SMA Treatment Landscape Heats Up as Another Generic Player Enters; Multiple Innovative Therapies Advance in Pipeline

　　【Pharmaceutical Network Industry DynamicsSpinal Muscular Atrophy (SMA) is a neurodegenerative rare disease. After onset, the motor function of patients gradually deteriorates until the end of life. For this disease, Biogen has developed a treatment drug, nusinersen sodium injection, which is also the world's first precision-targeted treatment for SMA. The drug is administered through intrathecal injection, directly delivering the medication to the cerebrospinal fluid surrounding the spinal cord, thereby improving motor function, increasing survival rates, and altering the course of SMA.
 

In China, Nusinersen Sodium Injection was launched on April 28, 2019. Subsequently, on December 3, 2021, the National Healthcare Security Administration announced that Nusinersen Sodium Injection had been officially included in the medical insurance coverage. After being included in the medical insurance, the drug saw a significant increase in sales volume. According to industry statistics, before entering the medical insurance catalog, its domestic sales in 2020 and 2021 were only 57.2413 million yuan and 155.1722 million yuan respectively. However, after being covered by medical insurance, the drug’s total sales in 2022 surged to 599 million yuan, with a growth rate of 286.53%. Nevertheless, it declined by 23.73% in 2023, reaching 460 million yuan. In 2024, the price of Nusinersen Sodium Injection dropped to 33,000 yuan per injection.
 

Currently, the generic version of Nusinersen Sodium Injection is attracting the layout of pharmaceutical companies in China. In October 2022, the National Medical Products Administration (NMPA) issued the "Reference Listed Drug Directory (61st Batch)," which included Nusinersen Sodium Injection in the reference listed drug directory. Against this backdrop, in September 2024, Yaoyou Pharmaceutical submitted an application for the market approval of its generic version of Nusinersen Sodium Injection, with hopes of securing the first generic approval for this product.
 

Information recently disclosed on the website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration shows that Qilu Pharmaceutical's Nusinersen Sodium Injection was submitted for marketing approval on January 3, making it the second company to file for the drug’s market approval following Yaoyou Pharmaceutical.
 

In addition to generic drugs, pharmaceutical companies in China are also actively developing innovative drugs for the treatment of SMA. For instance, EXG001-307 from Hangzhou Jiyin Biotechnology and GC-101 from Beijing Genecradle Therapeutics Co., Ltd. have both entered Phase 1/2 clinical trials.
 

Among them, the GC101 adeno-associated virus injection developed by Beijing Genecradle Therapeutics Co., Ltd. was included in the breakthrough therapy drug program by the CDE on December 11, 2024. Its proposed indication is Type 2 spinal muscular atrophy (SMA). In terms of drug type, GC101 is a therapeutic biological product, specifically designed for Type 2 spinal muscular atrophy. This drug attempts to repair or replace missing functions through gene therapy, potentially having a positive impact on patients' nervous systems. Recent studies show that patients treated with gene therapy have demonstrated significant improvements in motor function, undoubtedly paving a new path for the treatment of spinal muscular atrophy.
 

The IND application for SKG-0201, an adeno-associated virus gene therapy developed by Jiutian Biopharmaceuticals (Shanghai) / Lanyue Biopharmaceuticals Technology (Hangzhou), was accepted by the CDE on September 16, 2023. The drug has been approved for clinical trials and has entered Phase 1 clinical trials.
 

EXG001-307, developed by Hangzhou Jiayin Biotechnology, is a self-designed gene therapy drug targeting SMA through the SMN1 gene. It innovatively utilizes a neuron-specific promoter to enable specific expression in neural tissues (the therapeutic target organs), reducing toxicity in non-target tissues such as the liver and heart. Preclinical studies have preliminarily validated the design advantages of EXG001-307 in animals, and initial data from the Phase 1/2 clinical trials also demonstrate improved safety and significant efficacy.
 

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