Company Product Description Indication Status
American Cryostem Corp., of Eatontown, N.J. Atcell Autologous adipose-derived mesenchymal stem cell therapy Post-concussion syndrome  FDA cleared IND for phase I trial of retired athletes and military service members with confirmed diagnosis from 3 or more concussion or mild traumatic brain injuries; study expected to begin in fourth quarter of 2020
Athenex Inc., of Buffalo, N.Y. TCRT-ESO-A2 Autologous T-cell receptor T-cell therapy Solid tumors FDA cleared IND for phase I trial targeting NY-ESO-1-positive tumors in people who are HLA-A 02:01-positive 
Athersys Inc., of Cleveland, Ohio Multistem Multipotent adult progenitor cell therapy Acute respiratory distress syndrome FDA granted regenerative medicine advanced therapy designation
Bluebird Bio Inc., of Cambridge, Mass. Lentiglobin (betibeglogene autotemcel) HBB gene stimulator Sickle cell disease EMA granted PRIME designation
Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J., and Symbio Pharmaceuticals Ltd., of Tokyo Treakisym (bendamustine hydrochloride) PARP modulator Non-Hodgkin lymphoma; mantle cell lymphoma; chronic lymphocytic leukemia Japan PMDA approved ready-to-dilute liquid formulation to treat approved indications, replacing lyophilized formulation at launch in January 2021
GW Pharmaceuticals plc, of London Epidyolex (cannabidiol) Dual cannabinoid CB1/CB2 modulator Lennox-Gastaut syndrome; Dravet syndrome Australian TGA approved to treat seizures associated with indications in people 2 and older 
Neurorx Inc., of Radnor, Pa. RLF-100 (aviptadil) Vasoactive intestinal polypeptide Prevention of respiratory failure in COVID-19 infection Emergency use authorization submitted to FDA using same protocol as expanded access authorized in July 2020
Novartis AG, of Basel, Switzerland AVXS-101 intrathecal
SMN1 gene stimulator
Spinal muscular atrophy FDA recommended pivotal confirmatory study to supplement existing Strong data to treat older individuals with SMA
Pfizer Inc., of New York Xalkori (crizotinib)  ALK tyrosine kinase inhibitor Anaplastic large cell lymphoma  FDA accepted sNDA, granting priority review, to treat children with relapsed or refractory systemic disease that is ALK positive, setting PDUFA date in January 2021
Sinovac Biotech Ltd., of Beijing Coronavac Adjuvant inactivated SARS-CoV-2 vaccine COVID-19 infection China NMPA authorized phase I/II trial in children and adolescents ages 3 to 17

Notes

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