Information from the official website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA) shows that Qilu Pharmaceutical's Nusinersen Sodium Injection was submitted for marketing approval on January 3, making it the second company after Yaoyou Pharmaceutical to apply for the market launch of this drug. Once priced at 700,000 yuan per injection, Nusinersen Sodium has dropped to 30,000 yuan after being included in the national medical insurance.


Nusinersen is the world's first precision-targeted treatment for the rare disease Spinal Muscular Atrophy (SMA). By increasing SMN protein levels in SMA patients, it alters the disease progression and improves prognosis, making it a specific drug for treating SMA. In December 2016, the nusinersen injection developed by Biogen was approved for the first time in the United States and became the first specific drug for treating SMA approved by the U.S. Food and Drug Administration.


Original Research Drugs Achieve Increased Sales After Being Included in Medical Insurance


Since its launch, the global sales of Nusinersen Sodium have been on the rise, reaching a peak of $2.097 billion in 2019. The sales exceeded $2 billion in both 2019 and 2020. However, in 2019, with the emergence of competitors, the sales of Nusinersen Sodium began to decline. In May 2019, Novartis' SMN1 gene therapy (trade name: Zolgensma) was approved for marketing in the United States, becoming the world's first gene therapy for SMA. In August 2020, Roche's Risdiplam was approved for marketing in the United States. To date, these are the only three SMA drugs approved globally. Apart from Novartis' Zolgensma, the other two have been launched in China and included in the medical insurance catalog. In 2023, the global sales of Nusinersen Sodium were $1.741 billion, remaining the SMA-specific drug with the highest sales currently.


When Nusinersen Sodium Injection was first launched in China, it was priced at 699,700 yuan per unit, a price that made the vast majority of patients hesitate to seek treatment. Later, the price dropped to 550,000 yuan. In 2021, Xi'an Jiaotong University Second Affiliated Hospital (referred to as "Xi'an Jiaotong University Second Hospital") was embroiled in a public opinion whirlpool because of a hospital charge receipt containing a Western medicine fee of 550,000 yuan. This 550,000-yuan drug was Nusinersen Sodium Injection. A pediatric clinical doctor from a top-tier hospital in Beijing mentioned in an interview with The Beijing News in 2021 that Nusinersen Sodium treatment includes two parts: loading dose and maintenance dose. It needs to be injected intrathecally on the 1st day, the 14th day, the 28th day, and the 63rd day, and then every four months thereafter for life. According to the assistance program at that time, patients could enjoy a “1+5” assistance plan for the loading dose, which means buy 1 bottle and get 5 bottles free. The subsequent maintenance dose assistance plan was “1+2,” meaning that the patient’s annual treatment cost was 550,000 yuan.


In December 2021, Nusinersen was included in the National Medical Insurance Catalog through national medical insurance negotiations. The intense price negotiation that took place drew significant attention, particularly when Zhang Jin’ni, the representative of the national medical insurance negotiation team, said, "No small group should be abandoned," bringing tears to many families of SMA patients. On January 1, 2022, Nusinersen became accessible to more patients at a new medical insurance price of 33,000 yuan. According to a previous report by *China Medical Insurance*, from January to May 2022, the total number of patients using Nusinersen increased fivefold compared to before it was included in the medical insurance, and the total sales of the drug also nearly equaled its total sales in China over the two years since its launch. Additionally, according to data from Menet, in China's public medical institution terminals, the product's sales rose to nearly 600 million yuan in 2022, but fell by 23.73% in 2023 to 460 million yuan.


Two Chinese-produced generic drugs apply for marketing


And with the entry of domestically produced generic drugs, the price of Nusinersen may become more affordable. In October 2022, the National Medical Products Administration issued the "List of Reference Preparations for Generic Drugs (61st Batch)," which included Nusinersen Injection in the list of reference preparations, providing high-quality guidance for local pharmaceutical companies to produce generics.


In September 2024, Yaoyou Pharmaceutical submitted the market application for a generic version of Nusinersen Sodium Injection. This is also the first generic drug application in China under Category 4 for Nusinersen Sodium Injection, and it has the potential to become the first approved generic version of this product. Qilu Pharmaceutical Co., Ltd. is the second company to submit a market application for a generic version of Nusinersen Sodium Injection. However, even if the two domestically produced generics are approved, the core patent for Nusinersen Sodium in China does not expire until 2030, meaning that generics can only be marketed after the patent expires. Nevertheless, it can be foreseen that with the availability of generics, the financial burden on patients will be further reduced.


In addition to generic drugs, there are also pharmaceutical companies in China developing innovative drugs for the treatment of SMA. According to the Global New Drug Intelligence Database of Wisdom Bud queried by a reporter from The Beijing News, including the three approved drugs, there are 85 drug entries involving 341 development statuses. Among them, there are 36 drugs targeting SMN2 and SMN1. Regarding the R&D progress of these 36 drugs in China, besides the two approved special-effect drugs, Novartis' SMN1 gene therapy Zolgensma, which has been approved in overseas markets, is currently in Phase III clinical trials in China; EXG001-307 from Hangzhou Jiayin Biotechnology, Vesemnogene Lantuparvovec from Blueprint Biomedical (Guangzhou), and GC-101 from Beijing Jinsan Gene Technology have entered Phase 1/2 clinical trials. Notably, Beijing Jinsan Gene Technology's GC101 adeno-associated virus injection was included in the CDE’s Breakthrough Therapy Drug Program on December 11, 2024, with a proposed indication for Type 2 Spinal Muscular Atrophy. SKG-0201, an adeno-associated virus gene therapy developed by NineSky Biopharmaceuticals (Shanghai) / Lanyue Biomedical Technology (Hangzhou), has been approved for clinical trials and entered Phase I clinical trials. Enterprises such as Canbridge Pharma and Shenzhen TargetRx Biopharmaceuticals also have relevant layouts.


Reporter Wang Kala from The Beijing News

Proofread by Baoqing Liu