Company Product Description Indication Status
Alpine Immune Sciences Inc., of Seattle AlPN-101 Dual T-cell co-stimulation inhibitor Acute graft-vs.-host disease FDA granted 2 orphan designations for prevention and treatment of acute GVHD
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Verdiperstat Oral, brain-penetrant irreversible myeloperoxidase inhibitor Multiple system atrophy FDA granted fast track designation
Pharnext SA, of Paris PXT-3003 Oral fixed-dose combination of baclofen, naltrexone and sorbitol Charcot-Marie-Tooth disease type 1A U.K.’s Medicine and Healthcare products Regulatory Agency granted promising innovative medicine designation for use in patients 16 and older
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide Selective agonist of MC₄ receptor Alstrom syndrome FDA granted orphan designation
Sage Therapeutics Inc., of Cambridge, Mass. Zuranolone Positive allosteric modulator of GABAA receptor Postpartum depression, major depression and major depressive syndrome Following an FDA breakthrough therapy guidance meeting, identified 3 pathways supporting possible filing for approval to treat postpartum depression, acute treatment of major depressive disorder when co-initiated with a new antidepressant and as an episodic therapy in major depressive syndrome patients; 3 short-term clinical studies set to start in 2020
Synairgen plc, of Southampton, U.K. SNG-001 Inhaled formulation of interferon-beta-1a COVID-19 Received expedited approvals from U.K.’s Medicines and Healthcare products Regulatory Agency and Health Research Authority to conduct a clinical trial 


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