Company Product Description Indication Status
Astrazeneca plc, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Chronic kidney disease FDA granted breakthrough therapy designation
Biomarin Pharmaceutical Inc., of San Rafael, Calif. BMN-307 Gene therapy expressing phenylalanine hydroxylase Phenylketonuria  FDA granted fast track designation
Bluebird Bio Inc., of Cambridge, Mass. Elivaldogene autotemcel (Lenti-D) Gene therapy expressing ABCD1 Cerebral adrenoleukodystrophy EMA accepted the marketing authorization application
Innovation Pharmaceuticals Inc., of Wakefield, Mass. Brilacidin Defensin mimetic COVID-19 Submitted a request for a pre-IND meeting to discuss plans for a 120-patient phase II study
Kyowa Kirin Co. Ltd., of Tokyo Crysvita (burosumab) Monoclonal antibody targeting  fibroblast growth factor 23 X-linked hypophosphatemia European Commission approved the new indication, which includes all adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults
Mesoblast Ltd., of New York Remestemcel-L Allogeneic culture-expanded mesenchymal stem cells Pediatric steroid-refractory acute graft-vs.-host disease FDA issued a complete response letter and requested the company run at least 1 additional randomized, controlled study
Optibiotix Health plc, of Heslington, U.K., and Seed Health Inc., of Venice, Calif. DS-01 Multispecies, multistrain probiotic Irritable bowel syndrome FDA authorized the IND for a phase II study
Ovoca Bio plc, of Dublin BP-101 Synthetic peptide Decreased sexual desire in women, including hypoactive sexual desire disorder Russian Ministry of Heath didn't grant the marketing authorization due to issues with the chemistry, manufacturing and controls and labeling sections of the application
Pharmacyte Biotech Inc., of Laguna Hills, Calif. Cypcaps Genetically engineered human cells encapsulated using the Cell-in-a-Box technology  Locally advanced inoperable pancreatic cancer FDA placed the IND application on hold; the initial notification didn't give a reason for the decision
Sanofi SA, of Paris Avalglucosidase alfa Enzyme replacement Pompe disease EMA accepted the marketing authorization application


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