Company Product Description Indication Status
Cerecin Inc., of Singapore Tricaprilin Medium chain triglyceride Infantile spasms FDA granted rare pediatric disease designation
Eton Pharmaceuticals Inc., of Deer Park, Ill. Dehydrated alcohol Ethanol Methanol poisoning FDA accepted the NDA; PDUFA target action date is May 27, 2021
Neurocrine Biosciences Inc., of San Diego, and Xenon Pharmaceuticals Inc., of Burnaby, British Columbia NBI-921352  Nav1.6 sodium channel inhibitor SCN8A developmental and epileptic encephalopathy syndrome FDA requested nonclinical data to support proposed dosing in the pediatric study; companies plan to respond with a goal of starting the phase II study in 2021
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-COV2 2 monoclonal antibodies targeting SARS-CoV-2 COVID-19 Submitted a request to the FDA for an emergency use authorization
Revelation Biosciences Inc., of Menlo Park, Calif. REVTx‑99 Intranasal formulation of a synthetic version of monophosphoryl lipid A Healthy volunteers (eventually COVID-19) Received acknowledgment by the Therapeutic Goods Administration in Australia to conduct the phase I study of up to 32 participants
Rhythm AI Ltd., of London STAR Mapping System Identifies the drivers of disease for cardiac ablation procedure Persistent atrial fibrillation Received approval from the U.K. Medicines and Healthcare Products Regulatory Agency to proceed with the Rocstar study
X4 Pharmaceuticals Inc., of Boston Mavorixafor Antagonist of CXCR4 WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome FDA granted fast track designation
Xenon Pharmaceuticals Inc., of Burnaby, British Columbia XEN-496  Kv7 potassium channel modulator KCNQ2 developmental and epileptic encephalopathy Received a positive opinion from the EMA's Committee for Orphan Medicinal Products on the request for orphan medicinal product designation
Xortx Therapeutics Inc., of Calgary, Alberta XRx-101 Xanthine oxidase inhibitor Acute kidney injury in COVID-19 Received feedback from pre-IND submission to the FDA on plan to develop the drug

Notes

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