AveXis, a Novartis company, announced the European Commission (EC) granted conditional approval for Zolgensma® (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.
In Europe each year, approximately 550–600 infants are born with SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene. Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, halting disease progression. According to Pediatric Neuromuscular Clinical Research (PNCR) natural history study of SMA, almost all patients under the age of five years of age will be under 21kg with some patients at 6, 7 or 8 weighing below 21 kg. AveXis is planning a product presentation that allows for treatment of patients weighing up to 21 kg and is working with the European Medicines Agency (EMA) to finalize supply timelines.
“The EC approval of Zolgensma is a significant milestone for the SMA community, and further underscores the substantial clinical value of the only gene therapy for SMA, bringing new hope to those impacted by this rare, but devastating disease.” said Dave Lennon, president of AveXis. “Even under the current pandemic conditions, the urgent need to treat SMA has resulted in access pathways in France and Germany for Zolgensma, a potentially life-saving medicine delivered in a single dose. Additionally, we have met with more than 100 stakeholder organizations across Europe to discuss our “Day One” access program to enable rapid access with customizable options designed to work within local pricing and reimbursement frameworks.”
SMA is a significant burden to the healthcare system in Europe with cumulative estimated healthcare costs per child ranging between €2.5 to €4 million within the first 10 years alone. Zolgensma is a transformative one-time gene therapy for a progressive genetic disease and is consistently priced worldwide under a value-based framework, however final pricing and reimbursement decisions are determined at the local level. Designed to work within existing, local pricing and reimbursement frameworks, the “Day One” access program offers ministries of health and reimbursement bodies a variety of flexible options that can be implemented immediately to support swift access and broad reimbursement.
The “Day One” access program ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments. The program maintains the integrity of the local pricing and reimbursement frameworks with a variety of customizable options including:
Retroactive rebates ensuring early access costs are aligned with negotiated prices following local clinical and economic assessment processes
Deferred payments and installment options allowing reimbursement bodies to manage budget impact during the early access phase
Outcomes-based rebates negotiated following clinical and economic assessments can be applied to patients treated during the early access period
Robust training for treating institutions on administration and follow-up care
Access to RESTORE, a global registry of patients who have been diagnosed with SMA that draws upon existing country registries
Immediate access to Zolgensma, aligned to the label, is available in France through the ATU framework and expected shortly in Germany.
“Today’s approval brings tangible progress in harnessing the transformational power of gene therapy,” said Dr. Eugenio Mercuri, Professor, Pediatric Neurology, Catholic University, Rome, Italy. “The approval of Zolgensma represents an i...
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