Novartis' Zolgensma Receives CDE's Implicit Approval for Clinical Trials in China

On January 7, 2022, the Information Disclosure Center of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration showed that the clinical trial application for Zolgensma (OAV101 injection), an AAV gene therapy drug for treating Spinal Muscular Atrophy (SMA) developed by Novartis, had received tacit approval for clinical trials in China. Previously, on October 21, 2021, the clinical trial application for this drug had been accepted.

Zolgensma was approved by the FDA in May 2019 for the treatment of spinal muscular atrophy in patients under 2 years old. This therapy uses the scAAV9 vector to deliver the normal SMN1 gene into the patient's body through intravenous infusion, producing normal SMN1 protein, thereby improving the function of affected cells such as motor neurons.

Zolgensma, the world's first one-time gene therapy for SMA, is priced at $2.125 million (approximately RMB 13.58 million), also known as the "most expensive drug in history." Zolgensma has been approved in more than 40 countries. According to Novartis' financial report, Zolgensma's sales reached $920 million in 2020, a year-on-year increase of 151%. In the first half of 2021, its sales amounted to $634 million, a year-on-year increase of 69%. At this growth rate, Zolgensma is expected to enter the "billion-dollar club."

Currently, three SMA therapies have been approved worldwide: Zolgensma, Biogen's Spinraza, and Roche's Evrysdi, the latter two of which have already been approved in China.

Spinraza (Nusinersen Sodium Injection) was approved by the National Medical Products Administration in February 2019 for the treatment of 5q spinal muscular atrophy (accounting for approximately 95% of all SMA cases), making it the first drug approved in China to treat SMA. Previously priced at nearly 700,000 RMB per dose in China, Spinraza's cost was reduced to 550,000 RMB per injection after a price adjustment in 2021. In December 2021, Spinraza was included in the latest national medical insurance directory; according to related reports, the price dropped to 33,000 RMB per dose, with a reduction exceeding 90%. After being incorporated into the medical insurance, Spinraza has been scheduled for treatment by multiple children in Tianjin.

In terms of sales performance, Spinraza's global sales in 2020 were $2.05 billion, and in the first half of 2021, they were $1.02 billion, a year-on-year increase of 3.7%.

Evrysdi was approved for marketing by the FDA in August 2020, with global sales reaching 243 million Swiss francs (approximately 265 million US dollars) in the first half of 2021. In China, Evrysdi (Chinese trade name: Aimiaxin) was approved for marketing by the National Medical Products Administration in June 2021 for the treatment of SMA in patients aged two months and above. This is also the first orally administered disease-modifying treatment for SMA to be approved in China. The price of Evrysdi in China is 63,800 yuan (60mg/bottle). According to reports, the pricing of Aimiaxin in the United States depends on the patient's weight, with an annual treatment cost of approximately 2.37 million yuan.