Disease

Regulatory Science

Chiesi wins EC approval for lomitapide in children with HoFH

The European Commission has expanded lomitapide's indication to children aged five and older with homozygous familial hypercholesterolaemia, backed by Phase 3 data.
The European Commission has expanded lomitapide's indication to children aged five and older with homozygous familial hypercholesterolaemia, backed by Phase 3 data.
Diagnostics

Cloudbreak Pharma completes final visits for Phase 3 pterygium trial

Topline 12-month efficacy data for CBT-001, a potential first FDA-approved pterygium drug, are expected in Q3 2026.
Topline 12-month efficacy data for CBT-001, a potential first FDA-approved pterygium drug, are expected in Q3 2026.
Regulatory Science

BioCardia wins FDA backing for single-trial PMA path for CardiAMP

FDA confirmed that BioCardia's ongoing CardiAMP HF II trial may alone support a Premarket Approval application for its autologous cell therapy in ischaemic HFrEF.
FDA confirmed that BioCardia's ongoing CardiAMP HF II trial may alone support a Premarket Approval application for its autologous cell therapy in ischaemic HFrEF.

Fractyl Health: Revita holds 78% of GLP-1 weight loss at one year

One-year REVEAL-1 open-label data show a single endoscopic Revita procedure sustains most GLP-1-induced weight loss after drug discontinuation.
One-year REVEAL-1 open-label data show a single endoscopic Revita procedure sustains most GLP-1-induced weight loss after drug discontinuation.
Biotech

Hengrui and Kailera report ribupatide Phase 2 oral data at ADA 2026

Oral ribupatide delivered up to 12.1% mean weight loss at 26 weeks in a China Phase 2 trial, with no observed plateau and a
Oral ribupatide delivered up to 12.1% mean weight loss at 26 weeks in a China Phase 2 trial, with no observed plateau and a
Regulatory Science

Memo Therapeutics presents Phase II data on potravitug for BKPyV

Propensity-score-matched analysis showed potravitug nearly doubled viral clearance rates vs controls in kidney transplant recipients with BK polyomavirus infection.
Propensity-score-matched analysis showed potravitug nearly doubled viral clearance rates vs controls in kidney transplant recipients with BK polyomavirus infection.
Biotech

Zealand Pharma petrelintide hits 10.7% weight loss at 42 weeks

Phase 2 ZUPREME-1 data show the amylin analogue achieved double-digit weight reduction with near-placebo tolerability, ahead of planned Phase 3 initiation.
Phase 2 ZUPREME-1 data show the amylin analogue achieved double-digit weight reduction with near-placebo tolerability, ahead of planned Phase 3 initiation.
Regulatory Science

InnoCare orelabrutinib hits Phase IIb endpoints in SLE study

Orelabrutinib met primary and secondary endpoints in a 187-patient Phase IIb SLE trial, with 57% SRI-4 response versus 34% for placebo at week
Orelabrutinib met primary and secondary endpoints in a 187-patient Phase IIb SLE trial, with 57% SRI-4 response versus 34% for placebo at week
Diagnostics
Biotech

ACTG launches Phase 2 trial of dasatinib to shrink HIV reservoir

The global trials network has opened study A5413, testing whether the repurposed cancer drug dasatinib can reduce latent HIV by blocking CD4 cell proliferation.
The global trials network has opened study A5413, testing whether the repurposed cancer drug dasatinib can reduce latent HIV by blocking CD4 cell proliferation.
Regulatory Science

Attralus wins FDA orphan drug status for AL amyloidosis candidate AT-02

Zamubafusp alfa now holds four orphan designations globally as Attralus advances the pan-amyloid removal antibody through a Phase 2 trial.
Zamubafusp alfa now holds four orphan designations globally as Attralus advances the pan-amyloid removal antibody through a Phase 2 trial.
Genomics

Biogen's salanersen wins FDA Breakthrough Therapy tag for SMA

The designation is backed by Phase 1b data showing motor function gains in children who had a suboptimal response to prior gene therapy.
The designation is backed by Phase 1b data showing motor function gains in children who had a suboptimal response to prior gene therapy.
Pharmaceuticals
Regulatory Science

GH Research publishes Phase 2a PPD data for inhaled GH001

All ten patients in GH Research's open-label trial achieved remission within two hours of a single inhaled dose of mebufotenin, the company reports.
All ten patients in GH Research's open-label trial achieved remission within two hours of a single inhaled dose of mebufotenin, the company reports.
Regulatory Science

Gilead and Lakefront close Ouro Medicines deal for $1.675bn

The acquisition adds gamgertamig, a BCMAxCD3 T cell engager, to Gilead's inflammation portfolio; registrational studies are expected as early as 2027.
The acquisition adds gamgertamig, a BCMAxCD3 T cell engager, to Gilead's inflammation portfolio; registrational studies are expected as early as 2027.
Regulatory Science

Novartis ALIGN data show Vanrafia slows IgAN kidney decline by 34%

Final 2.5-year Phase III results, published in The Lancet, back a traditional approval filing for atrasentan in IgA nephropathy later this year.
Final 2.5-year Phase III results, published in The Lancet, back a traditional approval filing for atrasentan in IgA nephropathy later this year.
Diagnostics

Spectral Medical PMX shows sustained mortality benefit at 12 months

Tigris Phase 3 data show PMX reduced 12-month mortality to 52.8% versus 66.7% for standard care, with a 95.9% Bayesian probability
Tigris Phase 3 data show PMX reduced 12-month mortality to 52.8% versus 66.7% for standard care, with a 95.9% Bayesian probability
Regulatory Science

Abivax Phase 3 data show obefazimod hits all UC endpoints

Both doses of Abivax's oral miR-124 enhancer achieved placebo-adjusted remission rates above 39% at 44 weeks, with an NDA filing planned for
Both doses of Abivax's oral miR-124 enhancer achieved placebo-adjusted remission rates above 39% at 44 weeks, with an NDA filing planned for
Biotech

JJP Biologics: nebaprubart shows early efficacy in rare LAD trial

Interim Phase 1b data show rapid blister reduction and dapsone tapering in Linear IgA Disease, supporting a rheumatoid arthritis trial in Q3 2026.
Interim Phase 1b data show rapid blister reduction and dapsone tapering in Linear IgA Disease, supporting a rheumatoid arthritis trial in Q3 2026.
Regulatory Science

J&J nipocalimab meets Phasea 2 primary endpoint in lupus study

Phase 2 JASMINE data show nipocalimab reduced SLE disease activity through 52 weeks, with stronger responses in autoantibody-positive patients.
Phase 2 JASMINE data show nipocalimab reduced SLE disease activity through 52 weeks, with stronger responses in autoantibody-positive patients.
Genomics

Ziftomenib triplet shows 87% ORR in venetoclax-naive R/R AML

Blood publication of KOMET-007 data shows deep, durable responses for ziftomenib plus venetoclax/azacitidine in NPM1-mutated relapsed/refractory AML.
Blood publication of KOMET-007 data shows deep, durable responses for ziftomenib plus venetoclax/azacitidine in NPM1-mutated relapsed/refractory AML.
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