Shots:
The US FDA has granted BTD to venglustat for the treatment of neurological manifestations of type 3 Gaucher disease (GD3)
Designation was based on the P-III (LEAP2MONO) study assessing venglustat (QD, PO) vs imiglucerase (Q2W) in 43 pts (≥12yrs.), which showed significant improvements in neurological symptoms measured by a global test scores, incl. mSARA & RBANS
Venglustat received US…
Shots:
Mirum Pharmaceuticals has entered into an agreement to acquire Bluejay Therapeutics, gaining global rights to brelovitug with Breakthrough Therapy and PRIME designations, and expanding Mirum’s rare liver disease portfolio
As per the deal, Mirum will acquire all Bluejay shares for $250M in cash and $370M in Mirum stock, plus up to $200M in sales-based…

