Novartis C.E.O. Defends Company ’s Decision to Withhold False Data From the F.D.A.
Responding to the agency ’s stern rebuke, Vas Narasimhan, the company’s executive, tried to reassure investors that Novartis did not intentionally deceive the F.D.A. while seeking approval for its $2.1 million gene therapy.
Date: August 26, 2019 Issue #: 1579Summary: On August 6, 2019, the FDA issued a statement on data manipulation/inaccuracy issues withZolgensma, a virus vector-based gene therapy approved for one-time IV treatment of children
Abstract Antisense oligonucleotides (ASOs) are a widely used form of gene therapy, which is translatable to multiple disorders. A major obstacle for ASO efficacy is its bioavailability for in vivo and in vitro studies. To overcome this challenge we use cell-penetrating peptides (CPPs) for systemic delivery of ASOs. One of the most advanced clinical uses of ASOs is for the treatment of spinal muscular atrophy (SMA). In this chapter, we describe the techniques used for in vitro screening and analysing in vivo biodistribution of CPP-conjugated ASOs targeting the survival motor neuron 2, SMN2, the dose-dependent modif...
AveXis knowingly submitted manipulated data in its application for Zolgensma, the first gene therapy approved for spinal muscular atrophy, the agency says; the product will remain on market for now.Medscape Medical News
The FDA approved the gene therapy Zolgensma to treat children with a severe form of spinal muscular atrophy. The drug costs $2.125 million for a one-time treatment, CBS News reported.
The failure to report the issue has not put patients at risk, the F.D.A. said, but the drugmaker could face criminal and civil penalties.
On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy, but was informed recently by the product ’s manufacturer about a data manipulation issue
aro S Abstract Neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), Parkinson's, Alzheimer's, and Huntington's disease affect a rapidly increasing population worldwide. Although common pathogenic mechanisms have been identified (e.g., protein aggregation or dysfunction, immune response alteration and axonal degeneration), the molecular events underlying timing, dosage, expression, and location of RNA molecules are still not fully elucidated. In particular, the alternative splicing (AS) mechanism is a crucial player in RNA processing and represents a fundamental de...
CONCLUSION: Gene therapies have the potential to significantly influence the course of neuromuscular diseases. First positive intermediate results have been published and the first treatment has recently been approved in the USA. Long-term data on sustained effects and toxicity of gene therapies are not yet available. These novel treatment options will present new challenges for the healthcare systems concerning diagnosis, treatment and reimbursement. PMID: 31286145 [PubMed - as supplied by publisher]
AbstractOnasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the humansurvival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA). It has been developed by AveXis, a Novartis company, and was approved in May 2019 in the USA for the treatment of paediatric patients aged
Date: July 29, 2019 Issue #: 1577Summary: The FDA has approved onasemnogene abeparvovec-xioi (Zolgensma– Avexis), an adeno-associated virus vector-based gene therapy, for one-time IV treatment of children