Novartis Submits Zolgensma (Onasemnogene Abeparvovec) for Market Approval in China
Novartis
Drug Development and Manufacturing
Zolgensma is a gene therapy for SMA that achieves sustained expression of the SMN protein through a single intravenous injection, thereby halting disease progression. This was acquired by Novartis in 2018 for...$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 forPatients under two years old with Type I SMA.
Previously, Novartis disclosed that its total investment in the development of Zolgensma reached 9.4 billion US dollars.(Approximately RMB 66.9 billion), the R&D cost is enormous. Relatively, the pricing of Zolgensma is also not cheap, at$2.1 million per dose (approximately RMB 14.95 million)The priceRanked 9th on the "Top 10 Most Expensive Drugs Globally".
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 increased further 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-naïve Type 2 SMA patients aged 2-18 years, 20 participants are planned to be enrolled in China, with 125 participants internationally.
In December 2024, Novartis announced the STEER study.Achieved the primary endpoint. According to the Novartis press release, this isThe first inSMA patients aged two years and above who have not received treatmentExperimental gene therapies with clinical benefits in China.
The research results showed that, compared with the control group, the group receiving intrathecal injectionZolgensma Hammersmith Motor Function Scale Expanded Version for SMA Patients（HFMSE）Total score increased from baseline, which indicates the improvement of motor function in SMA patients. Detailed data will be announced 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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