|Acadia Pharmaceuticals Inc., of San Diego, and Neuren Pharmaceuticals Ltd., of Melbourne, Australia||Trofinetide||Synthetic analogue of the amino‐terminal tripeptide of IGF-1||Rett syndrome||FDA granted rare pediatric disease designation|
|Cytokinetics Inc., of South San Francisco||Reldesemtiv||Fast skeletal muscle troponin activator||Amyotrophic lateral sclerosis||EMA granted orphan designation|
|Janssen Pharmaceuticals Cos., of Allschwil, Switzerland, part of Johnson & Johnson||Ponesimod||S1P1R modulator||Relapsing multiple sclerosis||Submitted MAA to EMA seeking approval for use in adults|
|Lipocine Inc., of Salt Lake City||Tlando||Oral testosterone||Hypogonadism||FDA acknowledged receipt of resubmitted NDA seeking approval of the TRT candidate for use in adult males for conditions associated with a deficiency of endogenous testosterone|
|Nucana plc, of Edinburgh, U.K.||Acelarin||Derived from nucleoside analogue gemcitabine||Biliary tract cancer||EMA’s Committee for Orphan Medicinal Products issued positive opinion for orphan designation|
|Zosano Pharma Corp., of Fremont, Calif.||Qtrypta||Formulation of zolmitriptan delivered using intracutaneous microneedle system||Migraine||FDA accepted the NDA under the 505(b)(2) pathway, setting a PDUFA date of Oct. 20, 2020|
For more information about individual companies and/or products, see Cortellis.