According to data from the China Rare Disease Alliance, there are more than 20 million patients with various rare diseases in China, and more than 200,000 new patients are added every year, and the demand for drugs is large, but there is still a large unmet demand
for rare diseases in China.
In the face of this market that cannot be ignored, coupled with the release of China's rare disease catalog, more and more rare disease drugs have been included in medical insurance, and the marketing approval of rare disease drugs has accelerated, multinational pharmaceutical companies have increased their layout
.
It is reported that at the recent CIIE, which has attracted much attention from the industry, multinational pharmaceutical companies including AstraZeneca, Sanofi, Takeda Pharmaceutical, Teva and other multinational pharmaceutical companies have all carried new drugs for rare diseases, which shows that the domestic rare disease drug market is very attractive to foreign pharmaceutical companies
.
Among them, AstraZeneca, as an "old guest" of the Expo, exhibited its first product in the field of rare diseases, eculizumab injection, this year
.
This is a prescription drug
that has been approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children.
Among them, paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive and life-threatening rare disease of the blood system, which is more common in young adults in China
.
Atypical hemolytic uremic syndrome (aHUS) is complement-mediated thrombotic microangiopathy (CM-TMA)
caused by an overreaction of the complementate system to attack healthy cells.
Previously, AstraZeneca said that in order to drive the development of global rare disease medicine, the group has integrated the superior resources of Ruison Pharmaceutical in the field of rare diseases, and is making every effort to accelerate the introduction of global innovative rare disease drugs into China, and actively promote the localization of
rare disease diagnosis and treatment in China.
Teva, a leading Israeli pharmaceutical company, participated in the Expo for the first time, and this time brought a new drug for rare diseases, Titan
.
It is reported that the product is the first approved deuterated drug in China, used to treat Huntington's disease, commonly known as "chorea", "chorea disease" is a rare autosomal dominant neurodegenerative disease, because most patients will appear involuntary dance-like movements, the disease currently lacks effective treatment drugs, Titan or bring hope to patients
.
At present, Antaltan has passed the priority review and approval process and has been approved for listing in China and has entered the medical insurance catalog
.
Takeda Pharmaceutical's Malibavir is an Asian debut at the Expo, which is mainly used for the treatment of anti-cytomegalovirus (CMV) infection or disease after transplantation, and is currently the first drug approved in the world for the treatment of refractory (with or without drug resistance) (R/R) cytomegalovirus (CMV) infection or disease after adult organ transplantation or hematopoietic cell transplantation
.
Studies have shown that the proportion of patients in the maribavir treatment group who meet the defined endpoint of viral clearance is more than twice that in the conventional antiviral treatment group, and that there are fewer
associated side effects than conventional antiviral therapy.
In addition, Sanofi brought a number of new drugs for rare diseases to the Expo, including the first exhibition of the world's first approved nanobody drug Cablivi, which is the first drug
targeted for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).
Thrombotic thrombocytopenic purpura is a serious thrombotic microangiopathy, which is a rare clinical emergency and critical disease, and the disease progresses rapidly and affects the life and health
of patients.
The cause is not clear, but it is considered to be related
to infection and immune abnormalities.
The clinical manifestations of the disease are diverse, and the main clinical features include thrombocytopenic purpura, microangiopathic hemolysis, psychoneurologic symptoms, fever, and renal impairment
.
Another blockbuster rare disease drug unveiled by Sanofi is the new generation of enzyme replacement therapy (ERT) drug Nexviazyme for Pompe disease, which has been granted "breakthrough therapy" designation by the FDA and is expected to greatly improve respiratory function and exercise capacity
in patients with Pompe disease.
According to reports, Pompe disease is a rare autosomal recessive genetic disease, if patients are not treated in time, it will lead to irreversible progressive damage to multiple organs and systems, and Nexviazyme is expected to bring new treatment options
to patients.
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