Last week 13 treatments won the orphan drug qualification Junshi, Cinda have been shortlisted

Drug Name: Axicabagene CiloleucelResearch and Development Company: Kite PharmaTreatment of Disease: Extranodal Zone marginal lyoma, Lymph node fringe lymphoma (Nodal marginal zone) lymphoma)Introduction: Axicabagene Ciloleucel, a genetically engineered autologous T-cell immunotherapy that targets CD19, was launched in the U.Sin October 2017 under the name YescartaThe drug is suitable for treatment in adult patients with recurrent or refractory large B-cell lymphoma after treatment by two or more lines, including unexplained diffuse large B-cell lymphoma (DLBCL), primary isolation of large B-cell lymphoma, high-level B-cell lymphoma, and DLBCL derived from prosytoplatic lymphomaDrug: AMP945Research and Development: Amplia TherapeuticsTreatment of Diseases: Pancreatic CancerProfile: AMP945 developed for Amplia Therapeutics a highly selective oral adhesion kinase inhibitor inhibitor (Focal Adhesion Kinase, FAKi) that has shown good results in preclinical studiesAmplia Therapeutics plans to conduct Phase 1 clinical trials of AMP945 in healthy volunteers later this year, and if successful, Phase 2 clinical studies will be conducted in cancer patients in 2021 AMP945 has a variety of ways to work and has the potential to be an attractive drug candidate for treatment stodgy cancers such as pancreatic, ovarian, breast and lung cancer drug name : Efavaleukin alfa (AMG592) Research and Development Enterprise : Amgen treatment of disease : Transplant anti-host disease introduction: Transplant anti-host disease is a complication of transplanting tissue and organs from others, usually associated with stem cell transplantation (e.g bone marrow transplant) According to the website, Efavaleukin alfa is an IL-2 mutant Fc fusion protein Currently, a clinical phase 1 study of rheumatoid arthritis, systemic lupus erythematosus and transplant anti-host disease is currently under way drug name : APR-OD031 Research and Development Company : Applied Pharma Research Treatment of Disease sin : Phenylketonuria (PKU) Profile: APR-OD031 is a slow-release amino acid mixture, modified using patented drug delivery technology Physio Technology ™, capable of producing small coat particles for oral administration, processing and gradually releasing amino acids in the intestines APR-OD031 is designed to reduce phenylanine fluctuations and muscle protein hydrolysis in patients with non-reactive PKU of sandpropyption The company's Physiomimic Technology ™ mask the taste and smell of amino acids, improve patients' drug compliance, and make up for the lack of smelly traditional formulations drug name : Retifanlimab (INCMGA-0012) Research and Development Company : Incyte Corporation treatment of disease : cancer profile: INCMGA-0012 is an anti-PD-1 monoclonal antibody According to the website, INCMGA0012 is currently in the clinical proof of concept (PoC) stage Meanwhile, Incyte is exploring the efficacy of INCMGA0012 monodotherapy in high-satellite instability endometrial cancer and Merkel cell carcinoma drug name : Sintilimab (IBI308) Research and Development Company : Cinda Bio
Treatment Disease : T-cell lymphoma Introduction: Sintilimab is an all-humanized IgG4 monoclonal antibody bio-innovative drug, which binds to PD-1-specific binding to the surface of T cells, blocking this immune escape pathway, reactivating T cells and releasing cell factors to kill tumor cells In December 2018, Sintilimab was approved for listing in China for Hodgkin's lymphoma drug name : Sodium nitrite research and development company : AdimaBio treatment of disease : sickle cell anemia : sickle cell anemia (SCD) is a cause of beta-peptide chain 6th glutamate is replaced by proline, so that hemoglobin S (HbS) abnormal genetic hemoglobindisease, so that red blood cells have a sickle-like name Previous studies have shown that sodium nitrite may prevent red blood cell deformation by activating hemoglobin in low oxygen conditions, reducing platelet aggregation and blood cells glued to the walls of blood vessels, thus preventing possible blockages in blood vessels in patients with SCD drug name : NDX33-o (Thien-99m radioactive lymoplastonic) research and development enterprise : NexEos Diagnostics treatment of disease s : diagnostic treatment of acidophilic cytoplasm introduction: NDX33-o is a new type of non-invasive imaging agent for the diagnosis, treatment monitoring and management of acidophilic cytophilis (EoE) Eosinophils contain a major alkaline protein that damages tissue deposited after the release of eosinophils and causes inflammation NDX33-o can be combined with the main alkaline protein of eosinophils, imaging at X-ray, and in addition to confirming pain caused by EoE, the image will accurately identify the location of the affected tissue in the esophagus, the degree to which the esophagus is affected, and the severity of the disease drug name : Triprei mono-anti-united axiani research and development enterprise : Junshi bio-
treatment of disease : mucosal melanoma introduction: mucosal melanoma in the white species is a rare melanoma subtype, there is currently no mature treatment guide, traditional chemotherapy and monotherapy immunotherapy are not effective The Triprei mono-resistance developed by Junshi Bio is the first domestically produced PD-1 drug successfully listed in China, and is currently approved in China for local progression or metastatic melanoma after the failure of previous standard treatment Junshi Bio has conducted more than 30 trials of Triprei single-drug therapy and combination therapy worldwide, including 14 key registered clinical trials, as well as a Phase 1b clinical trial for multiple solid tumors in the United States, involving nasopharyngeal, urinary skin cancer, lung cancer, stomach cancer, esophageal cancer, liver cancer, breast cancer, and more Drug Name : SEL120 Research and Development Company : Ryvu Therapeutics Treatment of Disease : Introduction to Acute Myeloid Leukemia : SEL120 is a selective CDK8 kinase inhibitor CDK8 is involved in the process of transcription activation and suppression The drug targets CDK8 and its bypass CDK19, which has the potential to treat hetic malignancies and solid tumors with transcription disorders CDK8 inhibitors can also be used as immunotumor therapy due to their established role in inhibiting NK cytotoxicity and tumor monitoring In March 2019, Ryvu Therapeutics obtained FDA approval to launch its SEL120 program for the treatment of acute myeloid leukemia (AML) and high-risk bone marrow hyperplasia syndrome (HR-MDS) and to deliver the first patient in September 2019 drug name : Xenon Nanoparticles Research and Development Enterprise : Cerion Nanomaterials treatment of diseases : amyotrophic lateral sclerosis : amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that is mainly affected by the cerebral cortex, brain stem and spinal motor neurons The study found that oxidative stress may be one of the major causes of neuronal damage, while the nanoparticles of radon dioxide can be watered down with reactive oxygen and active nitrogen, which may alleviate ALS symptoms drug name : Epalrestat Research and Development Company :Perlara PBC treatment of disease : pmM2-CDG profile: pmM2-CDG deficiency of glycosethane 2 is a glycosaccia monogene congenital disease, with approximately 800 patients worldwide Perlara uses the platform of genetically engineered animals (yeast, nematodes, fruit flies and zebrafish) in phenotype screening to identify orphan candidates more quickly and easily Epalrestat is a non-competitive inhibitor of aldehyde reducase, which works by reversingly inhibiting the dealdehyde reducing enzyme, which inhibits sorbitol accumulation in neurons and reduces damage to nerve cells References: 1, Kite's Yescarta ™ (Axicabagene Cildellucel) Becomes First CAR T Therapy Approved by the FDA for The Age of Patients With Relapsed or Refractory Large B-Cell Lymphoma 2 Times Or More Retrieved March 28, 2020, from 2) FDA Awards Amplia Orphan Drug for Specific Cancer Retrieved March 28, 2020, from Retrieved March 28, 2020, from Retrieved March 28, 2020, from Retrieved March 28, 2020, from s/product/products
s6, Innovent Receives an Approval from the US FDA to Initiate Clinical Trials for its Anti-OX40 Monoclonal Antibody IBI101 Retrieved March 28, 2020, from the last day of 2018, count the last year's priority review approved innovative drugs! Retrieved March 28, 2020, from Retrieved March 28, 2020, from Retrieved March 28, 2020, from Retrieved March 28, 2020, from Retrieved March 28, 2020, from Retrieved March 28, 2020, from s13, Synthesis and biomedical applications of ceriumiums - A Review Retrieved March 28, 2020, from Perlara PBC Retrieved March 28, 2020, from Retrieved March 28, 2020, from original title: Orphan Drug Weekly , 13 therapies have been qualified for orphan medicine, Andshi, Cinda, Amgen, Kite therapy have been shortlisted