Disc Medicine’s FDA Meeting Just Reset the Bitopertin Timeline: What the Street Should Actually Be Watching
After a Feb 2026 CRL rejection, Disc Medicine secured FDA alignment on a Phase 3 pathway for bitopertin in...
Rare Disease
Sanofi’s Venglustat Priority Review Is a Rare Disease Pricing License, Not Just a Clinical Win
FDA's 6-month priority review clock for Sanofi's venglustat in type 3 Gaucher disease—affecting fewer than 1,000 Americans—sets up one...
Regeneron Priced Otarmeni at Zero — And That’s the Most Expensive Move in Ultra-Rare Disease This Decade
Regeneron's FDA-approved gene therapy Otarmeni targets just 20–50 U.S. births yearly. Its free pricing strategy could cost every rare...
Fortress Biotech’s $205M Voucher Sale Is the Smartest Capital Move No One Is Talking About
Fortress Biotech just converted a rare pediatric disease priority review voucher into $205 million in cash — while the...
Biogen’s SPINRAZA High-Dose Approval Isn’t a Label Update — It’s a Lifecycle Defense Strategy in a Therapy Area That Is Running Out of Time
The FDA's approval of a high-dose nusinersen regimen looks like a routine label expansion. Read it as a commercial...