Company Product Description Indication Status
Aruvant Sciences Holdings Ltd., of Basel, Switzerland ARU-1801 HBG gene stimulator Sickle cell disease FDA granted orphan drug designation in the indication
Baudax Bio Inc., of Malvern, Pa. Meloxicam (intravenous) Cyclooxygenase 2 inhibitor Pain FDA set Feb. 20, 2020, PDUFA date for NDA to treat moderate to severe pain
Celularity Inc., of Warren, N.J. CYNK-001 Placental hematopoietic stem cell-derived allogeneic NK cell therapy Glioblastoma multiforme FDA cleared IND application
Genprex Inc., of Austin, Texas Oncoprex TUSC2 gene stimulator Non-small-cell lung cancer FDA granted fast track designation for the immunogene therapy in combination with osimertinib (Tagrisso, Astrazeneca plc)
Inhibitor Therapeutics Inc., of Tampa, Fla. Suba-itraconazole Lanosterol-14 demethylase inhibitor Prostate cancer FDA cleared IND application for phase IIb combination study with docetaxel and prednisone in metastatic castration-resistant disease, with radiographic progression-free survival as primary endpoint
Jazz Pharmaceuticals plc, of Dublin JZP-258 (sodium oxybate + potassium oxybate + calcium oxybate + magnesium oxybate) GABA B receptor agonist Cataplexy; narcolepsy NDA submitted to FDA to treat cataplexy and excessive daytime sleepiness in individuals 7 and older with narcolepsy; with submission, company redeemed rare pediatric disease priority review voucher, acquired in 2018 from Spark Therapeutics Inc.
Ra Pharmaceuticals Inc., of Cambridge, Mass. Zilucoplan Amyotrophic lateral sclerosis Complement C5 factor inhibitor FDA cleared IND for Healey ALS platform trial, with zilucoplan selected as an initial candidate
Supernus Pharmaceuticals Inc., of Rockville, Md. SPN-812 (viloxazine hydrochloride) Serotonin norepinephrine modulating agent Attention deficit hyperactivity disorder FDA accepted NDA, assigning PDUFA date of Nov. 8, 2020


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

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