Zolgensma, most expensive drug to land China

A drug hailed as the "most expensive drug in the world" has been recently approved by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). Manufactured by Novartis Gene Therapies, the drug is called Zolgensma for treating patients with Spinal Muscular Atrophy (SMA).

The clinical trial application was submitted in China as early as October 21, 2021, and now it has received an implied license, which is good news for babies and children suffering with a rare genetic disorder.

The gene therapy called "Zolgensma" does not come cheap, with a reported list price of $2.15 million（equivalent to 13.48 million yuan） per dose. At present, Zolgensma has been approved in nearly 40 countries and regions around the world.

In 2020, Zolgensma was included in Japan's medical insurance, and patients only need to pay 30% of the fee. In March 2021, it was included in the national medical service system of the United Kingdom.

Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting in muscles used for movement. Babies born with severe type 1 SMA usually have a life expectancy of just two years.

At present, three SMA therapies have been approved globally. In addition to Zolgensma, Biogen's Spinraza and Roche's Evrysdi have also been approved in China.

Since 2017, the price negotiation mechanism has cut the price of marketed drugs. China updated its National Medical Insurance Drug List (NRDL) annually, with an average price cut of 50.64%.

In the negotiation of 2021, 7 drugs for rare diseases were included in the list, including Spinraza. So far, the price of a dose of Spinraza has dropped from 700,000 yuan to more than 30,000 yuan, benefitting 20 plus SMA patients in 11 Chinese provinces.

After the negotiation, more drugs for rare diseases will be included in the NRDL, so that more patients can afford them.

Online sources; Trans-editing by Mo Yingyi