New drugs for rare diseases accelerate the application of these pharmaceutical companies into China! Series one

Source: ihealth 2018-05-24 National Drug Administration and National Health Commission jointly released the announcement on optimizing the relevant matters of drug registration review and approval Among them, the application for registration of new drugs for rare diseases in China is of great benefit "For the prevention and treatment of serious life-threatening diseases and rare disease drugs without effective treatment, the drug review center of the State Food and Drug Administration (hereinafter referred to as the drug review center) shall establish a communication and exchange mechanism with the applicant, strengthen the guidance of drug research and development, give priority to the registration applications included in the scope of priority review and approval, review, inspection, approval and other links We should allocate resources and speed up the review and approval " In addition, if the applicant for registration of imported drugs for rare diseases considers that there is no ethnic difference through research, he / she can submit the clinical trial data obtained abroad to directly apply for drug registration This means that pharmaceutical companies can use the data obtained from foreign clinical trials to directly apply for registration of new drugs in China Prior to the announcement, new drugs for rare diseases had already enjoyed the preferential treatment of clinical trials Now, the announcement goes further and proposes that "for the above-mentioned clinical trial application of imported drugs that have been accepted before the announcement and have been applied for clinical trial reduction or exemption, if it meets the requirements of the measures for the administration of drug registration and related documents, the import can be directly approved." Other incentives include: simplifying drug testing based on product safety risk control The procedures for re registration and filing of imported drugs shall be cancelled After the application for re registration of imported drugs is accepted, all information shall be transferred to the drug examination center for review and approval With such a favorable policy, which rare diseases and new drugs of which pharmaceutical companies are likely to take the lead in entering China, apply for new drug registration and use for patients as soon as possible? According to the order of the first list of rare diseases published by the National Health Commission, ihealth combed the new rare disease drugs under research and approved abroad, and presented them to you This is the first part of the series New drugs and pharmaceutical companies with rare diseases beginning with a to D were collected ALS company: chemist's main new drug: cc001 chemist's new drug cc001 obtained US $243000 in 2016 for the development of phase I clinical trials to treat ALS The new oral drug has been tested in healthy volunteers As a kind of progressive muscular atrophy, MDA of muscular dystrophy patients Association and ALS Association (target ALS Foundation) cooperate and invest US $100000 to reward researchers who can discover ALS biomarkers 2 Cystic fibrosis (CF) Company: vertex summit pharmaceutical's main new drug: kalydeco (ivacftor) Company in Boston focuses on the production of new drugs for cystic fibrosis The main selling CF drug is kalydeco (ivacaftor) Kalydeco has been approved in the United States, the European Union, Australia and Canada for the treatment of cystic fibrosis patients with CFTR gene specific mutations In May last year, kalydeco was also approved for use in CF patients (2 years and older) with one of 23 residual functional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene Patients are expected to be just over 900 in the United States 3 Congenital myotonic malnutrition (NDM) Company: Gilead science major new drug: ranolazine hydrochloride ranolazine hydrochloride is currently approved for the treatment of angina pectoris Gilead and Ohio children's Hospital jointly carried out clinical trials to treat the symptoms of congenital tonic malnutrition and type 1 tonic malnutrition 4 Exon 51 of progressive muscular dystrophy (including DMD, BMD, etc.) is the first DMD drug approved by exon 51 of eteplirsen Sarepta company In patients' skeletal muscle, the drug can increase the content of dystrophin protein 2) Emflaza (difcote) PTC pharmaceutical was approved by FDA in February 2017, becoming the second approved new DMD drug Emflaza is an anti-inflammatory glucocorticoid drug Although other countries have been on the market for a long time, they have not been approved to treat DMD Marathon then sold emflaza to PTC for $140 million 3) Ptc-124 (ataluren) PTC company can treat nonsense mutation and interact with ribosome to help it cross immature terminator and complete dystrophin protein translation Although conditional approval was obtained in the EU, ptc-124 was rejected twice by the US FDA because the end point of clinical trial was not reached 4) Raxone is a synthetic analogue of coenzyme Q10 As a transporter in the mitochondrial electron transport chain, coenzyme Q10 can increase the production of ATP in cells Raxone has potential value in delaying the loss of respiratory function in patients with DMD At present, it has entered the third phase of clinical trials UK regulators have agreed to allow the drug to enter the national early access drug program (eams) for free use by DMD patients 5) Ma-0211, a new drug for mitochondria, was developed by mitobridge company of anstar The drug is PPAR δ regulator, which can increase the function and synthesis of mitochondria by regulating cell metabolism It is currently in phase I clinical trials 6) An oral sustained release agent of collagen I synthesis inhibitor of ht-100 Akashi company, which can alleviate inflammation and promote muscle growth, was developed by Akashi company In February 2016, a patient death event occurred in its high dose group clinical trial, resulting in the clinical trial of the drug being stopped FDA agreed to resume clinical trials of ht-100 The drug is currently in phase II clinical trials.