The 19 Chinese companies will be eligible for 35 FDA orphan drugs by 2020

In recent years, the trend of "going out to sea" of Chinese pharmaceutical companies has become more and more obvious, not only in terms of the external authorization of the project, but also in the enthusiasm of the "China-U.S. double report", especially in recent years, more and more domestic pharmaceutical companies have obtained FDA orphan drug qualification projects.
the U.S. Passed the Orphan Drugs Act in 1983 to develop new drugs for rare diseases that sicken fewer than 200,000 people in the United States, the FDA will provide policy support.
's FDA-granted orphan drug eligibility provides policy support for subsequent research and development, registration, and commercialization, including tax credits for clinical trial fees, exemptions for new drug applications, and seven years of market exclusive access without patent impact.
according to incomplete statistics, 26 products from 19 Chinese companies have obtained a total of 35 FDA orphan drug qualifications (by accreditation), accounting for 8% of the fda's annual eligibility for orphan drugs.
among them, Yasheng Pharmaceuticals was identified as the biggest winner with 8 orphan drug qualifications with 4 drug bags, followed by Baiji Shenzhou and Junshi Bio with 4 and 3 respectively.
From the field of disease, Chinese companies in 2020 to obtain FDA orphan drug qualification is mostly concentrated in the field of cancer, accounting for up to 86% (30/35), the largest number of six cancers are stomach cancer (4), T-cell lymphoma (3), soft tissue sarcoma (2), esophageal cancer (2), nasopharyngeal cancer (2), liver cell carcinoma (2).
From the point of view of drug targets, immuno-checkpoint PD-1/PD-L1, apoptosis signal protein Bcl-2, MDM2 and popular target BTK, CLDN18.2 are the most qualified drugs by Chinese companies in the FDA.
a brief introduction to the orphan medicine of each company.
2020 is a bumper year for Assassin Pharma, with the core variety Aeriebatinib declared on the market in China and four new drugs being approved from the FDA for eight orphan drugs.
APG-115 is an MDM2-p53 inhibitor that has a high binding affinity for MDM2, restores p53 tumor inhibition activity by blocking MDM2-p53 interactions, and is the first MDM2-p53 inhibitor to enter clinical phase in China.
APG-115 has won three orphan drug qualifications for stomach cancer, acute myeloid leukemia (AML) and soft tissue sarcoma (STS).
gastric cancer is considered a rare disease with fewer than 200,000 people in the United States; AML is a highly heterogeneous heterogeneous hemolytic malignancies, with 19940 new AML cases expected in the United States by 2020 11,180 people die from the disease; STS is a generalization of malignancies that occur in soft tissues (fat, muscle, nerve, fibrous tissue, blood vessels) throughout the body, with about 12,000 confirmed cases and 5,000 deaths in the United States each year.
APG-2575 is a Bcl-2 selective small molecule inhibitor that restores the programed death mechanism (apoptosis) of tumor cells by selectively inhibiting the Bcl-2 protein.
three adaptation certificates for APG-2575 eligibility for orphan drugs were Fahrenheit globulinemia (WM), chronic lymphocytic leukemia (CLL), and multiple myeloma (MM).
WM is a rare lymphocyte tumor that accounts for less than 2% of non-Hodgkin's lymphoma patients in the United States; CLL is an adult leukemia characterized by a large number of cloned B lymphocytes in lymphatic tissues such as peritonal blood, bone marrow, spleen and lymph nodes, and in 2020 CLL patients in the United States were lower than MM is a plasma cell cloning disease that can cause symptoms such as hypercalcemia, anemia, kidney damage, bone damage, accounting for 1.8% of all cancers, 18.2% of hematositomas, and an annual incidence rate of about 69 per 100,000 in the United States.
APG-1252 can repair apoptosis by selectively inhibiting Bcl-2 and Bcl-xL proteins, and its orphan drug is recognized as small cell lung cancer (SCLC).
SCLC is a rare, highly invasive malignant tumor, which accounts for about 13-15% of lung cancer type (by tissue pathology credit type), and its five-year survival rate is low.
treatment options are few, especially for patients with relapsed/resoccic SCLC, and the effectiveness of all treatment methods is limited.
Arrbatini (HQP1351) is a new oral third-generation Bcr-Abl inhibitor that targets Bcr-Abl mutations, including T315I, which was approved by the FDA in May as an orphan drug for chronic myeloid leukemia (CML).
is a rare malignant blood disease with an annual incidence rate of about 19,000 per 100,000 people in the United States.
Baiji Shenzhou (4 items) for Reilly pearl monoantigen is designed to avoid binding to Fc-infested in macrophages, against the immuno-checkpointing subject PD-1 humanized IgG4 monoclonal antibody.
be eligible for an orphan drug for stomach cancer, including gastroesophageal cancer, awarded by the FDA.
Reilly-Zhudan was approved in China at the end of 2019 for the treatment of patients with relapsed or refractic classic Hodgkin's lymphoma (R/R cHL) who underwent at least second-line system chemotherapy;
Zebtinib, an oral BTK small molecule inhibitor developed in Baiji Shenzhou, was also awarded by the FDA in August this year for three orphan drug qualifications for lymph node lymphoma, spleen edge lymphoma, and mucosal-related lymph node outer edge tumor.
Lymphoma (MZL) is a group of inert lymphatic system malignancies that originate from B cells in the edge zone of the lymph nodes, accounting for 8% to 15% of all non-Hodgkin's lymphomas.
zebtinib was approved by the FDA in November 2019 for the treatment of treated sleeve lymphoma (MCL).
June this year, Zebtinib was approved for listing in China for adult MCL patients who have received at least one treatment in the past and adult CLL/SLL patients who have received at least one treatment in the past.
(3 items) Ripley monoanti is China's first successfully listed domestic PD-1 drug.
March, May and September 2020, the FDA issued three adaptive orphan drug qualifications for mucosal melanoma, nasopharyngeal cancer, and soft tissue sarcoma to Terripri monoantigen.
mucosal melanoma is a rare melanoma subtype in white people, accounting for about 1.3% of all melanoma patients; Globally, nasopharyngeal cancer is actually a rare disease, affecting only about 1.2 out of every 100,000 people, with more than 40% of the world's nasopharyngeal cancer patients coming from China and the incidence in southern China particularly high, about 10 times that of the north and 20 times that of the world.
Xinda Bio (2) Xindili monoantigen was approved by the NMPA in December 2018 for the treatment of relapsed/refractic classic Hodgkin's lymphoma, and was added to the new National Health Insurance Directory in November 2019, becoming the only PD-1 to enter the new National Health Insurance Directory at that time.
the drug was approved by the FDA in March and April 2020 for T-cell lymphoma and esophageal cancer orphans.
T-cell lymphoma (TCL) is a type of non-Hodgkin's lymphoma (NHL), the incidence rate of about 10% of the NHL, with racial and regional characteristics, heterogeneity, invasive, high malignancy and poor efficacy characteristics;
incidence of esophageal cancer in the United States is very low, with about 300,000 people dying each year from esophageal cancer worldwide, and China has the highest incidence of esophageal cancer in the world.
Pharmaceuticals (2 items) Shugli monoanti (CS1001) is an anti-PD-L1 monoclonal antibody in the study.
the drug was approved by the FDA in July and October this year for the treatment of hepatocellular carcinoma and T-cell lymphoma orphans.
noted that Shugli monoantigen has also been identified by the FDA as a breakthrough therapy for the treatment of adult recurrence or refractic external natural killer cell/T cell lymphoma.
CDE received a listing application for its combined chemotherapy first-line treatment for non-small cell lung cancer (squamous and non-squamous) in November.
CS1003 is a humanized recombinant IgG4 monoclonal antibody targeting PD-1 and is being developed for immunotherapy for a variety of tumors.
fda in July this year granted CS1003 orphan drug eligibility for treatment of hepatocellular carcinoma.
liver cell carcinoma is a very invasive disease, China is a big country of liver cancer, coupled with the lack of effective treatment drugs, resulting in poor overall prognosis of patients.
Corninger (2) Nwolly monoanti is a subdermal injection of PD-1/L1 antibodies developed by Corning Jerry/Thinkdy, and bile pathogenic cancer adaptation was eligible for FDA orphan drugs in January 2020.
is currently a rare adenocarcinoma in the world, with an overall five-year survival rate of 2% to 30%.
countries such as the United States, the United Kingdom, France and Australia have a lower incidence rate of less than 6 per 100,000, although in some countries and regions in the Asia-Pacific region, the incidence of bile tube cancer is relatively high.
KN046 is a PD-L1/CTLA-4 dual-specific antibody, thymus epitheline tumor (TET) adaptation certificate was approved by the FDA orphan drug in September this year.
TAT includes a variety of chest tumors, non-surgical or metastatic thymus epithal tumor prognostode difference.
For patients who failed chemotherapy treatment with platinum-containing drugs, there is currently no approved standard treatment, the objective mitigation rate of after-line chemotherapy or targeted therapy is less than 20%, and the medium survival of patients with multiple-line therapy recurrence is less than 12 months, and effective therapeutic drugs are urgently needed to improve patient outcomes.
Group (2) SYSA1801 is a Cludin-18.2 all-human monoclonal antibody-MMAE drug conjudder (ADC) developed by Conjupro Biotherapeutics, a subsidiary of Stone Pharmaceutical Group, and has been eligible for FDA orphan drug adaptation.
the current treatment of stomach cancer drugs and therapy is very limited, mainly using conventional chemotherapy and surgical excision, etc. , is not satisfied with the clinical needs.
NBL-015, an all-human anti-Cludin 18.2 monoclonal antibody developed by Nova Rock Biotherapeutics, a subsidiary of Stone Pharmaceuticals, was approved as an orphan drug for pancreatic cancer in December.
pancreatic cancer, which kills more than 430,000 people worldwide each year, is one of the most malignant tumors, with an overall one-year survival rate of only about 24% and a five-year overall survival rate of about 9%, ranking fourth in the U.S. for cancer-related deaths.
other companies in addition to the above-mentioned seven companies have won a number of orphan drug qualifications, as well as Ze-Chi Pharmaceuticals, Dizhe Pharmaceuticals, Garcos and other 12 enterprises have also obtained orphan drug qualifications.
The following is a brief introduction to these products: TNP-2092 is a multi-target conjunctivity molecule developed by Danno Pharmaceuticals to achieve biofilm sterilization by inhibiting the synergy of RNA polymerase, DNA rotary enzyme and topological isomerase IV, and was approved by FDA Orphan Pharmaceuticals in January 2020 for the treatment of artificial joint infections.
the product has been approved by fda qualified anti-infected products (QIDP), priority review, fast track and orphan drug qualification.
Cotrani (STP705) is a nucleic acid interference drug (siRNA therapy) developed by Sanno Pharmaceuticals with anti-tissue fibrosis and anti-inflammatory therapeutic effects, which uses two targets, TGF-beta1 and COX-2 siRNA oligonucleotides, to enhance the import efficiency and safety of the drug through histamine-lysine copolymer polypeptide nanoparticles (PNP) to enhance the import efficiency and safety of the drug to block fibrosis and inflammation.
February, the drug was awarded the title of orphan drug for hepatocellular carcinoma (HCC) by the FDA, and previously qualified as an orphan drug for the treatment of primary sclerosis bileitis and bile tube cancer.
NIP292 is a small molecule ROCK inhibitor developed by China Resources Pharmaceuticals with multiple mechanisms such as anti-inflammatory, anti-fibrosis, dilation of blood vessels, repair of endoskin damage.
February 2020, China Resources Pharmaceuticals announced that NIP292 had been approved by the FDA as an orphan drug for the treatment of idiolytic pulmonary fibrosis (IPF).
KL-A167 (HBM9167) is a recombinant humanized IgG1 monoclonal antibody for PD-L1 introduced by Platinum Pharmaceuticals from Columbote and has the right to develop and commercialize worldwide, except in Greater China.
February 2020, and Platinum Pharmaceuticals announced that HBM9167 had been approved by the FDA for Clinical Phase II trials and was awarded orphan drug qualification for the treatment of nasopharyngeal cancer.
HY1003 is a new biologic drug developed by Wuhan Yanyuan Bio-Using Plant Expression Platform, which was approved as DRU-201 by FDA Orphan Drug in February 2020 9-7242, used to treat emphysema caused by α-1 antitase deficiency (AATD), which is associated with about 800,000 people in the United States and about 3 million worldwide.
Idala's rightol tongue tablet, a compound developed by Inoi Bio, was approved by the FDA in August for the treatment of amyotrophic lateral sclerosis (ALS) orphan drug.
the drug's active ingredients are Idalafon and (-) -2- alcohol, the former a free-based sculven agent and the latter an inflammatory inhibitor, which work together to reduce nerve cell damage and death.
caused by freegens is thought to be one of the causes of nerve cell death in ALS patients.
GLR2007 is a cell cycle protein-dependent kinase 4/6 (CDK4/6) inhibitor that was approved by the FDA in September this year for the treatment of polygonal glioblastoma (GBM).
GBM is one of the most morbid and invasive primary brain tumors, and although surgery, chemotherapy and radiation therapy can achieve some results, the patient survives in the middle