Take stock | more than 20 new Chinese drugs that will be eligible for FDA orphan drugs by 2020

According to incomplete statistics, more than 20 innovative products from Chinese biopharmaceutical companies will be eligible for orphan drugs by the FDA in 2020.
these products, there are PD-1/L1 inhibitors, CAR-T therapy, antibody association drugs (ADCs), dual-specific antibodies, gene therapy and other innovative therapies, involving JAK, Claudin 18.2, SHP2, Bcl-2, HER2 and other targets.
among them, Bcl-2 inhibitor APG-2575, MDM2 inhibitor APG-115, BTK inhibitor Zebutini of Baiji Shenzhou, and resonant biologic anti-PD-1 monoantitor Ripley monoantigen were eligible for 3 or more orphan drugs in 2020.
addition, products from Keystone Pharmaceuticals, Stone Pharmaceuticals Group, Xinda Bio, Corninger, and Platinum Pharmaceuticals are eligible for this special qualification.
this article, we have an introduction to some of the products that have attracted much attention.
1, Drugs: APG-2575 Research and Development Enterprise: AXA Pharmaceutical Treatment Diseases: Fahrenheit GlobulinEmia (WM), Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma (MM) APG-2575 is A new oral Bcl-2 selective small molecule inhibitor developed by Yasheng Pharmaceuticals restores the mechanism of procedural death of tumor cells (apoptosis) by selectively inhibiting Bcl-2 protein, thereby killing tumors and intended to be used to treat a variety of blood malignancies.
2020, APG-2575 has won three orphan drug qualifications in the United States for WM, CLL and MM.
January, APG-2575 was again eligible for the FDA's fourth and fifth orphan drug for the treatment of acute myeloid leukemia (AML) and fable lymphoma (FL).
, APG-2575 has been licensed for phase 1b/2 clinical trials in the United States, China and Australia and is advancing the clinical development of multiple blood tumor adaptations worldwide simultaneously.
2, drugs: APG-115 research and development enterprises: Aachen Pharmaceuticals Treatment of diseases: stomach cancer, AML, soft tissue sarcoma (STS) APG-115 by Aachen Pharmaceuticals independently developed, is an oral, highly selective small molecule MDM2 inhibitor.
the drug has a high binding affinity for MDM2 and restores p53 tumor inhibition by blocking MDM2-p53 interactions.
2020, APG-115 has been eligible for three FDA orphan drugs, with symptoms such as stomach cancer, AML, and STS.
Currently, APG-115 is conducting clinical studies in China and the United States on a number of single-drug or combination treatment options for multiple solid tumors and blood tumors, including advanced solid/metastatic melanoma, salivary adenocarcinoma, AML/bone marrow hyperplate abnormal syndrome (MDS).
3, drugs: Zebtinib Research and Development Enterprise: Baiji Shenzhou Treatment of diseases: lymph node edge area lymphoma (MZL), spleen edge area lymphoma, mucosal-related lymph node outer edge area lymphoma Zebutinib is an oral BTK small molecule inhibitor independently developed by Baiji Shenzhou, which is characterized by maximizing the specific binding of BTK targets, thereby minimizing the toxic side effects of targeting.
zebtinib has shown good efficacy and safety in clinical trials for a variety of B-cell cancers.
November 2019, the FDA announced accelerated approval of Zebutinib for the treatment of treated sleeve lymphoma (MCL).
June 2020, Zebtinib was approved for listing in China for adult MCL patients who have received at least one treatment in the past and adult CLL/small lymphocyte lymphoma (SLL) patients who have received at least one treatment in the past.
addition, Zebtinib has been awarded three FDA orphan drug qualifications in 2020, with MZL, spleen limbic lymphoma, and mucosal-related lymph nodes.
4. Drugs: Terripri single anti-drug research and development enterprises: Junshi biological treatment of diseases: soft tissue sarcoma, nasopharyngeal cancer, mucosal melanoma Ripley monoantibas is Junshi Bio independently developed anti-PD-1 monoantibasic drugs, approved in China in 2018 for non-excisive or metastatic melanoma treatment.
applications for the listing of two new adaptations for nasopharyngeal and urethra cancers have been submitted in China and have been included in the priority review process.
the U.S., Ripley's single-drug treatment for nasopharyngeal cancer has been recognized by the FDA as a breakthrough therapy, and Junshi Bio expects to submit the product's first biological product licensing application (BLA) to the FDA this year for the adaptation.
addition, the FDA granted Ripley one fast-track qualification (mucosal melanoma) and three orphan drug eligibility (mucosal melanoma, nasopharyngeal cancer, soft tissue sarcoma).
, Ripley's monoantigeno-anti-acetone treatment for mucosal melanoma was approved by the FDA in March 2020.
Phase 1b clinical data for the first-line treatment of advanced mucosal melanoma showed that patients were treated with an objective remission rate (ORR) of 48.3% and a disease control rate (DCR) of 86.2%, with a medium progression-free survival (mPFS) of 7.5 months.
5, Drugs: KN046 Research and Development Enterprise: Corning Jerry Treatment Diseases: Thymus Epithelioid Tumor, HER2 Positive or HER2 Low Expression Gastric Cancer and Gastroesophageal Connection Cancer KN046 is Corning Jerry's own research and development of PD-L1// CTLA-4 dual-specific antibodies, whose innovative design includes: the use of different mechanisms of CTLA-4 and PD-L1 monolithic antibody fusion composition, can target the high expression of PD-L1 tumor microencase and remove the suppression of tumor immunity Treg.
U.S., it has FDA approval to go directly to Phase 2 clinical trials.
September 2020, KN046 was granted FDA-granted orphan drug for the treatment of thymus epitheliote tumors.
two registered clinical trials of KN046 are currently under way.
December 2020, KN046 and KN026 combination therapies were awarded orphan drug qualification by the FDA for the treatment of HER2-positive or HER2 low-expression gastric cancer and gastroesophageal joint cancer (GC/GEJ).
KN026 is an anti-HER2 dual-specific antibody developed by CorningErry that binds to both non-overlapping tables of HER2, resulting in more effective HER2 signal blocking.
studies have shown that KN046 combined with KN026 showed good results in HER2-positive solid tumors.
, CorningEry is currently conducting a critical Phase 2 clinical study (SEARCH-01) of this combination therapy, including a key clinical study for the treatment of advanced stomach cancer and gastroesophageal concomitant cancer.
6. Drugs: Xindili monoanti-anti-research and development enterprises: Cinda biological treatment diseases: T-cell lymphoma, esophageal cancer Xindili mono-antibody as a PD-1 monoclonal antibody, in December 2018 by China's State Drug Administration (NMPA) approved the treatment of relapsed /refractic classic Hodgkin lymphoma.
, Xindili monoanti-resistance was approved in China as a new adaptive disorder for first-line treatment of non-squamous non-small cell lung cancer (NSCLC).
It is worth mentioning that the new adaptive drug listing application (sNDA) for the treatment of first-line squamous NSCLC patients, for the treatment of second-line squamous NSCLC patients and first-line liver cancer patients has also been accepted by NMPA.
the United States, the drug was approved by the FDA in 2020 as an orphan drug for the treatment of T-cell lymphoma and esophageal cancer.
7, Drugs: CLDN18.2 CAR-T (CT041) Research and Development Enterprise: Kozi BioTherapy Disease: Gastric Adenocarcinoma and Esophageal Gastric Conjugenal Adenocarcinoma CT041 is a humanized anti-CLD developed by Cozi Bio N18.2 In vitro CAR-T cell injection, it is proposed to develop adaptations for the treatment of ADVANCED gastric adenocarcinoma/esophageal gastric adenocarcinoma and pancreatic cancer that are positive for CLDN18.2 and progress or relapse after systematic treatment.
October 2020, Kozi Bio announced that the FDA had granted its CLDN18.2 CAR-T in-study drug orphan drug for the treatment of stomach adenocarcinoma and esophageal gastric conjuage adenocarcinoma.
8. Drugs: For Reilly Pearl Monoantitor Research and Development Enterprise: Baiji Shenzhou Treatment of Diseases: Gastric Cancer (including gastroesophageal binding cancer) For Reilly Pearl Monoantitor is designed to avoid binding to Fc subjects in macrophages, humanized IgG4 monoclonal antibodies for the immune checkpoints of PD-1.
the end of December 2019, Reilly-Zhudan resistance was officially approved in China for the treatment of patients with recurring or refractic classic Hodgkin's lymphoma (R/R cHL) who have underwent at least second-line system chemotherapy.
April 2020, its second adaptive urethra cancer was approved in China.
U.S., Tyrelli Pearl was approved by the FDA in May 2020 for the treatment of stomach cancer, including gastroesophageal combined cancer.
, there are currently 15 registered clinical trials in China and around the world.
9, Drugs: CS1003 Research and Development Enterprise: Keystone Pharmaceuticals Treatment Of Diseases: Hepatocellular Carcinoma (HCC) CS1003 is a humanized IgG4 monoclonal antibody targeted at PD-1 developed by Keystone Pharmaceuticals.
preliminary data from its Monotherapy Phase 1a clinical study showed that CS1003 was safe and toned, and anti-tumor activity was observed in a variety of tumor types.
2020, the product was eligible for FDA orphan drugs for the treatment of hepatocellular carcinoma.
has launched a global Phase 3 clinical trial of a combination therapy consisting of CS1003 and Lenvatinib, and will be the first subject to be given the drug in December 2019.
Lenvatinib is a tyrosine kinase inhibitor (TKI) for the treatment of patients with advanced hepatocellular carcinoma.
10, drugs: Shugli monoanti (CS1001) Research and development enterprises: Keystone Pharmaceuticals Treatment of diseases: T-cell lymphoma Shugli monoantigen is developed by Keystone Pharmaceuticals in the study of anti-PD-L1 monoclonal antibodies, is the closest to the human body of natural G-type immunoglobulin 4 (IgG4) monoantigen.
it has a lower risk of immunogenicity and associated toxicity in patients, which gives it a potentially unique advantage.
in China, the listing application for the first-line treatment of patients with advanced scaly and non-squamous non-small cell lung cancer with CS1001 combined chemotherapy has been accepted by NMPA.
it is worth mentioning that the adaptation of CS1001 for relapsed or refractic endocyst natural killer cells/T-cell lymphoma (R/R ENKTL) has been identified by FDA breakthrough therapy and incorporated into NMPA breakthrough treatment varieties.
, the product's adaptation to treat T-cell lymphoma has been approved for FDA orphan drugs.
11, Drugs: HBM9167 Research and Development Enterprise: and Platinum Pharmaceuticals Therapeutic Diseases: Nasopharyngeal Cancer HBM9167 (also known as KL-A167) is a recombinant humanized IgG1 monoclonal antibody for PD-L1 developed by Columbine Pharmaceuticals Holdings, and Platinum Pharmaceuticals owns the global development and commercialization rights of the drug candidate outside Greater China.
existing research, HBM9167 has shown good safety.
2020, phase 2 trials of HBM9167 were approved by the FDA and awarded orphan drug eligibility for the treatment of nasopharyngeal cancer.
12, Drugs: APG-1252 (palcitoclax) Research and Development Enterprise: AXA Pharmaceuticals Treatment of Diseases: Small Cell Lung Cancer (SCLC) APG-1252 is AXA Pharmaceuticals' independent research and development of B Cl-2/Bcl-xL dual-target inhibitors, which can restore apoptosis by selectively inhibiting Bcl-2 and Bcl-xL proteins, are clinically intended to treat SCLC, NSCLC, lymphoma and other tumors.
September 2020, APG-1252 was eligible for FDA orphan drugs for the treatment of small cell lung cancer.
APG-1252 is currently conducting clinical phase 1 dose climbing trials in the United States and Australia for patients with advanced cancer, 1b/2 stage trials in the United States for patients with relapse recurring SCLC with combined yew alcohol, and single-drug clinical phase 1 dose climbing trials for patients with SCLC in China.
13, Drugs: HQP1351 (Oribatini) Research and Development Enterprise: Asaan Pharmaceuticals Treatment of Diseases: Chronic Myeloid Leukemia (CML) HQP1351 is a third generation BCR-ABL/KIT inhibitor, targeting BCR-ABL mutants, including mutants carrying T315I mutations.
the drug has completed phase 1 clinical trials in drug-resistant CML patients in China, preliminary data show that it has achieved significant anti-tumor activity and good safety characteristics.
the drug is currently being treated in China in the treatment of difficult-to-treat CML patients in the key Phase 2 clinical study.
note that the product has submitted applications for new drug listing in China and has been included in the priority review, it has also obtained FDA fast-track eligibility and orphan drug eligibility.
14, Drugs: KN035 (Nwolly monoanti, envafolimab) Research and development enterprises: Corning Jerry / Thoughty Treatment disease: advanced bilionic cancer KN035 is PD-L1 mono-domain antibody Fc fusion protein, developed by Corning Jerry and Iddi, Sonic Pharmaceuticals has exclusive rights to market and promote its in China in all tumor adaptations.
according to Corning Jerry's announcement, KN035's differentiation advantage is that it can be injected under the skin and is suitable for patients who are not suitable for infusion, which can greatly improve patient compliance.
January 2020, the FDA granted orphan drug eligibility for KN035 advanced bile gallbladder cancer.
is currently conducting simultaneous clinical trials in China, the United States and Japan for multiple tumor adaptations.
, NMPA has accepted applications for the listing of new drugs for KN035 and has included the application in its priority review.
15, Drug: Jack tini research