FDA Grants Rare Pediatric Disease Designation to Vanda’s Investigational Therapy VCA-894A for Ultra-Rare CMT2S
The designation makes Vanda eligible for a Priority Review Voucher upon approval.
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#XY Event-Driven Stocks↓
SkinHealth Systems’ SkinStylus Gains FDA Clearance for Eye Wrinkles, Becoming Only Device Cleared for Three Major Indications
Now the only microneedling device cleared for acne scars, abdominal scars, and eye wrinkles.
Agios Wins FDA Priority Review for First Oral PK Activator in Sickle Cell Disease
PDUFA target date is November 1, 2026. If approved, it would be the first oral PK activator for SCD patients.
JPMorgan Raises Eli Lilly Price Target to $1,400 on Mounjaro’s Obesity and Pediatric Momentum
Health Canada has approved Mounjaro for children with type 2 diabetes.
Aligos Therapeutics Shares Soar on Major China Licensing Agreement and Regulatory Milestone for Chronic Hepatitis B Treatment
Aligos Therapeutics announces $25 million upfront payment and Breakthrough Therapy Designation in China for pevifoscorvir sodium.
Vertex to Acquire Crinetics for $10 Billion in Major Rare Disease Expansion
The deal adds PALSONIFY (paltusotine) and late-stage CAH therapy to Vertex’s portfolio.