Japan’s MHLW grants orphan status to Kyowa and Orchard’s MLD therapy
OTL-200, also referred to as atidarsagene autotemcel, addresses the fundamental genetic issue in MLD.
Regulation
FDA ‘green list’ flagging illegal GLP-1RA compounders is long overdue
Big pharma companies hit with FDA warning letters in drug ad clampdown
FDA chief blames decades of regulatory failure for misleading pharma ads
Five takeaway quotes from RFK Jr’s combative Senate hearing
FDA creates new pathway for rare disease drugs, analysts unconvinced
Novo’s facility issues delay FDA decisions on Regeneron’s Eylea HD
Prasad’s shock FDA return reignites regulatory uncertainty
