Novartis' $2.1M-per-dose Gene Therapy Zolgensma (Onasemnogene Abeparvovec) Nominated for Priority Review in China for SMA Treatment

Zolgensma is a gene therapy for SMA that achieves sustained SMN protein expression through a single intravenous injection, thereby halting disease progression. This is Novartis' breakthrough in 2018 with$8.7 billionThe product acquired through the acquisition of AveXis was approved by the FDA for the first time in May 2019 and is used forType I SMA patients under two years old.

Previously, Novartis disclosed that its total investment in the development of Zolgensma reached 9.4 billion US dollars.(Approximately RMB 66.9 billion), the research and development costs are enormous. Relatively speaking, the pricing of Zolgensma is also not cheap, at$2.1 million per dose (approximately RMB 14.95 million)The priceRanked 7th on the "List of the World's Top 10 Most Expensive Drugs."

Screenshot source: DXY整理

From the perspective of sales, Zolgensma achieved $160 million in sales in the first quarter after its launch, and its sales reached $1.351 billion in 2021.Sales in 2022 further increased to$1.37 billion, with sales of $1.214 billion in 2024.

Insight database shows,April 22nd,Zolgensma First clinical trial initiated in China. This is aRandomized, Sham-Controlled, Double-Blind, Phase III Clinical Trial, Part of the Global Phase III STEER Study（NCT05089656）In the Chinese part, for treatment-naive Type 2 SMA patients aged 2-18 years, 20 participants are planned to be enrolled in China, with a total international enrollment of 125 participants.

In December 2024, Novartis announced the STEER study.Achieve the primary endpointAccording to the Novartis press release, this isThe first inUntreated SMA patients aged two years and aboveExperimental gene therapies with clinical benefits in China.

Screenshot source: Corporate official website

The research results show that, compared with the control group, receiving intrathecal injectionZolgensma Hammersmith Motor Function Scale Expanded Version for SMA Patients（HFMSE）Total score increased from baseline, which indicates the improvement in motor function of SMA patients. Detailed data will be disclosed at the upcoming medical conference in 2025.

In terms of safety,ZolgensmaThe safety profile was favorable, with similar rates of overall adverse events and serious adverse events between the two groups. The most common adverse events were upper respiratory tract infection, fever, and vomiting.

The Insight database shows that, currently, globally, only three new drugs for SMA have been approved, respectively.PTC TherapeuticsRisdiplamIonis Pharmaceuticals NusinersenAndNovartisTheZolgensmaIt is worth mentioning that the other two are chemical drugs, and only one from Novartis is a gene therapy drug.

Screenshot source: Insight database

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