Rocket’s gene therapy Kresladi wins FDA approval in rare immune disease

Upon approval, Rocket has been awarded a priority review voucher, which currently fetch around $200m on the secondary market.
Healio

FDA approves once-weekly basal insulin for adults with type 2 diabetes

The FDA approved a once-weekly, long-acting basal insulin for adults with type 2 diabetes as an alternative to daily basal ...
STAT

FDA approves Rocket gene therapy for rare immune disorder

The Food and Drug Administration approved on Thursday a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), ...
MedPage Today on MSN

FDA Signals Regulatory Flexibility With Accelerated Approval of Rare Disease Drug

Tividenofusp alfa is the first approved treatment for neurologic complications of Hunter s ...
Fierce Pharma

FDA approves Denali's Hunter syndrome drug, handing rare disease community a win

The FDA has approved Denali Therapeutics’ enzyme replacement therapy for a genetic lysosomal storage disease, breaking the ...

FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...

Denali gains on FDA approval of lead asset (update)

Denali Therapeutics (DNLI) stock jumps on FDA approval of the company's lead asset, Avlayah developed with Royalty Pharma ...
Fierce Pharma

Corcept's lead drug bounces back from FDA snub with different approval as Lifyorli in ovarian cancer

The FDA has given the all-clear to Corcept’s class-first Lifyorli (relacorilant) to treat adults with platinum-resistant ...

FDA approves new higher-dose version of Wegovy shots

European drug regulators approved the higher-dose version of Wegovy in February. Called Wegovy HD, the higher-dose drug ...
TCTMD

FDA Approves JenaValve’s Trilogy for Aortic Regurgitation

This marks the first dedicated transcatheter heart valve approved for high-risk patients with symptomatic severe AR.

Rocket Receives FDA Approval for Genetic Immunodeficiency Treatment

Rocket Pharmaceuticals said the Food and Drug Administration has granted accelerated approval for its therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion ...
CURE

FDA Approves PD-L1 Test to Guide Keytruda Use in Esophageal and GEJ Cancer

FDA approves a PD-L1 test to help identify patients with esophageal or gastroesophageal junction (GEJ) cancer who may benefit ...