CDER "innovation promotes health" 2017 new drug approval and other drug treatment progress report 36 pages complete translation

Welcome to read our new health frontier report, new drug approval and other drug treatment progress in 2017 Since 2012, CDER has issued new drug summary every year, which shows the role of CDER in the process of introducing innovative drugs that are safe and effective for patients This year, we expanded the scope of our report to include more valuable information New drugs mean new progress in the treatment of patients' diseases So will the 2017 report you are about to see, and it has more content Our report this year will also focus on non novel drugs that play a significant role in treatment As in previous years, many important therapeutic advances in 2017 have involved new uses and new groups of approved drugs, such as children's drugs Our report also highlights the innovative management tools that cders use to improve efficiency and accelerate review and approval Readers should pay attention to the fact that the date of our approval decision is earlier than the target date set by the parliament on user payment agreement, and most drugs approved in the United States are earlier than the rest of the world CDER has played an active role in FDA's efforts to coordinate manufacturers, monitor key drugs in shortage and work with manufacturers to overcome the shortage, speed up review and use powerful means to alleviate drug shortage during 2017 hurricane It is worth noting that CDER can not effectively evaluate the safety of all drug treatments without the support of extensive production, scientific research, medicine and organizations concerned about patients We would like to take this opportunity to thank many public-private partnerships and cders for their work in leading and participating in supporting innovation and healthy progress Patients are playing an increasingly decisive role in drug development and approval Our report also shares FDA's progress in making decisions from a patient perspective We hope our new report will provide patients with a deeper understanding of CDER's multi-channel support for innovation 2017 innovation and progress in 2017, the approval of fdacder new drug treatment has helped a large number of patients suffering from different diseases, from rare diseases to common diseases, so that they can gain hope in improving the quality of life and improving the survival opportunities of major diseases Rare diseases among many approvals to help patients with rare diseases, CDER has approved the first non blood products for sickle cell patients in nearly 20 years For the first time, the treatment was limited to the diagnosis of adult arteritis, a rare disease that causes vascular inflammation CDER has also approved a product for the treatment of batten's disease, which can lead to epilepsy, dementia and other weakened symptoms Infectious diseases we have approved a new antibiotic for the treatment of some types of severe skin infections, including complex urinary tract infections involving kidney infections We have approved two products for the treatment of chronic hepatitis C, a drug for the prevention of giant cell infection in the process of bone marrow transplantation, and the first product in the United States for the treatment of American trypanosomiasis (a rare parasitic disease that causes serious heart disease after years of infection) In the past year, there has been a lot of achievements in the treatment of nervous system disorders CDER has approved new drugs for the treatment of patients with tardive dyskinesia (high frequency side effects in psychotherapy), myasthenia gravis (rare neuromuscular diseases), Duchenne muscular dystrophy, multiple sclerosis, Lou Glick and Parkinson's disease Cancer treatment 2017 is also the year of approval of anticancer drugs These anticancer drugs include drugs for the treatment of some acute lymphoblastic leukemia, drugs for the treatment of Merkel cell cancer, drugs for the treatment of repeated acute episodes of granulocytic leukemia, drugs for the treatment of lymphoma, drugs for the treatment of ovarian epithelial hyperplasia, ovarian cancer and primary peritoneal cancer, special dosage forms of drugs for the treatment of liver cancer, breast cancer and colorectal cancer We approved the first new anti-cancer drug based on the genetic characteristics of cancer cells rather than the location of tumors in the body Other developments were also in 2017, when CDER approved new treatments to reduce heart risk in diabetic patients, a new drug for moderate to severe eczema in adults and three methods for moderate to severe plaque psoriasis We also approved the first implantable product in the United States with a sensor that can record the amount of drug to be used Due to the damage caused by the hurricane in 2017, CDER and pharmaceutical manufacturers went to the disaster area to solve the drug shortage Drug treatment approved by CDER in 2017 In 2017, CDER approved a variety of drug treatments to improve public health in the United States, including: new drugs, usually containing more innovation, and may help improve clinical treatment; new and expanded uses of FDA approved drugs; biological analogues, highly similar to the therapeutic biological products approved by FDA These approvals increase consumer choice and market competition; new formulations or producers of FDA approved drugs have advantages over the original products, such as they can be taken on an empty stomach; new dosage forms increase the value of FDA approved drugs, such as chewable tablets designed for patients who cannot swallow; This report outlines these approvals and examples, emphasizing that these new approvals can provide novel treatment options that patients need New drugs are often innovative products to meet previously unmet treatment needs or to improve patient treatment programs The active ingredients or excipients of innovative drugs have not been approved in the United States before The report lists all the innovative drugs approved in 2017 and the information that CDER considers to be significant progress In 2017, CDER approved 46 innovative drugs: new molecular entities under NDA application or new therapeutic biological products under BLA application The innovative drugs approved by CDER in 2017 are listed as follows: Note: the above information is as of December 31, 2017 In individual cases, if necessary, FDA will change MNE status or apply for a new bla For example, when there is new information that can affect the type and status of the original application Innovative drugs approved by CDER annually: in 2008-2017, 46 innovative drugs were approved by CDER The figure below shows the ten-year situation from 2008 to 2017, with an average of 31 innovative drugs approved each year The impact of new drug approval the new drug approved by CDER in 2017 deserves attention due to its potential positive role and unique contribution to high-quality treatment In 2017, CDER of first in class identified 15 (33%) of 46 new drugs as the first drug, as a sign of their strong positive impact on American health The mechanism of action of these drugs is often different from that of existing therapies Among the new drugs approved by FDA in 2017, the first ones are besponsa, brineura, dupixent, emflaza, giapreza, hemlibra, idhifa, macrilen, mepsevii, ocrevus, prevymis, radiova, rhompressa, rydapt, and xermelo Examples of notable first drugs in 2017 include dupixent (dupilumab) in the treatment of moderate eczema (atopic dermatitis) in adults, and ocrevius (ocrelizumab) in the treatment of adult multiple sclerosis and primary progressive multiple sclerosis Drugs for rare diseases in 2017, 18 of the 46 innovative drugs approved by CDER were used to treat rare diseases or "orphan" diseases (i.e the number of patients is less than 200000) Patients with rare diseases often lack medicine or have no treatment The orphan drugs approved in 2017 include aliqopa, alunbrid, austedo, bavencio, benznidazole, besponsa, brineura, calsequence, emflaza, hemlibra, idhifa, macrilen, mepsevii, prevymis, radicava, rydapt, xermelo, and zejula Examples of drugs approved in 2017 to promote the treatment of patients with rare diseases: brineura (cerliponase alfa) is a special form of treatment for batten's disease, a rare disease that can cause progressive neurological dysfunction, including seizures, visual problems / blindness, personality and behavior changes, dementia and loss of ability to walk, speak and communicate Hemlibra (emisizumab) is used to prevent bleeding or to reduce the frequency of bleeding events in patients with hemophilia A who have developed inhibitors of factor VIII This is the first non blood product approved for this condition Other approved innovative drugs: in addition to the first-line products and orphan drugs mentioned above, innovative drugs in 2017 also include these noteworthy examples, which were approved for the first time in the United States and may significantly improve disease treatment in the following aspects: In 2017, aliqopa (copanlisib) was approved as an innovative drug to enhance public health in a wide range of fields for the treatment of relapsed follicular lymphoma in adults Bavencio (avelumab), a slowly developing non-Hodgkin's lymphoma with at least two systemic therapies, was used to treat patients over 12 years old with rare metastatic Merkel's cancer, including those who had previously received chemotherapy; Benznidazole is used to treat trypanosomiasis Americana, a parasitic infection that can cause serious heart disease several years after infection and can affect swallowing and digestion This is the first drug approved for this rare disease in the United States Besponsa (inotuzumab ozogamicin) is used to treat adult blood cancer called B-cell precursor acute lymphoblastic leukemia, and calsequence (acalabrutinib) is used to treat adult patients with mantle cell lymphoma who have experienced at least one advanced treatment Deflazacort is used to treat Duchenne's muscular dystrophy (DMD) over 5 years old, a rare genetic disorder leading to severe muscular atrophy; giapreza (angiotensin II) is used to treat distributed or vasodilation shock hypotension, which remains low pressure despite treatment with perfusion and pressor drugs; idhifa (enasidenib) For the treatment of recurrent acute myeloid leukemia, a blood cancer caused by gene mutation; ingrezza (valbenazine) for the treatment of tardive dyskinesia, a common side effect of antipsychotic therapy, often manifested as involuntary tremors of the face and body, other uncontrolled movements; mavyreet (glecaprevir and pibrentasvir) It is used to treat chronic hepatitis C or mild hepatitis C with genotype 1-6 and no cirrhosis in adults, including patients with severe kidney disease and hemodialysis; mepsevii (vestronidase Alfa vjbk) is used to treat a type 7 mucopolysaccharidosis, a rare gene abnormality, bone deformity caused by enzyme deficiency, development delay, hepatosplenomegaly, airway stenosis, etc.; nerlynx (neratinib) Adjuvant treatment for expansion - treatment to further reduce cancer recurrence after the first treatment, such as early HER-2 positive breast cancer Prevymis (letermovir) is used to prevent cytomegalovirus infection in perioperative bone marrow transplant recipients; radicava (Edaravone) is used to treat amyotrophic lateral sclerosis, often referred to as Glick's disease, a rare disease that attacks and kills nerve cells that control autonomic muscles; rydapt (midostarurin) It is used to diagnose a disease called acute myeloid leukemia caused by FLT3 gene mutation in adults; siliq (brodalumab) is used to treat adult moderate and severe plaque psoriasis, a disease in which the body's immune system sends wrong signals to skin cells, resulting in erythema and scales in many parts of the body; symproic (naldemine) For the treatment of opioid receptor induced constipation in adults; tremfya (guselkumab) for the treatment of moderate and severe plaque psoriasis in adults; trucance (plecantide) for the treatment of chronic congenital constipation caused by unknown causes in adults; vaborbactam an