Arrowhead’s plozasiran clears CHMP: what a positive opinion for FCS means for siRNA’s regulatory arc in Europe
Plozasiran clears CHMP but the real test is reimbursement. Europe's HTA bodies will now decide whether clinical FCS diagnosis is enough.
Pharma & Biotech
Pharma & Biotech
Can Genentech’s Enspryng become the first approved treatment for MOGAD after the Phase III METEOROID win?
Genentech’s Enspryng cut MOGAD relapse risk by 68% in Phase III. Read what the data could mean for regulators, clinicians, and the rare disease market.
Pharma & Biotech
Drug Farm’s DF-003 shows early signal in ROSAH syndrome, but can ALPK1 inhibition hold up in pivotal testing?
Drug Farm’s DF-003 showed early Phase 1b benefit in ROSAH syndrome. Read what the ALPK1 data could change next.
Pharma & Biotech
Acadia Pharmaceuticals broadens Rett syndrome treatment flexibility with DAYBUE STIX rollout in the United States
Acadia Pharmaceuticals has launched DAYBUE STIX for Rett syndrome in the U.S. Read what this means for adoption, tolerability, and market strategy.
Pharma & Biotech
What Affinia Therapeutics’ UPBEAT trial reveals about the next phase of inherited heart failure gene therapy
Affinia Therapeutics has cleared AFTX-201 for a Canadian BAG3 DCM trial. Read why the gene therapy’s design and risks matter now.
Pharma & Biotech
How Soligenix is trying to turn tolerability and treatment logistics into a differentiated regulatory and commercial story
Soligenix published HyBryte versus Valchlor data in CTCL. Read what it changes for approval, adoption, and competitive positioning.
Pharma & Biotech
How Chiesi Global Rare Diseases is positioning lomitapide for earlier lifecycle management in HoFH
CHMP backs paediatric lomitapide for HoFH. Read what this means for clinicians, regulators, and rare disease access across Europe.
Pharma & Biotech
Can Drug Farm turn ALPK1 inhibition into the first real disease-modifying approach for ROSAH syndrome?
Drug Farm won FDA orphan drug designation for DF-003 in ROSAH syndrome. Read what this rare disease milestone could change next.
Pharma & Biotech
Oral Pompe therapy S-606001 moves to Phase 2 as Shionogi expands rare disease focus
Shionogi's S-606001 enters global Phase 2 as the first oral GYS1 inhibitor tested as an add-on to ERT in late-onset Pompe disease. Read the analysis.
Pharma & Biotech
GenSight Biologics begins LHON treatment under France compassionate access as REVISE Phase II moves forward
GenSight Biologics begins treating ND4-LHON patients under France's AAC compassionate access program. Analyse what REVISE and AAC mean for the regulatory pathway.