These 13 domestic new drugs have obtained the qualification of FDA orphan drugs

These days, everyone is talking about the movie "I am not the God of medicine" In the film, the prototype of "Lenin" is Novartis' Gleevec (imatinib mesylate) The drug was first approved as orphan drug, and then it was close to the annual sales peak of 5 billion US dollars, making it a super blockbuster drug It is the perfect explanation of "wild lily has spring" in the pharmaceutical industry In the United States, once a new drug obtains the status of FDA orphan drug, it will enjoy tax relief, application fee exemption, R & D funding, accelerated approval and market monopoly for seven years after its launch, and may even obtain a valuable priority review certificate Therefore, from the perspective of new drug research and development, to enter the market as orphan drugs and enjoy the market monopoly period and then expand the indications, is a "win by surprise" clinical development strategy In view of the fact that the drugs approved by FDA are relatively easy to be recognized by other markets, for domestic innovative drug R & D enterprises, if they choose the right indications, they can seize the opportunity of domestic registration by applying for FDA's "orphan drug qualification identification" and adopting the development strategy of China and the United States double reports, so as to realize "overtaking at a curve" According to incomplete statistics, 13 new drugs from Chinese pharmaceutical companies have obtained 17 FDA orphan drug qualifications Among them, bgb-3111, Btk inhibitor of Baiji Shenzhou, obtained 3 items, while androtinib of Zhengda Tianqing and cotsiranib (stp705) of sannuo medicine obtained 2 items of FDA orphan drug qualification China's innovative drug group 01: butylphthalide soft capsule ALS, also known as motor neuron disease and gradually frozen human disease, has been recognized as the "ice bucket challenge" popular on the Internet four years ago ALS is caused by progressive degeneration of motor nerve cells, resulting in muscle weakness and atrophy of limbs, trunk, chest and abdomen, as well as speech, swallowing and respiratory dysfunction, until death of respiratory failure Although the incidence rate is very low, ALS is a great threat to the quality of life and life of patients The current clinical medication has no obvious effect Butylphthalide is a national class 1 innovative drug with independent intellectual property rights, which is jointly developed by the group and the Chinese Academy of Medical Sciences It is mainly used to treat acute ischemic stroke In September 2002, Shiyao group obtained the first-class new drug certificate and trial production approval document of butylphthalide raw materials and soft capsules In 2005, the product was officially launched for the treatment of mild and moderate acute ischemic stroke In April 2010, Petrochemicals Group launched sodium butylphthalide chloride injection In 2015, the sales volume of butylphthalide exceeded 1.5 billion yuan, which is one of the heavyweight varieties of Petrochemicals Group It is understood that Petrochemicals Group has no compound patent for butylphthalide, and its capsule and injection patents expire in 2023 and 2022 respectively The patent for the compound for acute ischemic stroke will also expire in 2023 In order to prolong the life cycle of the product, Petrochemicals Group has also applied for new indications of butylphthalide, L-butylphthalide, and preparation patents of butylphthalide related substances See: "butylphthalide" of stone drug group has been recognized as an orphan drug by FDA 02 stone drug group: about 15-20% of gastric cancer patients with dp303c are HER2 positive, and the rate of tumor patients with gastroesophageal junction are HER2 positive is higher The total number of gastric cancer diagnosed in the United States is less than 200 thousand, which is a rare disease But the incidence rate is high in Asian countries such as China, Japan and South Korea Dp303c is a highly selective antibody to HER2 coupled with a cytotoxin It is a new targeted drug for the treatment of HER2 positive gastric cancer It is independently developed by Sinopharm group, with independent intellectual property rights, and has applied for a number of patents in the United States, China and other countries Dp303c showed a good anti gastric cancer effect in mice inoculated with NCI-N87 cells and SK-BR-3 cell line expressing HER2 Therefore, Shiyao Group believes that dp303c can further improve the treatment efficiency of gastric cancer, reduce side effects and prolong the survival period of patients See: the FDA granted the orphan drug qualification of antibody coupling drug "dp303c" of Shijiazhuang Pharmaceutical Group 03 Sinopharm: cotsiranib is a class 1.1 chemical developed by Sinopharm (project code: stp705) It is a kind of nucleic acid interference drug with anti fibrosis and anti-inflammatory effects It is composed of two targets TGF - β 1 and COX-2 siRNA oligonucleotides It can exert the synergistic advantages of its combination and inhibition at the same time It can enhance the efficiency and safety of drug delivery by using histidine lysine copolypeptide nanoparticles (PNP), and block the activity of fibrosis and inflammation directly and efficiently Cortani has been approved by FDA in 2016 and CFDA in China in 2017 to reduce hypertrophic scars Phase IIa clinical trials are underway in the United States, and Chinese clinical trials are underway Because the mechanism of drug action is clear, it has a wide range of adaptability and good curative effect for various inflammatory and fibrosis related diseases in theory In August and December 2017, cortani was awarded the orphan drug qualification of treating primary sclerosing cholangitis and cholangiocarcinoma by FDA rare disease product development office See: stp705, a new nucleic acid interference drug of shengnuo pharmaceutical, won the qualification of FDA orphan drug 04 stone Pharmaceutical Group: Mitoxantrone Hydrochloride liposome mitoxantrone is a widely used broad-spectrum anti-tumor drug in clinic Because of its serious cardiotoxicity and adverse reactions such as myelosuppression, its clinical application is severely restricted After mitoxantrone was made into lipid formulation, the drug's pharmacokinetics, tissue distribution, efficacy and toxicity had significant changes Compared with the ordinary preparation, the efficacy and safety of mitoxantrone were significantly increased Mitoxantrone Hydrochloride liposome is independently developed by the group of Chinese Materia Medica, and there is no report of this product in the world at present Sinopharm group has independent intellectual property rights in this project It has applied for 7 domestic patents and 2 international patents, and has been authorized by the European Union and other 10 countries The United States, Japan and other 7 countries are under trial The product is currently in phase II clinical trials in China Peripheral T-cell lymphoma is a group of aggressive tumors with heterogeneous morphological changes of mature T-cell immunophenotype It accounts for 7% of all cases of non Hodgkin lymphoma (NHL) Mitoxantrone Hydrochloride liposome was awarded orphan drug qualification for treatment of peripheral T-cell lymphoma by FDA rare disease product development office in September 2017 See: Mitoxantrone Hydrochloride liposome of Petrochemicals Group has obtained the FDA orphan drug qualification certificate 05 dongyangguang Pharmaceutical Co., Ltd.: iffenidone Yinfenidone (hec585) is a class 1 new drug independently developed by dongyangguang Pharmaceutical Co., Ltd for the treatment of fibrosis It first submitted a clinical application to Guangdong Provincial Bureau on June 17, 2016, and was accepted by CDE on August 23, 2016 It was approved by CDE on March 22, 2017 On August 10, 2017, it was approved by FDA as an orphan drug for the treatment of idiopathic pulmonary fibrosis (IPF) Dong Yangguang submitted the ind clinical trial application of iforinone to the US FDA at the same time, and obtained the FDA's permission to carry out the clinical trial on February 22, 2017; on May 2 of the same year, the first administration of iforinone was successfully completed in the phase I clinical trial center of Covance (US) Idiopathic pulmonary fibrosis is the most common and serious form of fibrosis, but the pathogenesis is not clear The 3-year mortality rate is as high as 50%, which is worse than cancer Prior to the marketing of pirfenidone, there was no effective treatment for idiopathic pulmonary fibrosis, so only lung transplantation and conservative treatment were used According to the information disclosed by Dong Yangguang, the in vitro and in vivo pharmacodynamic experiments show that the activity of iffenidone is more than 100 times higher than that of pirfenidone, and it has good pharmacokinetic characteristics and safety It is speculated that iffenidone is the me better drug of pirfenidone See: severe Dongfangguang class 1 new drug "ifonilone" obtained the FDA orphan drug qualification certification 06 Tongli biology: l-praziquantel (R) - praziquantel (l-praziquantel) of Tongli biological medicine in Suzhou obtained the FDA orphan drug qualification certification for the treatment of schistosomiasis There are about 250 million people infected with Schistosoma in the world Praziquantel is a synthetic derivative of pyrazine isoquinoline, also known as cyclopraziquantel As a highly effective broad-spectrum antiparasitic drug, praziquantel is a major breakthrough in the history of chemotherapy for parasitic diseases, and it is the first drug to treat a variety of parasitic diseases on the market In the past few years, who hopes to control the global schistosomiasis epidemic by providing praziquantel, but it has been facing the following difficulties: (1) Limited global praziquantel supply (less than 30% of annual demand); (2) Due to the excessive and unpleasant taste of praziquantel tablets caused by the presence of dextroquinolone, children in the epidemic area (~ 50% of the threatened population) lack of compliance with the drug; (3) the production of praziquantel is facing more and more strict environmental protection pressure; (4) the extra cost of producing and transporting useless dextran Through the self-developed enzyme chemical combination technology, 50% of the useless and bitter d-praziquantel in praziquantel was removed from Suzhou Tongli biomedical Co., Ltd., and a cost-effective synthetic route of l-praziquantel suitable for industrial scale-up production was obtained 07 Zhengda Tianqing: androtinib, an inhibitor of VEGFR and PDGFR, was modified on the basis of sorafenib, a similar drug on the market After more than 10 years of research and development achievements by the R & D team of Zhengda Tianqing, it has dual effects of anti-tumor angiogenesis and tumor growth inhibition As a 1.1 new drug developed by Zhengda Tianqing, arotinib was first declared in July 2010 It is a kind of drug that Zhengda Tianqing and the outside world have high hopes for On June 19 this year, the product was approved for marketing by the State Drug Administration The overseas rights and interests of androtinib are developed by advanchen laboratories, a Sino US joint venture drug R & D company founded by Chen Guoqing In December 2015 and June 2017, the FDA awarded the qualification of orphan drugs of arotinib in the treatment of ovarian cancer and soft tissue sarcoma 08 Hengrui medicine: apatinib mesylate is a small molecule tyrosine kinase inhibitor of vascular endothelial growth factor receptor-2 (VEGFR-2) As a new small molecule targeted anti-tumor drug, apatinib, through highly selective competition for the ATP binding site of VEGFR-2 in cells, blocks downstream signal transduction, inhibits the production of tyrosine kinase and thus inhibits the formation of new blood vessels in tumor tissue, is used for the treatment of advanced gastric cancer, and is the only oral preparation of targeted drugs for advanced gastric cancer Apatinib was originally a tyrosine kinase inhibitor discovered by advanchen laboratories, and then awarded the development right of China to Jiangsu Hengrui Pharmaceutical Co., Ltd and successfully developed it On October 17, 2014, it was approved by the State Food and Drug Administration for listing, and the exclusive development right except for China has been granted to LSK biopartners / bukwang On June 15, 2017, FDA granted apatinib the qualification of orphan drug for gastric cancer treatment 09 Hongyun Huaning: anti human endothelin receptor a monoclonal antibody injection anti human endothelin receptor a single