Novartis' AveXis unit announced Friday that the FDA has approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of patients under two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the SMN1 gene. The company noted that Zolgensma is the first gene therapy approved in the US for the treatment of patients with SMA, including those who are pre-symptomatic at diagnosis. Acting FDA Commissioner Ned Sharpless commented "today's approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases."
Novartis has priced Zolgensma, which is administered via a single intravenous infusion, at a cost of $2.13 million. The company also unveiled a plan that would allow insurers to pay for the gene therapy over five years, averaging out to annual installments of $425 000, which it said is comparable to prices for other treatments of rare disorders. Novartis previously estimated  that the treatment would be cost effective from $4.6 million to $5.4 million, although CEO Vas Narasimhan recently indicated that "we want to be far lower than that."
Meanwhile, the Institute for Clinical and Economic Review (ICER) issued a final report  last month concluding that Zolgensma would be cost-effective up to $900 000 based on a commonly used threshold per quality-adjusted life year (QALY) gained, and cost-effective up to $1.5 million using an alternate measure per life year (LY) gained. In an update to its final report issued Friday after the annoucement of Zolgensma's launch price, ICER noted that early results of the Phase III SPR1NT trial have been "encouraging" for use of Zolgensma in patients with presymptomatic SMA. As a result, the group estimates that Zolgensma could be considered cost-effective between $1.1 million to $1.9 million based on the commonly used threshold per QALY gained, and between $1.2 million to $2.1 million per LY gained.
ICER president Steven Pearson stated "it is a positive outcome for patients and the entire health system that Novartis...chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families."
AveXis president Dave Lennon remarked "we are partnering to accelerate coverage decisions with both government and commercial payers." He added "we are offering a pay-over-time model for this one-time treatment to accommodate the current structure of the US healthcare system and we have also established outcomes-based agreements with payers because we believe in the long-term value of Zolgensma and are willing to stand behind the therapy." AveXis stated that it is in advanced negotiations with more than 15 payers, some of which have already preliminarily agreed to terms (for additional analysis, see ViewPoints: Payers hopeful that Zolgensma may spark new thinking in gene therapy cost models ).
Approval of Zolgensma was supported by data from the ongoing Phase III STR1VE trial and previously completed Phase I START trial. In interim STR1VE results unveiled last month, 21 of 22 patients who received the gene therapy, also known as AVXS-101, were alive and event-free at the time of analysis, while previously reported data from the START trial showed that all 15 patients were alive and event-free at 20 months of age.
Analysts expect sales of Zolgensma to reach $2 billion by 2022, versus a projection of $2.2 billion for Biogen and Ionis Pharmaceuticals' SMA treatment Spinraza (nusinersen). Spinraza, approved by the FDA in 2016, requires infusion into the spinal canal every four months. Its list price of $750 000 for the initial year and $375 000 annually thereafter, has also been deemed excessive by ICER. The therapy generated $1.7 billion in revenue last year.
For related analysis, read ViewPoints: Novartis' Zolgensma sets the new status quo . See also, KOL Views Results: Room for all three leading SMA agents but interest especially keen for gene therapy, says leading paediatric neurologist .