Why BridgeBio Pharma, Inc.’s BBP-418 priority review could redefine the future of LGMD treatment
BridgeBio Pharma, Inc.’s BBP-418 wins FDA Priority Review for LGMD2I/R9. Discover what this could mean for rare muscular dystrophy treatment.
Pharma & Biotech
Pharma & Biotech
What PepGen’s 5 mg/kg FREEDOM2 cohort reveals about the risk-reward profile of PGN-EDODM1
PepGen’s FREEDOM2 data hint at PGN-EDODM1 activity in DM1, but bigger questions remain. Read what the 5 mg/kg cohort really changes.
Pharma & Biotech
Can Ono Pharmaceutical bring clearer treatment sequencing to advanced GIST in Japan?
Ono Pharmaceutical has filed ripretinib in Japan for advanced GIST. Read why the move could reshape late-line treatment access and sequencing.
Pharma & Biotech
Gilead targets durable autoimmune remission with $2.18bn Ouro Medicines acquisition
Gilead Sciences acquires Ouro Medicines for up to $2.18bn, adding BCMA T cell engager gamgertamig to its inflammation pipeline. Analysis of what the deal reveals.
Pharma & Biotech
Will pediatric KONFIDENT-KID data accelerate KalVista’s sebetralstat approval in hereditary angioedema?
KalVista's sebetralstat pediatric trial data heads to Madrid. What it means for the HAE on-demand market and a 2026 FDA filing. Read the analysis.
Pharma & Biotech
IntraBio leverages prior Niemann-Pick approval to seek FDA expansion of levacetylleucine into Ataxia-Telangiectasia
IntraBio files the first-ever FDA application for an A-T therapy. Analyse the Phase III evidence, regulatory pathway, and what approval would mean.
Pharma & Biotech
Curatis licenses corticorelin to Neupharma in CHF 83.5m Japan deal
Curatis and Neupharma sign a CHF 83.5m Japan licensing deal for corticorelin in peritumoral brain edema. Read our analysis of what the deal means.
Pharma & Biotech
United Therapeutics targets 2026 FDA submission for Tyvaso IPF indication after TETON-2 success
TETON-2 phase 3 data show nebulized treprostinil slows IPF lung decline and cuts clinical worsening risk. Analysis of what the NEJM results mean.
Pharma & Biotech
Hansa Biopharma files BLA for imlifidase in kidney transplant desensitization: What this changes for U.S. patients
Hansa Biopharma files for FDA approval of imlifidase to enable kidney transplants in highly sensitized patients. Find out what changes next.
Pharma & Biotech
How Hansa Biopharma is targeting a transplant blind spot with Imlifidase
Hansa Biopharma AB has submitted a Biologics License Application to the U.S. Food and Drug Administration for imlifidase as a desensitization treatment in highly sensitized adult patients awaiting deceased donor kidney transplants. The submission is supported by the pivotal Phase 3 ConfIdeS trial, and the company is seeking a priority review that could lead to