FDA expands Repatha indication to adults at risk of major cardiovascular events from uncontrolled LDL-C

The FDA also expanded Repatha’s use as monotherapy to include patients with homozygous familial hypercholesterolemia

FDA approves Coya Therapeutics’ trial of COYA 302 for ALS

As part of its partnership with Dr. Reddy’s Laboratories (DRL), FDA acceptance also triggers a $4.2 million milestone payment to Coya

FDA grants ODD to Keros KER-065 for treatment of Duchenne muscular dystrophy

KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB

FDA approved Dawnzera in US as first and only RNA-targeted prophylactic treatment for hereditary angioedema

Dawnzera demonstrated significant and sustained HAE attack rate reduction and long-term disease control

FDA lifts hold on Rocket Pharma's gene therapy trial

Rocket will also collaborate with investigators to implement an immunomodulatory regimen more closely reflecting that administered in the Phase 1 pediatric cohort

FDA approves first immunotherapy for recurrent respiratory papillomatosis

Tonix receives FDA approval of Tonmya for treatment of fibromyalgia

FDA grants breakthrough therapy designation to Ifinatamab Deruxtecan for lung cancer patients

Fourteenth breakthrough therapy designation granted by FDA across the oncology portfolio of Daiichi Sankyo

AAVantgarde Bio receives FDA fast track designation for AAVB-039 in stargardt disease

The therapy delivers the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations

FDA restrict 7-OH opioid products to protect consumers

7-OH is a concentrated byproduct of the kratom plant and has shown potential for abuse by binding to opioid receptors