On May 13, 2024, PUMCH held a handover ceremony for the temporary import of viltolarsen, a drug for Duchenne Muscular Dystrophy (DMD), a rare disease. PUMCH President Zhang Shuyang received the little blue box symbolizing hope for patients with this rare disease from Zhou Song, President of China Sinopharma International Corporation, witnessed by Li Linkang, Executive Director of the China Alliance for Rare Diseases, and Wang Qing, Deputy Director of the Management Committee of the Beijing Capital Airport Economic Zone. Then, Dai Yi, Deputy Director of the Department of Neurology, PUMCH, issued the first prescription for this drug on the Chinese mainland. “This is amazing! We can’t thank PUMCH enough!” said the parents of a child patient, with tremendous joy on their faces.

▲Dai Yi, Deputy Director of the Department of Neurology, PUMCH, wrote the first prescription for the drug

▲A pharmacist from the Department of Pharmacy, PUMCH was giving instructions on the drug use

DMD is a hereditary neuromuscular disease and the most common type of progressive muscular dystrophy. It was included in China’s first catalog of rare diseases. Approved in the United States in February 2017 for the treatment of DMD, viltolarsen is characterized by a lower incidence of adverse reactions, better patient compliance, and overall better efficacy than other corticosteroids.

To meet the medication needs of DMD patients in China, PUMCH, according to the requirements on using drugs in urgent clinical demand, submitted an application to the National Medical Products Administration for the temporary import and use of viltolarsen for DMD patients at PUMCH, the designated venue. With the concerted efforts of multiple departments, it took only 62 working days for the approval to be granted and the drug to arrive. The drug arrived at PUMCH at 9 a.m. on May 11, 2024, and on May 13, eight children from across the country received their medication.

▲The drug handover ceremony, from left to right: Li Linkang, Executive Director of the China Alliance for Rare Diseases; Zhang Shuyang, Prsident of PUMCH; Zhou Song, President of China Sinopharma International Corporation; Wang Qing, Deputy Director of the Management Committee of the Beijing Capital Airport Economic Zone

During the drug handover ceremony, President Zhang Shuyang stated that the introduction of one drug for one rare disease is just the beginning. The success of this model promises the introduction of more drug varieties and benefits for more patients with various diseases. President Zhang Shuyang pointed out that rare diseases are a matter of public welfare and governance and thus warrant the joint efforts of government departments, medical institutions, and research institutes to create a favorable social environment. She called for continued collaboration among these parties to improve the availability of medications for rare diseases, aiming for “lower incidence, early diagnosis, and access to medical care, drugs and medical insurance coverage”. As the only national-level leading hospital of the National Rare Disease Diagnosis and Treatment Collaborative Network, PUMCH, always patient-centered, will explore and establish a “PUMCH model” as the “Chinese solution” for the diagnosis and treatment, and research, and medical insurance coverage of rare diseases.

Written by Chen Xiao

Photographed by Sun Liang

Edited by Xiao Xiong

Translated by Liu Haiyan

Reviewed by Wang Yao