Innovative medicines often mean new treatment options for patients and advancements in health care for the public. The U.S. Food and Drug Administration's (FDA) Center for Drug Evaluation and Research (CDER) conducts a comprehensive evaluation of proposed drugs based on necessary study design elements and other factors required in a drug application. As of December 31, 2023, the FDA has approved a total of 55 new drugs in 2023, including 37 new molecular entities and 18 biological products. The following is a summary and introduction to new molecular entity drugs approved by CDER in 2023. This article does not include new therapeutic biologics, vaccines, allergy products, blood and blood products, plasma derivatives, cell and gene therapy products, or other products approved by the Center for Biologics Evaluation and Research in 2023.
Brenzavvy
On January 20, 2023, Theracos Bio Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) approved Brenzavvy for use as an adjunct to diet and exercise to improve blood sugar control in adults with type 2 diabetes. The active ingredient of the drug is bexagliflozin, bexagliflozin It is a sodium-glucose co-transporter 2 (SGLT2) inhibitor. This product cannot be used in patients with type 1 diabetes or the treatment of diabetic ketoacidosis, as well as patients who are allergic to bexagliflozin. It is not suitable for patients with end-stage renal disease or those who are currently undergoing treatment. Treatment of type 2 diabetes in patients receiving dialysis.
Jaypirca
On January 27, 2023, Eli Lilly announced that the FDA has accelerated the approval of its new generation BTK inhibitor Jaypirca, which is suitable for the treatment of relapsed or refractory mantle cell lymphoma (mantle cell lymphoma) after receiving at least two lines of systemic therapy (including BTK inhibitors). MCL) adult patients. The active ingredient of this product is pirtobrutinib, which is the first and only non-covalent (reversible) BTK inhibitor approved by the FDA and the first in the world.
Orserdu
On January 27, 2023, the FDA approved Elacestrant, a subsidiary of Menarini's subsidiary Stemline Therapeutics, to receive FDA accelerated approval under the trade name Orserdu for postmenopausal women with ER+, HER2-, and ESR1 mutations whose disease has progressed after at least first-line endocrine therapy. or adult male patients with advanced or metastatic breast cancer. Elacestrant is a selective estrogen receptor modulator (SERD) that can dose-dependently degrade estrogen receptor α and inhibit estradiol-dependent ER-directed gene transcription and tumor growth. This is also FDA-approved The first oral SERD approved.
Jesduvroq
On February 1, 2023, GlaxoSmithKline announced that the FDA approved the marketing of its hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) Jesduvroq. The active ingredient of this product is daprodustat, which is suitable for the treatment of people who have undergone dialysis. Jesduvroq is not suitable for use in adults with anemia caused by chronic kidney disease of at least four months and who are not on dialysis. Jesduvroq becomes the first oral drug marketed in the United States to treat anemia caused by chronic kidney disease.
Filspari
On February 17, 2023, Travere Therapeutics announced that the FDA had accelerated approval of its drug Filspari, whose active ingredient is sparsentan, an endothelin and angiotensin II receptor antagonist. This is the second drug approved by the FDA to treat IgA nephropathy in adults.
Skyclarys
On February 28, 2023, Reata Pharmaceuticals announced that the FDA approved a new drug application for the Nrf2 agonist Skyclarys for the treatment of Friedreich's ataxia (Friedreich's ataxia) in adults and adolescents 16 years and older. Skyclarys is the first It is also the only drug suitable for patients with Friedrich's ataxia. The active ingredient of the drug is omaveloxolone, which has received Orphan Drug, Fast Track and Rare Pediatric Disease designations from the US FDA.
Zavzpret
On March 9, 2023, Pfizer announced that the FDA approved its drug Zavzpret. It is used to treat acute migraine in adult patients. The active ingredient of the drug is Zavegepant Hydrochloride. Zavegepant is a third-generation high-affinity, highly selective small molecule CGRP receptor antagonist that reversibly blocks the CGRP receptor, thereby inhibiting The biological activity of CGRP neuropeptide can relieve and prevent migraine attacks.
Daybue
On March 10, 2023, Acadia announced that the FDA approved Daybue for the treatment of Rett syndrome in people over two years of age. Daybue is a new synthetic analog of the amino-terminal tripeptide of insulin-like growth factor I (IGF-1). The active ingredient, trofinetide, is designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function. Daybue became the first, and currently only, drug approved to treat Rett syndrome.
Rezzayo
On March 22, 2023, Cidara Therapeutics announced that the FDA approved Rezzayo, whose active ingredient is Rezafungin Acetate, for the treatment of candidemia and invasive candidiasis in adults. The drug is a novel echinocandin that works by inhibiting beta-1,3-glucose synthase, thereby disrupting the integrity of the fungal cell wall. This is the first approved treatment for invasive Candida infections in more than a decade.
Joenja
On March 24, 2023, Pharming Pharmaceutical Group announced that the FDA approved the marketing of Joenja for the first time. The active ingredient of the drug is Leniolisib Phosphate, which is used to treat PI3Kδ hyperactivation syndrome (APDS) in adults and children aged 12 years and above. Joenja is an oral inhibitor that selectively targets phosphatidylinositol 3-kinase delta (PI3Kδ) and is the first FDA-approved therapy for APDS.
Qalsody
On April 25, 2023, Biogen announced that the FDA approved the marketing of Qalsody. The active ingredient of the drug is tofersen for the treatment of adults with amyotrophic lateral sclerosis (ALS, ALS) with SOD1 gene mutations. This is More than 200 years after the discovery of ALS, this is the fourth drug to treat ALS that has been launched globally, and it is also the only biomarker-based therapy to receive accelerated approval from the FDA for the treatment of ALS related to genetic mutations.
Veozah
On May 12, 2023, Astellas announced that the FDA approved the listing of Veozah. The active ingredient of the drug is Fezolinetant, which is used to treat moderate to severe vasomotor syndrome (VMS) caused by menopause. Fezolinetant is an NK3 receptor antagonist. By antagonizing NK3 receptors, blocking the combination of neurokinin B (NKB) and kisspeptin/neurokinin B/dynorphin (KNDy) neurons, it regulates neuronal activity in the thermoregulatory center, ultimately restoring balance and alleviating VMS.
Miebo
On May 18, 2023, Bausch+Lomb Corp. and Novaliq GmbH announced that the FDA approved the marketing of Miebo. The active ingredient of the drug is perfluorhexyloctane, which is used to treat the signs and symptoms of dry eye syndrome. Miebo, a fluorinated alkane, is an anhydrous, one-component, preservative-free eye solution that is the first and only FDA-approved eye solution to directly target tear evaporation for dry eye disease treatment method.
Xacduro
On May 23, 2023, Innoviva announced that the FDA approved the marketing of Xacduro, which contains the active ingredients Sulbactam and Durlobactam, for the treatment of hospital-acquired bacteria caused by susceptible strains of Acinetobacter baumannii acetate in people 18 years of age or older. pneumonia and ventilator-associated bacterial pneumonia. In this combination, Sulbactam is a drug that is structurally related to penicillin and is responsible for killing Acinetobacter baumannii, while Durlobactam protects Sulbactam from being degraded by enzymes produced by Acinetobacter baumannii.
Posluma
On May 25, 2023, Blue Earth Diagnostics announced that the FDA approved the listing of Posluma, whose active ingredient is Flotufolastat F-18 Gallium, for use in radioactive positron emission tomography (PET) detection of prostate-specific membrane antigen (PSMA) positivity. lesions, in men with suspected metastatic prostate cancer who are candidates for initial definitive therapy or in whom recurrence is suspected due to elevated serum prostate-specific antigen (PSA) levels.
Paxlovid
On May 25, 2023, Pfizer announced that the FDA approved the marketing of Paxlovid. The active ingredients of the drug are Nirmatrelvir and Ritonavir. It is used to treat adult patients with mild to moderate COVID-19 infection. Paxlovid is the fourth FDA-approved drug. The first drug used to treat COVID-19 in adults and the only oral antiviral drug.
Inpefa
On May 26, 2023, Lexicon Pharmaceuticals announced that the FDA approved the listing of Inpefa. The active ingredient of the drug is sotagliflozin. Inpefa is a sodium-glucose co-transporter 2 (SGLT2) inhibitor used to reduce the risk of heart failure or chronic Risk of cardiovascular death, emergency heart failure visits, and hospitalization for heart failure in adults with kidney disease, type 2 diabetes, and other cardiovascular risk factors.
Litfulo
On June 23, 2023, Pfizer announced that the FDA approved Litfulo, whose active ingredient is Ritlecitinib Tosylate, for use in patients with severe alopecia areata aged 12 years and above. Litfulo is a kinase inhibitor that inhibits Janus kinase 3 (JAK3) and tyrosine kinases expressed in the hepatocellular carcinoma (TEC) kinase family.
Vanflyta
On July 20, 2023, Daiichi Sankyo announced that the FDA approved the listing of Vanflyta. The active ingredient of the drug is Quizartinib Dihydrochloride, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) with FLT3-ITD mutations. Quizartinib is the first FLT3 inhibitor approved by the FDA specifically for the treatment of FLT3-ITD-positive AML, and covers the three treatment phases of newly diagnosed AML - induction, consolidation and maintenance therapy in non-transplant patients.
Xdemvy
On July 24, 2023, Tarsus announced that the FDA approved Xdemvy, whose active ingredient is Lotilaner, for the treatment of Demodex blepharitis. Lotilaner is a mite-selective inhibitor of gamma-aminobutyric acid (GABA)-gated chloride channels. Inhibition of these GABA chloride channels causes paralysis in the target organism, resulting in its death. The drug directly targets Demodex, the source of Demodex blepharitis. This marks the first and only FDA-approved radical treatment specifically for Demodex.
Izervay
On August 4, 2023, Astellas Pharma announced that the FDA approved the marketing of Izervay developed by its subsidiary Iveric Bio. The active ingredient of the drug is Avacincaptad Pegol Sodium, which is used to treat geographic atrophy (GA) caused by macular degeneration (AMD). Izervay is a new C5 complement protein inhibitor, containing avacincaptad pegol, which weakens the activity of the complement system through targeted attack on C5, thereby potentially slowing the development of map atrophy.
Sohonos
On August 16, 2023, Ipsen announced that the FDA approved the marketing of Sohonos. The active ingredient of the drug is Palovarotene, which is used to reduce the production of new ossified tissue in patients with progressive muscular ossification (FOP). Palovarotene is an oral, selective retinoic acid receptor gamma (RARγ) agonist that mediates interactions between receptors, growth factors and proteins in the retin signaling pathway to reduce the development of new abnormal bones. form. Sohonos is the first FDA-approved drug to treat FOP.
Aphexda
On September 8, 2023, BioLineRx announced that the FDA approved the listing of Aphexda. The active ingredient of the drug is Motixafortide Acetate. Aphexda is a hematopoietic stem cell mobilizing agent suitable for use in combination with filgrastim (G-CSF) to mobilize multiple myeloma. The patient's hematopoietic stem cells are transferred to the peripheral blood for collection and subsequent autologous transplantation. Aphexda is an innovative CXCR4 inhibitor administered via subcutaneous injection. Aphexda is the first innovative drug to receive FDA approval for multiple myeloma stem cell mobilization in ten years.
Ojjaara
On September 15, 2023, GSK announced that the FDA approved the marketing of Ojjaara. The active ingredient of the drug is Momelotinib Dihydrochloride, which is suitable for the treatment of moderate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis ( polycythemia vera and essential thrombocythemia) in adults with anemia. Ojjaara is a new drug with a differentiated mechanism of action that targets JAK1, JAK2 and ALK2. Ojjaara is the first and only treatment suitable for patients with myelofibrosis and anemia.
Exxua
On September 22, 2023, Fabre-Kramer Pharmaceuticals announced that the FDA approved the listing of Exxua. The active ingredient of the drug is Gepirone Hydrochloride, which is suitable for the treatment of adult depression (MDD). Exxua is the first oral selective 5-HT1A receptor agonist approved by the FDA for the treatment of MDD.
Rivfloza
On September 29, 2023, NovoNordisk announced that the FDA approved the listing of its RNAi therapy Rivfloza injection. The active ingredient of the drug is Nedosiran Sodium. It is suitable for reducing the risk of primary hyperoxaluria type 1 aged 9 years and above and with relatively low renal function. better urinary oxalate levels in patients with Rivfloza, the first approved RNAi therapy developed by Novo Nordisk. Rivfloza binds to the mRNA expressing liver lactate dehydrogenase (LDH) and inhibits the expression of this protein, significantly reducing the overproduction of oxalate.
Velsipity
On October 12, 2023, Pfizer announced that the FDA approved the marketing of Velsipity. The active ingredient of the drug is Etrasimod Arginine. Velsipity is a sphingosine 1-phosphate receptor modulator that can partially and reversibly block lymphocytes. The ability to efflux from lymphoid organs, thereby reducing the number of lymphocytes in the peripheral blood, is indicated for the treatment of moderately to severely active ulcerative colitis in adults.
Zilbrysq
On October 17, 2023, UCB announced that the FDA approved the marketing of Zilbrysq. The active ingredient of the drug is Zilucoplan Sodium, which is used to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. As a complement C5 inhibitor, Zilbrysq inhibits complement-mediated neuromuscular junction damage through its targeted mechanism of action. Zilbrysq is the first new, once-daily, subcutaneous macrocyclic peptide C5 complement inhibitor approved to treat these patients.
Agamree
On October 26, 2023, Santhera Pharmaceuticals and ReveraGen BioPharma jointly announced that the FDA approved the listing of Agamree. The active ingredient of the drug is Vamorolone. Agamree is a corticosteroid that exerts anti-inflammatory and immunosuppressive effects through the glucocorticoid receptor. It is suitable for Treats Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.
Fruzaqla
On November 8, 2023, Chi-Med and Takeda jointly announced that the FDA approved the marketing application of Fruzaqla. The active ingredient of the drug is fruquintinib, which is used to treat patients who have previously received fluorouracil, oxaliplatin and irinotecan-based treatment. Chemotherapy, anti-vascular endothelial growth factor ("VEGF") therapy, and anti-epidermal growth factor receptor (EGFR) therapy in adults with metastatic colorectal cancer.
Defencath
On November 15, 2023, CorMedix announced that the FDA approved the listing of Defencath. The active ingredients of the drug are Heparin Sodium and Taurolidine. It is suitable for reducing the risk of renal failure in a limited population of adult patients with renal failure who receive long-term hemodialysis through a central venous catheter (CVC). Related bloodstream infections (CRBSI), Heparin is an anticoagulant solution often used on catheters when they are not in use to prevent potentially dangerous blood clots from forming. Taurolidine is a thiadiazine antibacterial agent developed by CorMedix Company. It can be used as a broad-spectrum antibacterial agent against a variety of bacteria.
Augtyro
On November 15, 2023, BRISTOL announced that the FDA approved Augtyro, whose active ingredient is repotrectinib, for the treatment of adult patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC). Repotrectinib is the first new generation ROS1, pan-TRK and ALK inhibitor, which has the advantages of stronger anti-cancer efficacy, multi-target broad-spectrum anti-cancer, and the potential to overcome drug resistance.
Truqap
On November 16, 2023, Astrazeneca announced that the FDA approved the listing of Truqap. The active ingredient of the drug is Capivasertib. Truqap is used in combination with Faslodex for the treatment of hormone receptor (HR) positive and human epidermal growth factor receptor 2 (HER2) negative. Adult patients with locally advanced or metastatic breast cancer. Truqap is a first-in-class AKT inhibitor and oral selective adenosine triphosphate (ATP) competitive inhibitor that inhibits all 3 isoforms of the serine/threonine kinase AKT (AKT1/2/3).
Ogsiveo
On November 27, 2023, SpringWorks Therapeutics announced that the FDA approved the marketing of Ogsiveo. The active ingredient of the drug is Nirogacestat Hydrobromide. Ogsiveo is an oral gamma secretase inhibitor for the treatment of adult patients with progressive desmoid tumors who require systemic therapy. This product is the first desmoid tumor treatment approved for marketing. The FDA previously granted Nirogacestat Breakthrough Therapy, Fast Track, and Orphan Drug designations for the treatment of desmoid tumors.
Fabhalta
On December 5, 2023, Novartis announced that the FDA approved the listing of Fabhalta. The active ingredient of the drug is Iptacopan Hydrochloride, which is suitable for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta is a complement factor B inhibitor that acts proximally in the immune system's alternative complement pathway to comprehensively control red blood cell (RBC) destruction both inside and outside blood vessels.
Filsuvez
On December 18, 2023, AMRYT announced that the FDA approved the marketing of Filsuvez. The active ingredient of the drug is Birch Triterpenes, which is suitable for the treatment of junctional or atrophic epidermolysis in patients 6 months and older.
Wainua
On December 21, 2023, Wainua, jointly developed by AstraZeneca and Ionis Pharmaceuticals, has been approved by the FDA in the United States for the treatment of polyneuropathy with hereditary transthyretin-mediated amyloidosis in adults. Wainua is a Antisense oligonucleotide-GalNAc conjugates cause degradation of mutant and wild-type TTR mRNA by binding to TTR mRNA, thereby reducing serum TTR protein and TTR protein deposition in tissues. It aims to slow the progression of the disease and improve patients' quality of life by inhibiting the production of misfolded mutant TTR proteins.
In summary, the number of new drugs approved by the FDA in 2023 hit a five-year high, and the proportion of drugs with innovative mechanisms reached a new high. Small molecule drugs are still the main force in innovative drugs, while the number of gene therapies approved by CBER hit a record, and innovations continue to emerge.