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In May 2018, the First Batch of Rare Disease Catalogue jointly released by five ministries and commissions in China became the main definition standard for rare diseases in China, including a total of 121 diseases
.
14 rare disease drugs are included in medical insurance
14 rare disease drugs are included in medical insuranceIn the National Basic Medical Insurance, Work-related Injury Insurance and Maternity Insurance Drug Catalogue issued by the National Medical Security Administration and the Ministry of Human Resources and Social Security, 7 new rare disease treatment drugs were added in 2020 and 2021 (Table 1), plus more than 40 categories of rare disease drugs covered by the previous medical insurance catalog, and the drugs included in the medical insurance catalogue have reached 62 categories, including 15 Category A and 47 Category B
.
In response to the proposal of some NPC deputies on "including rare disease drugs in the medical insurance catalog", the National Medical Insurance Administration issued a reply saying that all eligible rare disease drugs have been included in the payment scope of basic medical insurance, and some special rare disease drugs that far exceed the ability of funds and patients cannot be included in the basic medical insurance payment scope
.
Thirteen rare disease drugs have been approved for marketing in China
Thirteen rare disease drugs have been approved for marketing in ChinaFrom 2020 to 2021, a total of 13 rare disease treatment drugs were approved for marketing in China (Table 2).
Among the above rare disease drugs, ranalisumab (Dazeyou) is used to prevent hereditary angioedema (HAE).
Lanalilumab can inhibit the production of bradykinin by inhibiting plasma kallikrein, reduce the number of edema attacks, prevent asphyxia caused by laryngeal edema, and become the second domestic specific drug
for HAE after danazol.
In the treatment of transthyretin amyloidosis, two drugs have been approved for marketing, clobenzolic acid softgels (Vivanxin) for the treatment of adult wild-type or hereditary transthyretin amyloidosis cardiomyopathy, which is the only approved drug in the world for the treatment of transthyretin amyloidosis cardiomyopathy, which can reduce cardiovascular death and other adverse events; Chlorbenzolate glucosamine softgels (Veda Quan) are used for the treatment of transthyretin amyloidosis polyneuropathy
.
A variety of rare disease drugs were included in the varieties to be prioritized for review
A variety of rare disease drugs were included in the varieties to be prioritized for reviewIn addition to the drugs already on the market, some rare disease treatment drugs are included in the varieties
to be prioritized for review.
On June 10, 2020, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China announced that the new drug application for the SMN2 gene splicing regulator risdiplam oral solution powder proposed to treat spinal muscular atrophy will be included in the priority review
.
On September 29, CDE announced that two new drugs were included in the priority review: one of them was detuximab injection, which was used to treat a variety of neuroblastoma; The other is a Bruton's tyrosine kinase inhibitor (zebrutinib capsules), which was included in the priority review for adult Fahrenhei macroglobulinemia
.
On October 21, the CDE website announced that the two marketing applications of CD38 monoclonal antibody daratumab injection were included in the priority review variety, public information shows that daratumab is the world's first approved marketing of fully human monoclonal antibodies targeting CD38, earlier the drug has been approved in China for the treatment of adult patients with multiple myeloma, this submission is daratumumab in combination with lenalidomide and dexamethasone or with bortezomib and dexamethasone in combination, Treatment of adults with
multiple myeloma who have received at least first-line prior therapy.
On October 28, CDE announced that three new drug applications for brosolumab injection were to be included in the priority review, corresponding to 10 g/L, 20 g/L and 30 g/L, respectively
There are 3 different specifications, and the indications to be developed are all tumor-associated osteomalacia
.
On January 22, 2021, CDE announced that injectable stuximab was included in the priority review for the indication of multicenter Custerman disease adult patients with human immunodeficiency virus-negative and human herpesvirus 8-negative, which can inhibit the production
of abnormal immune cells by blocking interleukin-6-mediated inflammatory pathways.
On August 9, CDE included vitolaxen injection in the priority review and approval, and the gene deletion indicated for myotrophin was verified to pass 53
Duchenne muscular dystrophy for exon skipping therapy, an antisense oligonucleotide drug that works
by targeting dystrophin precursor mRNA.
Resources:
Resources:
[1] HE S, GAO Shiqi, HE Xinyue, et al.
New progress in the field of rare diseases in China (2020-2021)[J].
Union Medical Journal,2022,13(1):39-45.
DOI:10.
12290/xhyxzz.
2021-0248.
New progress in the field of rare diseases in China (2020-2021)[J].
Union Medical Journal,2022,13(1):39-45.
DOI:10.
12290/xhyxzz.
2021-0248.
