Becoming the first treatment for rare genetic disorder Prader-Willi syndrome, breakthrough therapy drug DCCR (diazoxide choline) gained U.S. FDA approval as Vykat XR the evening before its March 27 PDUFA date, sending shares of developer Soleno Therapeutics Inc. up 38%.

Nature Cell Co. Ltd. won U.S. FDA breakthrough therapy designation of its autologous adipose-derived mesenchymal stem cell therapy, Jointstem, March 20, becoming the first Korean company to earn the label in the field of cell therapy.

Rusfertide could become a blockbuster therapy for polycythemia vera, H.C. Wainwright analyst Douglas Tsao wrote March 4 after the injectable hepcidin mimetic peptide hit its primary endpoint and all four key secondary endpoints in the ongoing phase III Verify study.