DRUGapprovedgene therapy
onasemnogene abeparvovec
Brand names: Zolgensma
Mechanism
AAV9-mediated gene replacement delivering functional SMN1. Single IV infusion.
Approved indications
type1presymptomatic
Related claims (50)
| Type | Predicate | Conf | Source |
|---|---|---|---|
| drug efficacy | Onasemnogene abeparvovec-xioi (Zolgensma) is a costly drug (clinical trial result) | 80% | 37003633 |
| survival | Onasemnogene abeparvovec treatment is associated with an event-free survival of 96.5% (95%CI: 90.76 to 99.54, I2 = 32%, n = 66) in patients with SMA type 1 (clinical trial result). | 69% | 38713659 |
| motor function | Patients with ≥four SMN2 gene copies treated with onasemnogene abeparvovec monotherapy demonstrated improvements in motor function (observed in patient cohort). | 65% | 39260228 |
| safety | High vector dose or supraphysiological levels of SMN may cause adverse events such as liver toxicity associated with onasemnogene abeparvovec. | 65% | 38413837 |
| survival | Onasemnogene abeparvovec treatment is associated with a global survival of 97.56% (95%CI: 92.55 to 99.86, I2 = 0%, n = 67) in patients with SMA type 1 (clinical trial result). | 64% | 38713659 |
| motor function | Onasemnogene abeparvovec treatment is associated with a CHOP-INTEND score ≥ 40 points proportion of 87.28% (95%CI: 69.81 to 97.83, I2 = 69%, n = 67) in patients with SMA type 1 (clinical trial result). | 64% | 38713659 |
| gene expression | Intravenous administration of onasemnogene abeparvovec to SMA model mice resulted in sustained expression of survival motor neuron (SMN) protein (observed in mouse model). | 59% | 34980814 |
| drug efficacy | At a list price of $2.125 M, onasemnogene abeparvovec is cost-effective compared to nusinersen for SMA1 patients treated before age 2 years. | 59% | 33708361 |
| drug efficacy | SPR1NT trial results suggested that onasemnogene abeparvovec is effective in treating pre-symptomatic SMA (clinical trial result) | 59% | 37731145 |
| drug efficacy | Onasemnogene abeparvovec (Zolgensma®) was approved for the treatment of patients with SMA with up to three copies of the SMN2 gene or the clinical presentation of SMA type 1. | 59% | 32763124 |
| motor function | Intravenous administration of onasemnogene abeparvovec to SMA model mice resulted in improvement of motor function (observed in mouse model). | 59% | 34980814 |
| drug efficacy | Onasemnogene abeparvovec, a vector-based gene therapy, is an approved therapeutic drug for SMA treatment that improves motor function. | 59% | 35352647 |
| drug efficacy | For patients with three SMN2 copies, onasemnogene abeparvovec (OA) was less costly, with a similar efficacy profile compared with nusinersen (clinical trial result). | 59% | 40569556 |
| drug efficacy | Greatest improvements were seen in subjects who received onasemnogene abeparvovec treatment within 2 months of birth (clinical trial result) | 59% | 38223869 |
| drug efficacy | Intrathecal onasemnogene abeparvovec administration, at fixed, low doses, may enable treatment of heavier patients ineligible for weight-based intravenous dosing. | 59% | 36911944 |
| motor function | SMA I patients treated with onasemnogene abeparvovec show significant improvement on CHOP-INTEND motor assessment over 24 months. | 59% | 41854353 |
| drug efficacy | Children treated with onasemnogene abeparvovec have reduced sleep disordered breathing over time. | 59% | 37778092 |
| motor function | Stabilisation or improvement in bulbar or respiratory function was observed in 20/21 (95.2%) patients treated with onasemnogene abeparvovec (clinical trial result). | 59% | 35170254 |
| drug target | Gene therapies such as onasemnogene abeparvovec for spinal muscular atrophy (SMA) utilize adeno-associated virus 9 (AAV9) for targeted gene delivery. | 59% | 40018025 |
| survival | Onasemnogene abeparvovec showed the highest survival rate (95% [95% CI: 88, 100]) (clinical trial result). | 59% | 39604484 |
| drug efficacy | Efficacy data on demonstration of motor milestones suggest that Latin American patients with SMA may benefit from onasemnogene abeparvovec treatment (clinical trial result). | 59% | 40778350 |
| drug efficacy | Onasemnogene abeparvovec-xioi has shown significant efficacy in improving motor function (clinical trial result). | 59% | 40630496 |
| drug efficacy | This case series and accompanying literature review reinforce the efficacy and safety of onasemnogene abeparvovec in the treatment of SMA type 1 (observed in patient cohort). | 59% | 40630496 |
| drug efficacy | Onasemnogene abeparvovec is indicated for the treatment of paediatric patients with spinal muscular atrophy(SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene. | 59% | 40329332 |
| drug efficacy | Onasemnogene abeparvovec provides evidence for sustained clinical durability of the therapeutic dose (clinical trial result). | 59% | 33999158 |
| survival | All children with three SMN2 copies treated with onasemnogene abeparvovec before symptom onset survived without permanent ventilation at 14 months (clinical trial result) | 59% | 35715567 |
| motor function | At 6 months post-dose, 91.5% of infants treated with onasemnogene abeparvovec achieved a clinically significant ≥ 4-point improvement in CHOP INTEND score from baseline (clinical trial result). | 59% | 40622452 |
| gene expression | Onasemnogene abeparvovec delivers a functional transgene to restore SMN protein expression. | 59% | 35437095 |
| drug efficacy | Onasemnogene abeparvovec was efficacious in improving motor functioning in children with SMA (clinical trial result). | 59% | 38450080 |
| drug efficacy | Younger patients demonstrated superior therapeutic efficacy post-onasemnogene abeparvovec administration (clinical trial result) | 59% | 41283841 |
| drug efficacy | The treatment of SMA with nusinersen, onasemnogene abeparvovec, and risdiplam changes the disease phenotype with changes in motor function far exceeding any improvement in respiratory and nutritional function (clinical trial result). | 59% | 38905882 |
| drug efficacy | Patients receiving onasemnogene abeparvovec as switch therapy after prior treatment show slower motor improvement compared to monotherapy. | 59% | 41854353 |
| drug efficacy | Therapy with onasemnogene abeparvovec led to an improvement in the clinical picture of the treated subjects (clinical trial result) | 59% | 38223869 |
| drug efficacy | Onasemnogene Abeparvovec is a potent treatment for SMA leading to significant motor function improvements (observed in patient cohort). | 59% | 36855136 |
| drug efficacy | Nusinersen and onasemnogene abeparvovec treatment is associated with modest motor improvements in a patient with spinal muscular atrophy type 0 (clinical trial result) | 59% | 32515646 |
| drug efficacy | Only transitory improvement in functional outcomes was observed after Onasemnogene abeparvovec treatment (observed in patient cohort). | 59% | 39111255 |
| motor function | Better progress in motor function was observed in individuals who received Onasemnogene abeparvovec-xioi (OA) earlier and who were presymptomatic (clinical trial result). | 59% | 37149951 |
| motor function | Rapid and clinically significant improvements in motor function were observed for onasemnogene abeparvovec-treated patients with symptomatic SMA type 1 (clinical trial result). | 59% | 40622452 |
| drug efficacy | The limited dose of onasemnogene abeparvovec did not lead to marked clinical improvement (clinical trial result). | 59% | 40705999 |
| drug efficacy | The 2nd-generation vector showed better safety and improved efficacy in SMA mouse model in a head-to-head comparison between the second-generation vector and a benchmark vector, identical in design to onasemnogene abeparvovec. | 59% | 38413838 |
| safety | Diverging patterns in adverse reaction reporting suggest a stabilizing safety profile for nusinersen and potential emerging safety signals for risdiplam and onasemnogene abeparvovec. | 59% | 41149786 |
| drug efficacy | Onasemnogene abeparvovec-xioi has shown significant efficacy in improving survival rates (clinical trial result). | 59% | 40630496 |
| biomarker | SMN2 copy number is a predictor of motor improvement in SMA I patients treated with onasemnogene abeparvovec. | 59% | 41854353 |
| drug efficacy | Therapeutic plasma exchange and rituximab successfully reduced the AAV Ab titers to the range of eligibility for onasemnogene abeparvovec (clinical trial result). | 59% | 40705999 |
| motor function | 16/21 (76%) children gained at least one WHO motor milestone following treatment with onasemnogene abeparvovec (clinical trial result). | 59% | 35170254 |
| safety | Six (18%) had adverse events that were considered serious and related to onasemnogene abeparvovec (clinical trial result). | 59% | 34536405 |
| drug target | Nusinersen, onasemnogene abeparvovec-xioi, and risdiplam all target upregulation of SMN protein. | 59% | 35942665 |
| drug target | Onasemnogene abeparvovec-xioi is a treatment for SMA targeting SMN1 gene replacement. | 59% | 33003005 |
| drug efficacy | Nusinersen and onasemnogene abeparvovec treatment is associated with a 30-point improvement in CHOP-INTEND score (clinical trial result) | 59% | 32515646 |
| motor function | Treatment with onasemnogene abeparvovec has led to rapid motor function improvement, often within one month of dosing (clinical trial result). | 59% | 34980814 |
Off-Target Findings (0)
No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.