Novartis stands behind Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy

Basel, August 6, 2019 - Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma (onasemnogene abeparvovec-xioi). First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma. The FDA supports the continued marketing and use of Zolgensma for patients with spinal muscular atrophy (SMA) less than 2 years of age. We maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile. We remain steadfast that this important treatment remain available to pediatric patients with SMA less than 2 years of age.

On June 28th, AveXis voluntarily self-disclosed to the FDA and subsequently to other health authorities that some data previously submitted to the agency as part of our BLA package was inaccurate. Avexis had become aware of allegations of data manipulation in a specific animal testing procedure used in the development of the product. The assays in question were used for initial product testing and are not currently used for commercial product release. An investigation was immediately initiated to rapidly understand any implications and address the situation. Once we had interim conclusions from our investigations, we shared our findings with the FDA. As noted by the FDA, the data in question were a small portion of our overall submission and are limited to an older process no longer in use. 

At no time during the investigation did the findings indicate issues with product safety, efficacy or quality. We remain fully capable of releasing high-quality, fully compliant Zolgensma to patients in need. We have and will continue to work in close cooperation with the FDA to appropriately update our submission and address any quality gaps identified. We are committed to ensuring the highest levels of transparency and integrity with health agencies, as well as with the patients and providers we serve. We do not expect this to impact the timing of our ongoing Zolgensma regulatory filings and development programs. AveXis is committed to taking appropriate action to prevent future incidents across its portfolio of development programs.

About Zolgensma (onasemnogene abeparvovec-xioi)
Zolgensma (onasemnogene abeparvovec-xioi) is a proprietary gene therapy approved by the US Food and Drug Administration for the treatment of pediatric patients less than 2years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma represents the first approved therapeutic in a proprietary platform to treat rare, monogenic diseases using gene therapy. The therapy is also anticipated to receive approval in Japan and the European Union later this year.

About Spinal Muscular Atrophy (SMA) 
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons.8,9 The incidence of SMA is approximately 1 in 10,000 live births and it is the leading genetic cause of infant mortality.9,10 The most severe form of SMA is Type 1, a lethal genetic disorder characterized by rapid motor neuron loss and associated muscle deterioration, resulting in mortality or the need for permanent ventilation support by 24 months of age for more than 90 percent of patients if left untreated.11

Indication
Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patient less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. 

Limitation of Use:
The safety and effectiveness of repeat administration of Zolgensma have not been evaluated. 

The use of Zolgensma in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated.


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