Drug Development
51 articles RSS
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.
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.
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.
AXS-05 Reaches FDA Decision Deadline With April 30 PDUFA Date, Poised to Become Second-Ever Approved Drug for Alzheimer's Agitation
Axsome Therapeutics' AXS-05 faces its FDA verdict on April 30 for Alzheimer's disease agitation, backed by four Phase 3 trials but complicated by one failed study.
Dana-Farber's CAR-PRISM Trial Achieves 100% MRD-Negativity in High-Risk Smoldering Myeloma, Pushing CAR-T Into Pre-Cancer Territory
A single infusion of Johnson & Johnson's Carvykti rendered all 20 patients in a Dana-Farber phase 2 trial undetectable for residual disease, the first time CAR-T therapy has been tested in a precursor cancer setting.
FDA Clears Denali's AVLAYAH, the First Brain-Penetrating Enzyme Therapy, Cracking Hunter Syndrome's Neurologic Frontier After Two Decades
Denali's tividenofusp alfa becomes the first FDA-approved biologic engineered to cross the blood-brain barrier, breaking a long rare-disease rejection streak and validating a transport-vehicle platform that could unlock CNS access for many more drugs.
Xenon's Azetukalner Cuts Seizure Frequency by 53% in Phase 3 Trial, Poised for First-in-Class FDA Submission
Phase 3 data presented at AAN 2026 show azetukalner nearly halved seizure frequency in treatment-resistant focal epilepsy, with 38% of long-term patients achieving over a year of seizure freedom.