Since 2020, the domestic FDA orphan drug qualification products at a glance

"Pharmaceutical Network Market Analysis" orphan drugs are rare drugs, refers to the prevention, treatment, diagnosis of rare diseases of drugs, because of the small number of rare diseases, the drug market is small, research and development costs are high, difficult, many rare disease patients face the situation of no drugs available.
2019 Orphan Drugs Report, released in 2019 by Evaluate Pharma, a pharmaceutical market research organization, shows that the orphan drug market will grow at a compound annual rate of 12.3% from 2019 to 2024, reaching $242 billion by 2024.
about 20 million people with rare diseases in China, but only 5% of rare diseases currently have treatment options.
, orphan medicine will be a new blue sea for innovative pharmaceutical companies.
years, with the emergence of positive policies for orphan drugs, the community has paid more and more attention to the treatment of rare diseases, and promoted the accelerated listing of rare disease drugs in China.
Among them, orphan drug qualification will help the product in the United States follow-up research and development, registration and commercialization to enjoy certain policy support, including clinical trial fee tax credits, new drug application fee exemption and enjoy 7 years of market exclusive rights, etc., will reduce research and development investment to a certain extent, conducive to promoting clinical trials.
2020, a number of domestic pharmaceutical companies have been qualified for FDA orphan drugs, the author according to public information combing, made a brief inventory.
Recently, Gan Li Pharmaceuticals announced that the company's clinical phase 1 candidate drug GLR2007 has been approved by the U.S. Food and Drug Administration (FDA) orphan drug qualification.
it is understood that the drug is used to treat polygonal glioblastoma (GBM) and is designed to study drug safety, toerability and best administration strategies in patients with advanced solid tumors.
Junshi Bio September 18, Junshi Bio announced that the company's product Tripri single anti-use for the treatment of soft tissue sarcoma was awarded by the FDA orphan drug qualification, which is also the third orphan drug qualification obtained by Ripley single anti-treatment mucosal melanoma and nasopharyngeal cancer has been approved by the FDA orphan drug qualification.
soft tissue sarcoma is a rare heterogeneous tumor, its pathological type is complex, tumor heterogeneity is obvious, the current clinical treatment of soft tissue sarcoma drugs are mainly cytotoxic anti-tumor drugs, its adverse reactions are relatively large, and immunotherapy drugs are relatively lacking, so the development of immunotherapy drugs has important clinical significance and value.
March 27th, Junshi Bio announced that the company's own research and development of anti-PD-1 monoanti-Terripri monoanti (commodity name: Toyi) in the treatment of mucosal melanoma in the FDA orphan drug qualification.
, Aachen Pharmaceuticals announced that the FDA has awarded the company the qualification of APG-115 orphan drug for the treatment of stomach cancer in the original innovative drug MDM2-p53 inhibitor APG-115.
this is the first FDA-granted orphan drug qualification for APG-115, which has previously qualified three orphan drugs in two varieties.
It is understood that APG-115 is the first MDM2-p53 inhibitor to enter the clinical stage in China, has been in China and the United States to carry out a number of clinical studies to treat solid tumors and blood tumors, and in the treatment of stomach cancer in preclinical research has shown considerable potential.
in early September, Corning Jerry Pharmaceuticals-B announced that the FDA had awarded KN046 the orphan drug.
KN046 is a recombinant humanized PD-L1/CTLA-4 dual-specific antibody developed for Jiangsu Corning Jerry Biopharmaceutical Co., Ltd., a wholly owned subsidiary of the company, for the treatment of thymus epitheliote tumors.
this is also Corning Jerry Pharmaceuticals to obtain the second orphan drug qualification.
, Cinda Bio announced on April 14 that the FDA has granted the company's PD-1 inhibitor Dabershu (Cindili monoanti) orphan drug eligibility for treatment of esophageal cancer.
this is the third orphan drug qualification obtained by Thyda Bio Dabershu.
previously, the European Medicines Agency (EMA) and the FDA have also granted Dabshu) orphan drug qualifications, in which the EMA-granted adaptation is exocytocyte lymphoma, the FDA-granted adaptation is T-cell lymphoma.