Will sky-high-priced rare disease drugs be withdrawn from the market? Pharmaceutical companies respond: Special drug imports will be reintroduced into the country


The International Rare Disease Day has just passed, and the news that a rare disease drug will be withdrawn from the market in China affects people’s hearts. This drug, “Weimingzan”, is the only specific drug approved in the world for the treatment of mucopolysaccharidosis type IVA.

On March 1, a staff member of Bio-Wanli Pharmaceutical Company, the pharmaceutical company that produces Weimingzan, told reporters that the drug has not been withdrawn from the domestic market and there are sufficient domestic stocks of the drug. It is still too early to discuss how the drug will be imported into the country. ” It’s just that the IDL (Imported Drug Registration Certificate) will expire. If patients have other needs after expiration, we will find ways to meet them.”

A medical expert who has been engaged in research on the diagnosis and treatment of genetic diseases for a long time said in an interview with a news reporter that prenatal diagnosis and third-generation test tube technology can reduce the probability of second children suffering from mucopolysaccharidosis type IVA, and called for the establishment of a joint venture between medical insurance, enterprises, and Multi-party payment models such as charity, commercial insurance, and local relief funds can reduce the burden of medication for patients with rare diseases and make sky-high-priced drugs affordable for patients with rare diseases.

Pharmaceutical companies say Weimingzan still has drugs in stock in China

Mucopolysaccharidosis is a rare disease whose main clinical manifestations include growth retardation, skeletal deformities, etc. Without intervention and treatment, some patients will gradually become paralyzed until death.

Weimingzan is the world’s only drug for the treatment of mucopolysaccharidosis type IVA developed by the American company Biomim. It needs to be taken for life and can delay symptoms.

In 2019, Weimingzan was approved for marketing in China, and Weimingzan’s Import Drug Registration Certificate (IDL) in China will expire in May 2024.

In June last year, drug manufacturer BioPro Wanli Pharmaceuticals told reporters that the company had decided not to renew Weimingzan’s import drug registration certificate in China. This means that patients with mucopolysaccharidosis type IVA will face the dilemma of having no cure.

However, after the efforts of all parties, things turned around

On March 1, Zheng Yu, president of Zhengyu Mucopolysaccharide Rare Disease Care Center, told reporters that family members of patients with mucopolysaccharidosis type IVA actively reported to pharmaceutical companies, drug regulatory authorities and the National Health Commission that Zhengyu Mucopolysaccharide Rare Disease Care Center The Care Center reported the medication difficulties of the mucopolysaccharide patient group to the China Rare Disease Alliance.

Thanks to the unremitting efforts of all parties, the good news finally came

She learned that recently, the China Rare Disease Alliance is negotiating with the manufacturer of Weimingzan and plans to reintroduce Weimingzan into the domestic market as a special drug import after its IDL expires. “The price of the drug will then be higher than before.” Be low”.

On the same day, in response to this matter, the reporter contacted the staff of BioPro Wanli Pharmaceutical Company. According to his disclosure, Weimingzan has not withdrawn from the domestic market. “It’s just that the IDL has expired. After the expiration, if patients have other needs, we will find ways to meet them.” He also emphasized that “we have drugs in stock in China, and domestic patients must “You can buy medicines,” and the China Rare Disease Alliance has indeed contacted the company about follow-up issues regarding the expiration of the IDL. It believes that it is too early to discuss how the drugs can be imported into the country.

Public information shows that “Weimingzan” applied for marketing in mainland China in 2018. With the green channel opened by the Chinese government for rare disease drugs, it received marketing approval from the National Medical Products Administration in May 2019 and was listed in my country’s The first batch of “List of New Overseas Drugs in Urgent Clinical Needs”.

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