Zolgensma

GPTKB entity

Statements (64)
Predicate Object
gptkbp:instance_of gptkb:gene_therapy
gptkbp:administration_age up to 2 years old
gptkbp:advocacy supported by patient advocacy groups
gptkbp:affects studied for long-term effects
gptkbp:approves gptkb:European_Union
gptkb:FDA
gptkb:United_States
gptkb:muscular_dystrophy
May 2019
gene therapy for SMA
gptkbp:availability available in multiple countries
gptkbp:capacity 5 m L
gptkbp:clinical_trial Phase 3
positive outcomes
conducted in multiple countries
over 100 patients
STR1 VE
gptkbp:collaborations collaborations with research institutions
gptkbp:developed_by gptkb:Novartis
gptkbp:development gptkb:Ave_Xis
gptkbp:disease_targeted gptkb:SMA_type_1
gptkbp:duration single dose
gptkbp:effective_date gptkb:2019
gptkbp:eligibility diagnosed with SMA
gptkbp:formulation liquid solution
gptkbp:funding significant investment in research
gptkbp:healthcare administered by healthcare professionals
https://www.w3.org/2000/01/rdf-schema#label Zolgensma
gptkbp:indication children with spinal muscular atrophy
children under 2 years with SMA
gptkbp:ingredients gptkb:onasemnogene_abeparvovec
gptkb:onasemnogene_abeparvovec-xioi
gptkbp:is_effective_against improves motor function
gptkbp:is_monitored_by requires follow-up monitoring
gptkbp:manufacturer gptkb:Novartis
gptkbp:marketed_as gptkb:Zolgensma
Zolgensma brand name
gptkbp:mechanism_of_action gene replacement therapy
gptkbp:patient_access_programs available patient access programs
gptkbp:patient_population SMA patients
gptkbp:post-marketing_studies ongoing post-marketing studies
gptkbp:price approximately $2.1 million
gptkbp:product_type gptkb:pharmaceuticals
gptkbp:provides_guidance_on included in treatment guidelines
gptkbp:public_perception high public interest
gptkbp:regulatory_compliance approved
received multiple regulatory approvals
gptkbp:research_phase preclinical studies
gptkbp:route_of_administration intravenous
intravenous infusion
gptkbp:safety_features generally well tolerated
gptkbp:serves gptkb:muscular_dystrophy
gptkbp:service_frequency once
gptkbp:side_effect potential liver toxicity
gptkbp:supply_chain complex supply chain management
gptkbp:target_audience infants and young children
gptkbp:target_gene gptkb:SMN1_gene
gptkbp:treatment high success rate
improve survival
gptkbp:treatment_impact significant impact on quality of life
gptkbp:treatment_paradigm changed treatment paradigm for SMA
gptkbp:type_of_insurance covered by some insurance plans
gptkbp:bfsParent gptkb:Novartis
gptkbp:bfsLayer 4