Company Product Description Indication Status
Amo Pharma Ltd., of London AMO-02 Glycogen synthase kinase-3 beta inhibitor Congenital myotonic dystrophy FDA granted rare pediatric disease designation
Astrazeneca plc, of Cambridge, U.K. Calquence (acalabrutinib) Next-generation BTK inhibitor Chronic lymphocytic leukemia Approved in EU for adults
Axovant Gene Therapies Ltd., of New York AXO-AAV-GM2 Gene therapy Tay-Sachs disease and Sandhoff disease FDA lifted clinical hold and cleared IND to initiate registrational study
Bayer AG, of Leverkusen, Germany Finerenone Nonsteroidal, selective mineralocorticoid receptor antagonist Chronic kidney disease and type 2 diabetes Submitted NDA to FDA and MAA to EMA
Eusa Pharma Ltd., of Hemel Hempstead, U.K., and Beigene Ltd., of Beijing, China Qarziba (dinutuximab beta) Targeted immunotherapy High-risk neuroblastoma BLA accepted by China’s NMPA and granted priority review
Impel Neuropharma Inc., of Seattle INP-104 (dihydroergotamine mesylate) 5-HT 1d receptor agonist; 5-HT 1f receptor agonist; 5-HT 1b receptor agonist Migraine Submitted NDA to FDA for use in acute treatment of migraine with or without aura in adults
Liminal Biosciences Inc., of Laval, Quebec Ryplazim (plasminogen) Plasmin stimulator Congenital plasminogen deficiency FDA extended review period for BLA by 3 months, setting new PDUFA date of June 5, 2021
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 Vaccine COVID-19 FDA granted fast track designation
Orphazyme A/S, of Copenhagen, Denmark Arimoclomol Chaperonin stimulator; Hsp70 stimulator Niemann-Pick disease type C Submitted MAA to EMA
Sorrento Therapeutics Inc., of San Diego COVI-AMG (STI-2020) COVID-19 Spike glycoprotein inhibitor COVID-19 Filed IND to test in healthy volunteers and in patients with mild symptoms


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