Request an invite

Hemgenix: how long were the pivotal tox studies?

etranacogene dezaparvovec · Factor IX
Longest pivotal study
26 weeks
“At 26 weeks following IV administration of 5x1012 to 9x1013 gc/kg HEMGENIX in NHPs, high levels of vector DNA were detected in a dose-dependent manner in the liver.”
✓ VerifiedFDA summary basis for approval, p. 11Source ↗
Pivotal GLP studies
GLP single-dose IV studies in C57Bl/6 mice and cynomolgus monkeys (13-26 weeks observation); mouse paternal germline transmission study
“The single IV dose toxicology studies and the analysis of the safety parameters were performed under GLP compliance.”
✓ VerifiedEMA assessment report, p. 30Source ↗
Route in tox
Intravenous (single dose), mimicking clinical route
“IV-route of administration, the clinical route, is used in all toxicological studies.”
✓ VerifiedEMA assessment report, p. 30Source ↗

Same question, other gene therapies

Adstiladrin

nadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical)
22 (derived from the dosing and observation schedule)

Beqvez

fidanacogene elaparvovec · Factor IX
104 weeks

Casgevy

exagamglogene autotemcel · BCL11A enhancer (CRISPR-Cas9, ex vivo HSC)
20 weeks

Elevidys

delandistrogene moxeparvovec · Micro-dystrophin
24 weeks

Fayuvi

rebisufligene etisparvovec · SGSH (AAV9)
26 weeks

Genglycos

pariglasgene brecaparvovec · G6PC (AAV8)
26 weeks

Glybera

alipogene tiparvovec · LPL (AAV1)
26 weeks

Imlygic

talimogene laherparepvec · GM-CSF (oncolytic HSV-1)
12 (derived from the dosing and observation schedule)

Itvisma

onasemnogene abeparvovec (intrathecal) · SMN1 (AAV9, intrathecal)
52 weeks

Kebilidi

eladocagene exuparvovec · AADC
26 weeks

Kresladi

marnetegragene autotemcel · ITGB2 (LVV, ex vivo HSC)
4 weeks

Lenmeldy

atidarsagene autotemcel · ARSA (LVV, ex vivo HSC)
52 weeks

Luxturna

voretigene neparvovec · RPE65
31 weeks

Lyfgenia

lovotibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC)
26 weeks

Otarmeni

lunsotogene parvec · OTOF (AAV, intracochlear)
27 weeks

Papzimeos

zopapogene imadenovec · HPV6/11 antigens (gorilla adenovirus)
3 weeks

Roctavian

valoctocogene roxaparvovec · Factor VIII
26 weeks

Skysona

elivaldogene autotemcel · ABCD1 (LVV, ex vivo HSC)
13 (derived from the dosing and observation schedule)

Strimvelis

autologous CD34+ cells transduced with ADA (no INN) · ADA (gamma-retroviral, ex vivo HSC)
16 weeks

Tudriqev

vusolimogene oderparepvec · GALV-GP + GM-CSF (oncolytic HSV-1)
26 weeks

Vyjuvek

beremagene geperpavec · COL7A1 (HSV-1, topical)
9 (derived from the dosing and observation schedule)

Waskyra

etuvetidigene autotemcel · WAS (LVV, ex vivo HSC)
64 weeks

Zevaskyn

prademagene zamikeracel · COL7A1 (RV-modified keratinocyte sheets)
8 weeks

Zolgensma

onasemnogene abeparvovec · SMN1
26 weeks

Zynteglo

betibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC)
26 weeks

Summary across all 26 gene therapies →