Novartis' gene therapy Zolgensma approved in Europe for SMA

Novartis' AveXis division announced Tuesday that the European Commission granted conditional approval to Zolgensma (onasemnogene abeparvovec) for the treatment of certain patients with spinal muscular atrophy (SMA). Specifically, the one-time gene therapy is indicated for patients with 5q 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.

Zolgensma gained FDA approval last year with the product priced at around $2.1 million for a single intravenous infusion. At the time, the company unveiled a plan that would allow insurers to pay for the gene therapy over five years, averaging out to annual installments of $425 000, while the drugmaker later said that it would give away about 100 doses for free under a global managed-access programme.


Value-based pricing

Dave Lennon, president of AveXis, remarked "we have met with more than 100 stakeholder organisations across Europe to discuss our "Day One" access programme to enable rapid access with customisable options designed to work within local pricing and reimbursement frameworks." Lennon added "even under the current pandemic conditions, the urgent need to treat SMA has resulted in access pathways in France and Germany for Zolgensma."

According to Novartis, the "Day One" access programme is designed to ensure that the cost of patients treated before national pricing and reimbursement agreements are in place aligns with the value-based prices negotiated following clinical and economic assessments. The company noted that the programme features a number of options including retroactive rebates, deferred payments and installment options, as well as outcomes-based rebates.


Third regulatory approval

The approval in Europe marks the third for Zolgensma, which along with the US is also cleared in Japan. Novartis noted that regulatory decisions are anticipated in Switzerland, Canada, Australia, Argentina, South Korea and Brazil in late 2020 or early 2021.

Approval by the European Commission is based on results from the Phase III STR1VE-US and Phase I START trials, while the comparable late-stage STR1VE-EU study is ongoing. In the completed trials, Zolgensma demonstrated prolonged event-free survival, rapid motor function improvement, often within one month of dosing, and sustained milestone achievement, including the ability to sit without support, crawl and walk independently. Novartis noted that further supportive data came from the ongoing Phase III SPR1NT trial, with results demonstrating a rapid, age appropriate major milestone gain in pre-symptomatic patients.

The drugmaker said that in Europe it is planning a product presentation that allows for treatment of patients weighing up to 21 kg and is working with the European Medicines Agency to finalise supply timelines. Zolgensma, which Novartis gained via its $8.7-billion takeover of AveXis in 2018, generated sales of $170 million in the first quarter of this year.

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