List of new drugs approved by CDER in 2019! Novartis, Aberdeen, Genentech, Trinity

In 2019, the FDA Center for drug review and Research (CDER) approved 48 new drugs, including 37 new molecular entities (NME) and 11 biological product licensing applications (BLA) Compared with 59 in 2018, the total number decreased by 11, down 18.64% year on year Figure 1: Statistics of rare disease / orphan drugs approved by CDER in recent 10 years, of the 48 new drugs approved, 20 new drugs obtained orphan drug qualification (o), accounting for 41.67% of new drugs approved by CDER There are two characteristics of rare diseases, one is the small number of patients, and the United States is less than 200000 people The second is serious diseases endangering life and health In terms of priority review, if the CDER determines that the drug has the potential to make a substantial contribution to health care, the drug will be given priority review The drug is reviewed within 6 months instead of the standard 10 months Among the approved new drugs in 2019, 25 were identified as priority review (P), accounting for 52.08% of the 48 new drugs CDER uses a variety of regulatory methods to accelerate the development and approval of new drugs In addition to the priority review, these methods include fast track, breakthrough and accelerated approval In 2019, five drugs were approved for breakthrough treatment: zulresso, balversa, poly, turalio, rozlytrek In terms of enterprises, Novartis received the largest number of approvals, reaching five new drugs Secondly, first trimethoprim, Abercrombie and Genentech obtained two new drugs respectively Baiji Shenzhou has also obtained a new drug, zebutini, which is the first Chinese self-developed new anti-cancer drug approved for market in the United States Figure 2: in terms of the number of new drugs approved by enterprises in 2019, only no new drugs were approved in January New drugs were approved mainly in the second half of the year, with the largest number in August, and 9 new drugs were approved Figure 3: the number of new drugs approved in each month of 2019 is shown in the figure below, which is a summary of 48 new drugs approved in 2019 It comes from the FDA website, new drug approvals for 2019 Table 1: review of new drugs approved by the FDA in 2019 1 Jeuveau (prabotulinumtoxina xvfs) was approved by the FDA on February 1 by evolus company, jeuveau (prabotulinumtoxina xvfs) was used in adults to temporarily improve the appearance of moderate to severe frowns related to frowning and / or brow lowering activities The FDA approved jeuveau based on the data of two phase III randomized, multicenter, double-blind, placebo-controlled studies in the United States, both of which reached all primary endpoints, and confirmed the effectiveness of jeuveau in improving the severity of brow wrinkles relative to placebo: on the 30th day of treatment, 67.5% (ev-001 study) and 70.4% (ev-002 study) patients in jeuveau treatment group reached two points of compliance The placebo group was 1.2% and 1.3% respectively No serious drug-related adverse events were reported in either study 2 On February 6, the FDA approved the development of caplacizumab yhdp by Sanofi to treat adult acquired thrombocytopenic purpura (attP) Cablivi is the first specific attP treatment drug and the first approved nano antibody drug The effectiveness of cablivi was based on a clinical trial of 145 patients who were randomized to cablivi or placebo Two groups of patients received plasma exchange and immunosuppressive therapy The results showed that patients treated with cablivi had a faster improvement in platelet count and a reduction in the total number of attP related deaths and relapses during treatment compared to placebo Throughout the study period (drug treatment plus 28 day follow-up after discontinuation), the recurrence rate was 38% in the placebo group and only 13% in the cablivi group 3 On February 13, the U.S FDA approved the launch of Novartis' egaten (triclabendazole) for the treatment of patients over 6 years old with Fasciola This is the only FDA approved treatment for the disease Paragonimiasis, also known as liver fluke infection, is a neglected tropical disease There are about 2.4 million patients around the world, and another 180 million people are at risk It is caused by two parasitic flatworms that can enter the body when people eat food contaminated by larvae If not treated, Fasciola can cause severe pain and discomfort, leading to a decline in quality of life and productivity 4 Zulreso (brexanolone) on March 19, FDA approved zulreso (brexanolone) injection of sage therapeutics for the treatment of postpartum depression (PPD), which is the most common delivery complication This approval makes zulreso the first and only drug approved to treat PPD in the world Zulreso's approval was based on data from three multicenter, randomized, double-blind, parallel, placebo-controlled clinical studies These studies evaluated the efficacy and safety of zulreso injection in the treatment of moderate to severe PPD The study enrolled 18-45-year-old pregnant women who developed severe depressive symptoms no earlier than the third trimester and no later than the fourth week after delivery, and the time of the study was earlier than six months after delivery 5 Sunosi (solriamfetol) on March 20, FDA approved sunosi (solriamfetol) of jazz pharmaceutical company for the treatment of adult patients with daytime excessive sleepiness (EDS) related to narcolepsy or obstructive sleep apnea (OSA) The FDA's approval of sunosi is based on the data of phase III clinical project tones This project includes four randomized and placebo-controlled studies, namely: the treatment of EDs in adult patients with narcolepsy (the tones-2 study), the treatment of EDs in adult patients with OSA (the tone-3 study, the tone-4 study), and the long-term safety and maintenance effect study (the tones-5 study) for adult patients with narcolepsy or OSA in the treatment of EDs Data from these studies confirm sunosi's superiority over placebo in the treatment of EDS related to narcolepsy and OSA 6 Mayzent (siponimod) was approved by the US FDA on March 26 for marketing by Novartis Inc mayzent (siponimod) for the treatment of adult patients with recurrent multiple sclerosis (RMS), including active secondary progressive disease (active SPMS), recurrent remission disease (RRMS) and clinical isolated syndrome (CIS) This approval is based on mayzent's performance in a randomized, double-blind, placebo-controlled phase 3 clinical trial called expand Compared with placebo, mayzent significantly reduced the risk of confirmed disability progression (CDP) after 3 months, and 21% lower than placebo (P = 0.013) In addition, mayzent significantly delayed the risk of CDP by 6 months, which was 26% lower than that of placebo (P = 0.0058) At the same time, mayzent reduced the annual recurrence rate by 55% 7 Evenity (romosozumab aqqg) was approved by FDA on April 9 The indication is osteoporosis with high risk of fracture in postmenopausal women Evenity has become the first anti sclerostin monoclonal antibody drug approved for market in the world, which can accelerate bone formation and reduce bone absorption Evenity is jointly developed by Amgen and youshibi on a global scale In January, evenity was approved in Japan for use in men at high risk of fracture and postmenopausal women with osteoporosis, reducing the risk of fracture and increasing bone density This approval is also the first regulatory approval for evenity worldwide In early March of this year, evenity was on sale in the Japanese market At present, evenity is also under review by the European Drug Administration (EMA) 8 Balversa (erdafitinib) on April 12, FDA approved balversa (erdafitinib) of Janssen to be put on the market for the treatment of locally advanced or metastatic bladder cancer with FGFR3 or FGFR2 mutations in the progression of disease after chemotherapy with platinum therapy Balversa is an oral pan FGFR inhibitor FGFRs is a family of receptor tyrosine kinases, which can be activated by gene mutations in different tumors, thus promoting the survival and proliferation of tumor cells In addition, balversa is the first FDA approved targeted therapy for metastatic bladder cancer 9 Skyrizi (risankizumab rzaa) on April 23, FDA approved the listing of risankizi (risankizumab rzaa) for systemic treatment or phototherapy of patients with severe psoriasis and plaque psoriasis Skyrizi is an interleukin-23 (IL-23) inhibitor that is thought to be associated with several chronic immune-mediated diseases, including psoriasis and inflammation Skyrizi can selectively block IL-23 by binding with P19 subunit, so as to treat related skin diseases 10 Vyndaqel (tafamidis meglumine) on May 3, the FDA of the United States approved Pfizer's vyndaqel (tafamidis meglumine) for the treatment of adult patients with wild-type or hereditary transforming thyroxine protein amyloid myopathy (attr-cm), in order to reduce cardiovascular mortality and cardiovascular related hospitalization The FDA approval is based on data from the critical clinical phase 3 trial attr-act, the first global, double-blind, randomized, placebo-controlled clinical study on the treatment of the disease The data showed that in 30 months, compared with placebo, vyndaqel significantly reduced all-cause mortality and cardiovascular related hospitalization rate (P = 0.0006), 30% (P = 0.026) and 32% (p 1%) respectively, including stimulation of the application site, pruritus and sunburn 29 On October 7, the FDA approved the listing of Novartis' bevu (brolucizumab) for the treatment of wet age-related macular degeneration (wet AMD, namd) This approval is based on data from two head to head phase III hawk and harrier clinical studies The results showed that the average change of BCVA of patients who received beovu treatment for 48 weeks was not inferior to that of albosip, and the safety was equivalent Patients treated with beovu showed a significant reduction in central retinal thickness (CST) at week 16 and year 1, with less water accumulation in the intraretinal (IRF) and / or subretinal (SRF), and retinal fluid is a key indicator of disease activity 30 Scenesses (afamelanotide) was approved by FDA on October 8 by clinuvel company to be listed on the market for the treatment of adult patients with skin damage due to erythropoietic protoporphyria The efficacy of scenesses was validated in two clinical trials In one of the trials, a total of 93 patients with erythropoietic protoporphyria participated in the trial, and 48 patients received scenesses treatment The results showed that in the 180 days of treatment, the painless sunshine exposure time of scenesses group was 64 hours, significantly higher than that of placebo group (41 hours) 31 Fluorodopa F18 was approved by FDA on October 10, and it is a diagnostic agent for positron emission tomography (PET), which can help to diagnose adult patients with suspected Parkinson's disease (PS) 32 Reyvow (lasmiditan) on October 11, FDA approved reyvow (lasmiditan), a 5-HT1F receptor agonist of Lilly, for the treatment of adult acute migraine This is the first drug approved by FDA for the treatment of acute migraine in 20 years The approval is based on the results of two phase III trials called samurai and Spartan, respectively Reyvow significantly increased the proportion of patients without pain and other symptoms such as nausea, acoustic sensitivity and photosensitivity compared with placebo 33 On October 21, trikafta (elexacaftor / ivacaftor / tezacaftor) was approved by FDA to be on the market for the treatment of at least one f508del mutation in the gene of cystic fibrosis transmembrane transfer regulator (CFTR) for patients aged 12 and over