Biotech & Medicine
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Regenxbio's RGX-202 Hits Primary Endpoint in Pivotal Duchenne Trial, Clearing Path to a 2027 Accelerated-Approval Filing
Regenxbio's gene therapy RGX-202 met the primary endpoint of its pivotal Phase III AFFINITY DUCHENNE trial, with 93% of participants reaching the microdystrophin expression threshold.
AstraZeneca's Eneboparatide Frees 31% of Hypoparathyroidism Patients From Calcium and Vitamin D at Week 24 in CALYPSO Phase III
AstraZeneca's CALYPSO Phase III hit its composite primary endpoint with 31.1% of eneboparatide patients normalizing serum calcium and dropping supplements at week 24 versus 5.9% on placebo, with benefits sustained through a 52-week extension.
FDA Expands argenx's VYVGART to All Adult Myasthenia Gravis Serotypes, Reaching Seronegative Patients for the First Time
The May 8 sBLA approval extends efgartigimod's label to MuSK, LRP4 and triple-seronegative gMG patients after the 119-patient ADAPT SERON trial hit its MG-ADL endpoint with a 3.35-point improvement at week 4 (p=0.0068).
DECISION Trial Misses Primary Endpoint as Pooled Meta-Analysis of 9,013 Patients Finds Digitalis Cuts Heart Failure Events 15 Percent
Low-dose digoxin failed its primary endpoint in 1,001 Dutch heart-failure patients, but a JAMA meta-analysis of DECISION, DIGIT-HF and the 1997 DIG trial published the same day finds digitalis cuts the combined risk of cardiovascular death or first worsening event by 15 percent.
CRISPR Therapeutics and Vertex post $43 million CASGEVY revenue in Q1 2026 as pediatric submission lands with FDA Priority Voucher
First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.
Takeda's TAK-881 Matches HYQVIA in Pivotal Phase 2/3 Trial While Halving the Infusion Volume for Primary Immunodeficiency Patients
A 20% subcutaneous immunoglobulin paired with hyaluronidase delivered the same antibody exposure as HYQVIA in half the volume, clearing Takeda's path to global filings in fiscal 2026.
FDA Approves Veppanu From Arvinas and Pfizer, the First Protein-Degrader Drug Cleared for Any Cancer
Vepdegestrant becomes the first PROTAC ever approved by the FDA, treating ESR1-mutated advanced breast cancer after one prior endocrine therapy.
Chiesi to Buy KalVista for $1.9 Billion in Its Biggest-Ever Deal, Wrapping the First Oral HAE Pill Into a Growing Rare Disease Portfolio
Italian drugmaker Chiesi will pay $27 a share for KalVista Pharmaceuticals to acquire Ekterly, the first oral on-demand treatment for hereditary angioedema, in a deal expected to close in the third quarter.
UK Clinical Trial Reforms Take Effect, Cutting Set-Up Times to 122 Days and Making Trial Registration a Legal Requirement for the First Time
The MHRA and HRA's largest regulatory overhaul in 20 years went live April 28, introducing notifiable trials, a 14-day automatic-approval modification route, and mandatory transparency for UK studies.
FDA Launches Real-Time Clinical Trials Pilot With AstraZeneca and Amgen, Aiming to End Years-Long Reporting Lags
The FDA announced two proof-of-concept real-time cancer trials and opened public comment on a broader pilot program, betting that cloud and AI-mediated data flow can compress drug review timelines.
Lilly Bets Up to $2.25 Billion on Profluent's AI-Designed Recombinases, Pushing Gene Editing Beyond CRISPR Into Kilobase-Scale Territory
Eli Lilly's deal with Bezos-backed Profluent funds AI-designed recombinases that aim to insert entire genes, going beyond CRISPR's localized cuts.
FDA Awards National Priority Vouchers to Three Psychedelic Programs, Compressing Review Times to One to Two Months
Compass Pathways, Usona Institute, and Otsuka-acquired Transcend Therapeutics have been selected for FDA's Commissioner's National Priority Voucher program for psilocybin and methylone, six days after President Trump's executive order on serious mental illness.