Company Product Description Indication Status
Antengene Corp. Ltd., of Shanghai ATG-016 (eltanexor)  Selective inhibitor of nuclear export Intermediate- and higher-risk myelodysplastic syndrome after failure of hypomethylating agent-based therapy China's National Medical Products Administration approved the start of a phase I/II study testing the pharmacokinetics, safety and efficacy of ATG-016 monotherapy
Aprea Therapeutics Inc., of Boston Eprenetapopt Restores wild-type p53 conformation and function TP53-mutant acute myeloid leukemia FDA granted fast track designation
Ars Pharmaceuticals, of San Diego, and Recordati SpA, of Milan Neffy (epinephrine) Adrenaline nasal spray  Severe allergic reactions, including anaphylaxis EMA accepted the MAA
Astellas Pharma Inc., of Tokyo, and Fibrogen Inc., of San Francisco Evrenzo (roxadustat) HIF-PH inhibitor Anemia in chronic kidney disease Japan's Ministry of Health, Labour and Welfare approved the drug
Astrazeneca plc, of Cambridge, U.K. Forxiga (dapagliflozin) SGLT2 inhibitor Chronic heart failure Japan's Ministry of Health, Labour and Welfare approved the drug
Cassava Sciences Inc., of Austin, Texas Simufilam Restores the normal shape and function of altered filamin A Alzheimer's disease World Health Organization advised the U.S. Adopted Names Council to modify the chemical name of the company’s lead drug candidate to simufilam due to a potential trademark conflict with a drug marketed in the Far East
Exelixis Inc., of Alameda, Calif., and Takeda Pharmaceutical Co. Ltd., of Tokyo Cabometyx (cabozantinib) Kinase inhibitor Unresectable hepatocellular carcinoma that has progressed after prior systemic therapy Japan's Ministry of Health, Labour and Welfare approved the drug
Innovation Pharmaceuticals Inc., of Wakefield, Mass. Brilacidin Defensin mimetic Hospitalized COVID-19 Filed documentation with regulatory agencies, including an IND with the FDA, for conduct of a multinational phase II study
Iterum Therapeutics plc., of Dublin Oral sulopenem Penem anti-infective Uncomplicated urinary tract infections in patients with a quinolone non-susceptible pathogen Submitted an NDA to the FDA
Kadmon Inc., of New York Belumosudil (KD-025) Rho-associated coiled-coil kinase 2 inhibitor Chronic graft-vs.-host disease FDA accepted the NDA with a priority review; PDUFA target action date is May 30, 2021
Mirum Pharmaceuticals Inc., of Foster City, Calif. Maralixibat Apical sodium-dependent bile acid transporter inhibitor Progressive familial intrahepatic cholestasis type 2 EMA accepted the MAA
Otonomy Inc., of San Diego Otividex Sustained-exposure formulation of dexamethasone Ménière’s disease FDA confirmed use of the negative binomial model for analysis of the endpoint for the ongoing phase III study
Prevail Therapeutics Inc., of New York PR-006  AAV9 gene therapy expressing GRN Frontotemporal dementia European Commission granted orphan designation
Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici SpA, of Parma, Italy Pegunigalsidase alfa (PRX–102) Chemically modified stabilized version of alpha-galactosidase-A Fabry disease FDA extended the PDUFA target action date by 3 months to April 27, 2020
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Dupixent (dupilumab) Monoclonal antibody targeting interleukin-4 and interleukin-13 Severe atopic dermatitis European Commission extended the marketing authorization to include children 6 to 11 years of age
Rhythm Pharmaceuticals Inc., of Boston Imcivree (setmelanotide) Melanocortin-4 receptor agonist  Obesity due to pro-opiomelanocortin, proprotein convertase subtilisin/kexin type 1 or leptin receptor deficiency FDA approved the drug
Sedana Medical AB, of Stockholm Sedaconda (isoflurane) General anesthetic  Sedation Submitted marketing application to German Medicines Agency BfArM and a number of other agencies through the decentralized procedure 
Vertex Pharmaceuticals Inc., of Boston Symkevi (tezacaftor/ivacaftor) plus Kalydeco (ivacaftor) CFTR corrector/CFTR regulator  Cystic fibrosis European Commission approved the label extension to include patients ages 6 and older who have 2 copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or 1 copy of the F508del mutation and 1 copy of 1 of 14 mutations in the CFTR gene that result in residual CFTR activity
Y-mabs Therapeutics Inc., of New York Danyelza (naxitamab-gqgk) Monoclonal antibody targeting ganglioside GD2 Relapsed or refractory high-risk neuroblastoma in the bone or bone marrow  FDA approved the drug for use in patients who have demonstrated a partial response, minor response or stable disease to prior therapy
Zymeworks Inc, of Vancouver, British Columbia Zanidatamab Bispecific antibody targeting 2 non-overlapping epitopes of HER2 Previously treated HER2 gene-amplified biliary tract cancer FDA granted breakthrough designation

Notes

For more information about individual companies and/or products, see Cortellis.