) only

Kresladi

marnetegragene autotemcel · ITGB2 (LVV, ex vivo HSC)
CD18HYP and CD18KO LAD-I mice given vector-transduced murine Lin- cells: engraftment and enhanced neutrophil extravasation to inflamed sites

Lenmeldy

atidarsagene autotemcel · ARSA (LVV, ex vivo HSC)
As2-/- MLD mice given ARSA-LV murine HSPCs: improved motor conduction, rotarod coordination and neuropathology vs GFP-LV controls

Luxturna

voretigene neparvovec · RPE65
RPE65-mutant dogs: improved pupillary responses, ERGs and visual behavior, reduced nystagmus

Lyfgenia

lovotibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC)
Transgenic SCD mice given marrow cells transduced with a related βA-T87Q vector: βA-T87Q expression and correction of sickling

Otarmeni

lunsotogene parvec · OTOF (AAV, intracochlear)
Otof-Q828X homozygous deafness mouse: dose-dependent ABR threshold recovery with otoferlin expression in IHCs

Papzimeos

zopapogene imadenovec · HPV6/11 antigens (gorilla adenovirus)
HPV antigen-specific T-cell responses in immunized healthy wild-type C57BL/6 mice; no disease model

Roctavian

valoctocogene roxaparvovec · Factor VIII
Rag2-/- x FVIII-/- haemophilia A mice: blood loss and bleeding time almost corrected to wild-type levels

Skysona

elivaldogene autotemcel · ABCD1 (LVV, ex vivo HSC)
Healthy-donor Lenti-D HSCs in myeloablated immunodeficient mice: bone marrow and brain engraftment with ABCD1 expression and ALDP production

Strimvelis

autologous CD34+ cells transduced with ADA (no INN) · ADA (gamma-retroviral, ex vivo HSC)
SCID-hu xenograft mice: transduced CD34+ cells engrafted and produced vector-marked, transgene-expressing T and B cells

Tudriqev

vusolimogene oderparepvec · GALV-GP + GM-CSF (oncolytic HSV-1)
Human tumor cell lines in vitro; murine surrogate mRP1 in syngeneic lymphoma, colorectal and melanoma tumor-bearing mice

Vyjuvek

beremagene geperpavec · COL7A1 (HSV-1, topical)
Hypomorphic DEB mouse model and wild-type BALB/c mice: dermal transduction, COL7 expression, anchoring fibrils

Waskyra

etuvetidigene autotemcel · WAS (LVV, ex vivo HSC)
WAS-deficient mice transplanted with WAS LVV-transduced murine Lin- BM cells: increased B cells, platelets, granulocytes; improved T, B and DC function

Zevaskyn

prademagene zamikeracel · COL7A1 (RV-modified keratinocyte sheets)
Representative RVV-transduced keratinocyte grafts in mouse excisional wounds: cells stayed localized, Col7 expression up to 12 months

Zolgensma

onasemnogene abeparvovec · SMN1
SMNdelta7 SMA mouse model: single IV clinical vector lots gave dose-dependent survival improvement

Zynteglo

betibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC)
β-thalassaemic mouse model given BB305 LVV-transduced mouse BMCs: correction of the β-thalassaemic phenotype

Summary across all 26 gene therapies →