New Chemical Entities
Mary Johnson (han at labome dot com)
Synatom Research, Princeton, New Jersey, United States
DOI
//dx.doi.org/10.13070/mm.en.14.3205
Date
last modified : 2025-08-03; original version : 2024-05-23
Cite as
MATER METHODS 2024;14:3205
Abstract

A select list of FDA-approved new chemical entities.

2025
  • July 31, VIZZ (aceclidine ophthalmic solution 1.44%) from LENZ Therapeutics. Indicated for the treatment of presbyopia in adults. Aceclidine stimulates cholinergic muscarinic receptors located on smooth muscles.
  • July 28, SEPHIENCE (sepiapterin) from PTC Therapeutics. Indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU), to be used in conjunction with a phenylalanine (Phe)-restricted diet. Sephience is a phenylalanine hydroxylase (PAH) activator.
  • July 23, ANZUPGO (delgocitinib) from LEO Pharma. Indicated for the topical treatment of moderate-to-severe chronic hand eczema (CHE) in adults who have had an inadequate response to, or for whom topical corticosteroids are not advisable. Anzupgo is a topical pan-Janus kinase (JAK) inhibitor that blocks the activity of JAK1, JAK2, JAK3, and tyrosine kinase 2 (TYK2).
  • July 7, EKTERLY (sebetralstat) from KalVista Pharmaceuticals. Indicated for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. Sebetralstat is a novel plasma kallikrein inhibitor.
  • July 2, ZEGFROVYTM (sunvozertinib) from Dizal Jiangsu. Indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. Sunvozertinib is an inhibitor of EGFR.
  • June 11, IBTROZITM (taletrectinib) from Nuvation Bio. Indicated for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer. Taletrectinib is an inhibitor of tyrosine kinase ROS1.
  • May 28, TRYPTYR (acoltremon ophthalmic solution) from Alcon Labs. Indicated for the treatment of the signs and symptoms of dry eye disease. Acoltremon is an agonist of transient receptor potential melastatin 8 (TRPM8) thermoreceptors.
  • May 8, AVMAPKI FAKZYNJA CO-PACK, a combination of avutometinib and defactinib, from Verastem. Indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. Avutometinib is a MEK1 inhibitor and defactinib is an inhibitor of FAK and proline-rich tyrosine kinase-2 (Pyk2).
  • April 2, VANRAFIA (atrasentan) from Novartis Pharmaceuticals Corporation. Indicated for proteinuria reduction in primary immunoglobulin A (IgA) nephropathy. Atrasentan is an endothelin A receptor antagonist reducing proteinuria in glomerular disease.
  • March 28, QFITLIA (fitusiran) from Sanofi. Indicated for the prophylactic treatment of hemophilia A or B, with or without inhibitors. Fitusiran is an antithrombin-directed small interfering ribonucleic acid (siRNA) targeting antithrombin to promote hemostasis.
  • March 25, BLUJEPA (gepotidacin) from GlaxoSmithKline. Indicated for the treatment of uncomplicated urinary tract infections. Gepotidacin is a triazaacenaphthylene bacterial type II topoisomerase inhibitor targeting bacterial DNA replication. Targeted Genes: GyrA (DNA gyrase subunit A) and ParC (topoisomerase IV subunit C).
  • February 14, ROMVIMZA (vimseltinib) from Deciphera Pharmaceuticals / Ono Pharma. Indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. Vimseltinib is a switch-control kinase inhibitor of colony stimulating factor 1 receptor.
  • February 11, GOMEKLI (mirdametinib) from SpringWorks Therapeutics. Indicated for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. Mirdametinib is a kinase inhibitor targeting mitogen-activated protein kinase kinases 1 and 2 ( MEK1 and MEK2) in the MAPK pathway.
  • January 30, JOURNAVX (suzetrigine) from Vertex. Indicated for the treatment of moderate to severe acute pain in adults. Suzetrigine is a selective blocker of the NaV1.8 voltage-gated sodium channel expressed in nociceptors.
2024
  • December 20, ALYFTREK (vanzacaftor, tezacaftor, and deutivacaftor tablets) from Vertex. Indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. Vanzacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of select mutant forms of CFTR (including F508del-CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Deutivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface.
  • December 19, TRYNGOLZA olezarsen from Ionis Pharmaceuticals. Indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome. Olezarsen is an antisense oligonucleotide-GalNAc3 conjugate that binds to apoC-III mRNA leading to mRNA degradation and resulting in a reduction of serum apoC-III protein.
  • December 18, ENSACOVETM ensartinib from Xcovery. Indicated for the treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) who have not previously received an ALK-inhibitor. Ensartinib is a kinase inhibitor of anaplastic lymphoma kinase and inhibits other kinases including MET and ROS1.
  • December 13, CRENESSITY crinecerfont from Neurocrine Biosciences. Indicated as adjunctive treatment to glucocorticoid replacement to control androgens in adults and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia. Crinecerfont is a selective corticotropin-releasing factor type 1 receptor antagonist.
  • November 22, RAPIBLYK landiolol from AOP Orphan Pharmaceuticals GmbH. Indicated for the short-term reduction of ventricular rate in adults with supraventricular tachycardia including atrial fibrillation and atrial flutter. RAPIBLYK is a selective beta-1-adrenoreceptor antagonist.
  • November 22, ATTRUBY acoramidis from BridgeBio Pharma. Indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascularrelated hospitalization. Acoramidis binds transthyretin at thyroxine binding sites and slows dissociation of the TTR tetramer into its constituent monomers, the rate-limiting step in amyloidogenesis.
  • November 15, REVUFORJ revumenib from Syndax Pharmaceuticals. Indicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in patients 1 year and older. Revumenib is a menin inhibitor and blocks the interaction of both wild-type lysine methyltransferase 2A (KMT2A) and KMT2A fusion proteins with menin.
  • October 10, ITOVEBI inavolisib from Genentech. Indicated in combination with palbociclib and fulvestrant for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy. Inavolisib induces the degradation of mutated PI3K catalytic alpha subunit p110alpha / PIK3CA.
  • September 26, COBENFY xanomeline and trospium chloride from Bristol-Myers Squibb. Indicated for the treatment of schizophrenia in adults. Xanomeline binds to muscarinic receptors M1, M2, M3, M4, and M5 receptors and exhibits higher agonist activity at the M1 and M4 receptors. Trospium chloride antagonizes the muscarinic receptors primarily in peripheral tissues.
  • September 24, AQNEURSA levacetylleucine from IntraBio. Indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. The distinct molecular target for levacetylleucine in the treatment of NPC is unknown.
  • September 20, MIPLYFFA arimoclomol from Zevra Therapeutics. Indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C in patients 2 years of age and older. The mechanism(s) by which arimoclomol exerts its clinical effects in patients with Niemann-Pick disease type C is unknown.
  • August 19, LAZCLUZE lazertinib from Johnson & Johnson. Indicated in combination with amivantamab for the first-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test. Lazertinib is an inhibitor of epidermal growth factor receptor (EGFR) that inhibits EGFR exon 19 deletions and exon 21 L858R substitution mutations at lower concentrations than wildtype EGFR.
  • August 14, LIVDELZI seladelpar from Gilead Sciences. Indicated for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. Seladelpar is a peroxisome proliferator-activated receptor-delta agonist.
  • August 9, YORVIPATH palopegteriparatide from Ascendis Pharma. Indicated for the treatment of hypoparathyroidism in adults. Palopegteriparatide releases parathyroid hormone analog (1-34) at physiological conditions.
  • August 6, VORANIGO vorasidenib from Servier Pharmaceuticals. Indicated for the treatment of patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation following surgery including biopsy, sub-total resection, or gross total resection. Vorasidenib inhibits isocitrate dehydrogenase-1 and 2.
  • July 25, LEQSELVI deuruxolitinib from Sun Pharmaceutical Industries Limited. Indicated for the treatment of adults with severe alopecia areata. Deuruxolitinib is a Janus kinase (JAK) inhibitor. In an in vitro kinase activity assay, deuruxolitinib had greater inhibitory potency for JAK1, JAK2 and TYK2 relative to JAK3.
  • June 26, OHTUVAYRE ensifentrine from Verona Pharma. Indicated for the maintenance treatment of chronic obstructive pulmonary disease in adult patients. Ensifentrine inhibits PDE3 and PDE4 enzymes such as PDE3A and PDE3B.
  • June 18, SOFDRA sofpironium from Botanix. Indicated for the treatment of primary axillary hyperhidrosis in adults and pediatric patients 9 years of age and older. Sofpironium bromide is a competitive inhibitor of acetylcholine receptors that are located on certain peripheral tissues, including sweat glands.
  • June 10, IQIRVO elafibranor from Ipsen. Indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. Elafibranor and its main active metabolite GFT1007 are peroxisome proliferator-activated receptor (PPAR) agonists, both of which activate PPAR-alpha, PPAR-gamma, and PPAR-delta in vitro.
  • June 6, RYTELO imetelstat from Geron. Indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents. Imetelstat binds to the template region of the RNA component of human telomerase, inhibits telomerase enzymatic activity and prevents telomere binding.
  • April 26, XOLREMDI mavorixafor from X4 Pharmaceuticals. Indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. Mavorixafor blocks the binding of stromal-derived factor-1 alpha / CXC Chemokine Ligand 12 to the CXC Chemokine Receptor 4.
  • April 23, OJEMDA tovorafenib from Day One Biopharmaceuticals. Indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric lowgrade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. Tovorafenib inhibits mutant BRAF V600E, wild-type BRAF, and wild-type CRAF kinases.
  • March 27, VAFSEO vadadustat from Akebia Therapeutics. Indicated for the treatment of anemia due to chronic kidney disease in adults who have been receiving dialysis for at least three months. Vadadustat is a reversible inhibitor of HIF-prolyl-4-hydroxylases (PH)1, PH2, and PH3.
  • March 21, DUVYZAT givinostat from Italfarmaco. Indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. DUVYZAT inhibits histone deacetylases.
  • March 19, TRYVIOTM aprocitentan from Idorsia. indicated for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs. Aprocitentan inhibits the binding of endothelin-1 to ETA and ETB receptors.
  • March 14, REZDIFFRA resmetirom from Madrigal Pharmaceuticals. Indicated, in conjunction with diet and exercise, for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). Resmetirom is a thyroid hormone receptor-beta agonist.
2023
  • November 27, OGSIVEO nirogacestat from SpringWorks Therapeutics. Indicated for adults with progressing desmoid tumors who require systemic treatment. Nirogacestat inhibits gamma secretase and thus blocks proteolytic activation of the Notch receptor.
  • November 15, AUGTYROTM repotrectinib from Bristol-Myers Squibb. Indicated for the treatment of adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer. Repotrectinib is an inhibitor of proto-oncogene tyrosine-protein kinase ROS1 (ROS1) and of the tropomyosin receptor tyrosine kinases.
2022
2020
  • December 13, ORLADEYO berotralstat from BioCryst. Indicated for prophylaxis to prevent attacks of hereditary angioedema in patients 12 years and older. Berotralstat inhibits plasma kallikrein.
  • April 10, KOSELUGO selumetinib from AstraZeneca. Indicated for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas. Selumetinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2.
2019
  • September 12, IBSRELA tenapanor from Ardelyx. Indicated for treatment of irritable bowel syndrome with constipation in adults. Tenapanor hydrochloride inhibits sodium/hydrogen exchanger 3.
  • August 15, ROZLYTREK entrectinib from Genentech / Roche. Indicated for the treatment of: 1) adults with metastatic non-small cell lung cancer whose tumors are ROS1-positive; 2) patients 12 years of age and older with solid tumors that: a) have a neurotrophic tyrosine receptor kinase gene fusion without a known acquired resistance mutation, b) are metastatic or where surgical resection is likely to result in severe morbidity, and c) have progressed following treatment or have no satisfactory alternative therapy. Entrectinib is an inhibitor of the tropomyosin receptor tyrosine kinases (TRK) TRKA, TRKB, and TRKC, proto-oncogene tyrosine-protein kinase ROS1, and anaplastic lymphoma kinase with IC50 values of 0.1 to 2 nM.
2016
  • December 23, SPINRAZA nusinersen from Biogen. Indicated for the treatment of spinal muscular atrophy. Nusinersen, a modified antisense oligonucleotide, binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript, and increase the production of full-length SMN protein.
  • September 19, Exondys 51 eteplirsen from Sarepta Therapeutics. Indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Eteplirsen, an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass, binds to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing, and enables the production of a truncated yet functional dystrophin protein.
  • July 12, XIIDRA lifitegrast ophthalmic solution from Shire. Indicated for the treatment of the signs and symptoms of dry eye disease. Lifitegrast binds to the integrin lymphocyte function-associated antigen-1 (LFA-1) , a cell surface protein found on leukocytes and blocks the interaction of LFA-1 with its cognate ligand intercellular adhesion molecule-1.
2012
2011
ISSN : 2329-5139