Quality/GMPs

Ultragenyx published 96-week phase 3 data for GENGLYCOS, its AAV8 gene therapy for glycogen storage disease type Ia, in The Journal of Inherited Metabolic Disease, showing sustained cornstarch-intake reductions and continued glycemic control roughly six weeks after the therapy's FDA accelerated approval.

BeOne Medicines and Jazz Pharmaceuticals announced positive topline overall survival results from the second interim analysis of the Phase 3 HERIZON-GEA-01 trial, reinforcing the benefit of Ziihera-containing regimens just days after their FDA approval in first-line HER2-positive gastroesophageal adenocarcinoma.

GSK will begin a phase 3 trial in September 2026 for its investigational mRNA seasonal flu vaccine after phase II data showed stronger immune responses than licensed comparators. The candidate is designed to target both hemagglutinin and neuraminidase, a dual-antigen approach not yet tested in a late-stage mRNA flu trial.

AstraZeneca's Enhertu plus pertuzumab cut progression risk 44%, extending PFS past 3 years as the first new 1st-line HER2+ regimen in 10 years.

Novavax's partners Sanofi and Takeda have received regulatory approvals for an XFG variant-adapted formulation of Nuvaxovid, the company's protein-based, non-mRNA COVID-19 vaccine, across the US, EU, and Japan for the 2026-2027 vaccination season, positioning Novavax to continue earning royalties without maintaining its own commercial infrastructure.

PharmaEssentia's ropeginterferon alfa-2b-njft (Besremi) cut ET response rates to 43% versus 6% for anagrelide, backing a first-in-decades FDA nod for a new rare blood cancer treatment.

BioNTech has terminated the Phase 2 BNT122-01 trial of autogene cevumeran as an adjuvant monotherapy in ctDNA-positive resected colorectal cancer, following a DSMB recommendation citing a numerical imbalance in overall survival, while the drug's separate pancreatic cancer trial continues unaffected.

TSY-110 could become the first biosimilar rival to Roche's ado-trastuzumab emtansine (Kadcyla), a $2.5 billion HER2 breast cancer therapy.

The FDA has approved Lilly's tirzepatide (Mounjaro) to reduce major adverse cardiovascular events in adults with type 2 diabetes at high risk, based on the head-to-head SURPASS-CVOT trial showing non-inferiority to dulaglutide (Trulicity), making it the first GIP/GLP-1 receptor agonist with this indication.

FDA's 2025–2026 guidance changes would substantially streamline biosimilar development, including by reducing comparative efficacy and switching-study requirements. Here's what's confirmed, what industry has criticized about the existing pathway, and what's still unresolved.

The FDA has approved two zanidatamab-hrii-containing regimens for first-line treatment of HER2-positive advanced gastroesophageal adenocarcinoma, based on Phase 3 HERIZON-GEA-01 data showing a median overall survival of more than two years, marking the second FDA approval for the bispecific antibody in under two years.

The FDA has granted Rare Pediatric Disease Designation to Mahzi Therapeutics' MZ-1866, an investigational AAV9-TCF4 gene replacement therapy for Pitt Hopkins syndrome, as the Phase 1/2 UNITE study surpasses 50% enrollment.

The FDA has approved IMAAVY (nipocalimab-aahu), an FcRn-blocking antibody from Johnson & Johnson, as the first therapy specifically approved for warm autoimmune hemolytic anemia (wAIHA), based on Phase 2/3 ENERGY trial data showing durable hemoglobin response and reduced fatigue versus placebo.

The FDA has extended the PDUFA target action date for Capricor Therapeutics' deramiocel BLA from August 22 to November 22, 2026, after the company submitted 24-month HOPE-3 open-label extension data supporting a refined indication focused on upper limb function.

The FDA has accepted for priority review GSK's sBLA for Jemperli (dostarlimab) in previously untreated stage II/III dMMR/MSI-H locally advanced rectal cancer, based on AZUR-1 data, with a PDUFA date of February 2027 and eligibility for the National Priority Voucher program.

The company's first-in-class immune-boosting antibody could become the first approved treatment for a cancer with no existing options.

Regeneron Pharmaceuticals' garetosmab-grts has become the first approved therapy to shrink new bone lesions in FOP, cutting them by up to 94% as it targets the disease's root biology directly.

The FDA has approved Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy, to reduce cornstarch dependence in patients eight and older with GSDIa, the first approved treatment to target the disease's underlying genetic cause.

AbelZeta Pharma has reclaimed global rights to prizloncabtagene autoleucel from Janssen and secured FDA clearance to begin US trials, positioning the anti-CD20/CD19 bispecific CAR-T for patients who relapse after existing CAR-T therapies.

Australia's first commercial imlifidase transplant offers a path forward for patients whose antibodies had blocked a compatible kidney.

The FDA has granted Fast Track designation to C-1101, Consano Bio's investigational platelet-derived multi-protein biologic for chronic, painful lumbosacral radiculopathy, following the drug's recent IND clearance and as the company advances a Phase 1 trial evaluating the epidural injection.

The European Medicines Agency has validated the Marketing Authorization Application for PF-07307405, Pfizer and Valneva's six-valent OspA-based Lyme disease vaccine candidate, beginning formal review of what could become the first Lyme disease vaccine available in Europe.

Spevatamig's fast track status targets a cancer where 5-year survival is under 20%, pairing a novel mechanism with pembrolizumab.

China's NMPA has approved ivonescimab plus chemotherapy for first-line advanced squamous non-small cell lung cancer, based on Phase III HARMONi-6 data showing the PD-1/VEGF bispecific antibody combination reduced the risk of death by 34% compared with a PD-1 inhibitor plus chemotherapy.

Not all regulatory milestones are equal, and the differences can determine how fast a therapy reaches patients. Read this guide to 4 key FDA and EU pathways.