BioMarin seeks FDA approval for Voxzogo in hypochondroplasia after Phase 3 success
BioMarin presents Voxzogo skull growth research and Phase 3 hypochondroplasia data. Examine clinical evidence, FDA reviews and 2027 prospects.
Pharma & Biotech
Pharma & Biotech
AviadoBio AVB-101 shows 90% progranulin increase in Phase 1/2 ASPIRE-FTD trial
AviadoBio has reported dose-related increases in cerebrospinal fluid progranulin, including a 90% rise in its highest-dose cohort, alongside encouraging early safety and neurodegeneration biomarker findings. The 17-patient experience supports continued investigation of AVB-101, but evidence that the gene therapy slows dementia progression is still lacking.
Pharma & Biotech
Celltrion presents LIBERTY-CD and LIBERTY-UC post hoc findings at ACG 2026
Celltrion's Zymfentra shows 71.4% Crohn's remission in a matched analysis, with new findings on treatment interruption and antibodies.
Pharma & Biotech
Sebela Pharmaceuticals reports positive ACG 2026 data for tegoprazan in reflux disease
Sebela's tegoprazan improves heartburn and regurgitation in Phase 3 NERD data. Examine efficacy, FDA review and competition.
Pharma & Biotech
Skyrizi reaches 73.1% remission in a Crohn’s subgroup. Can AbbVie secure a new FDA dosing option?
AbbVie reports new Skyrizi and Rinvoq Crohn's disease data, including Phase III remission results and long-term findings. Examine the evidence.
Pharma & Biotech
Mirum’s EXPAND study tests maralixibat for treatment-resistant cholestatic pruritus
Mirum will reveal LIVMARLI EXPAND Phase 3 results on October 12, testing whether maralixibat can treat itching in additional rare liver diseases.
Pharma & Biotech
One ABCL635 injection cut hot flashes by 83%. Can AbCellera prove the benefit lasts?
AbCellera will reveal new ABCL635 safety and symptom data after reporting an 83% hot flash reduction. Can its antibody sustain the benefit?
Pharma & Biotech
Can Spruce Biosciences prove its Sanfilippo treatment slows brain decline? New data could strengthen its FDA case
Spruce Biosciences prepares MPS IIIB disease progression data as TA-ERT approaches its FDA filing and confirmatory trial milestones.
Pharma & Biotech
Why LEO Pharma is investing in real-world evidence for difficult dermatological diseases
LEO Pharma presents 21 dermatology posters covering Anzupgo, Spevigo and Adbry. Examine adolescent FDA review, clinical data and growth prospects.
Pharma & Biotech
Can ME Therapeutics’ CD19 CD22 CAR designs address resistance in B-cell cancers?
ME Therapeutics extends its CD22 nanobody option to February 2027. Examine dual-target CAR development, licensing and clinical challenges.