Regulatory Authority Actions

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.

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.

Varsetatug masetecan (Varseta-M) posted a 32% response rate in colorectal cancer, backing its new FDA fast track designation.

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.

Vanda's single-dose antibody was shown to clear severe pustular flares within weeks and is now recognized as a priority treatment across 3 continents.

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.

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

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.

Zamubafusp alfa's FDA fast track status highlights the therapy's ability to remove toxic amyloid already damaging the heart and kidneys in AL amyloidosis patients.

Early Parkinson patients could gain a first disease-modifying option as ACI-7104 clears a major FDA hurdle after strong interim results.

BI-1808 plus pembrolizumab posted a 24% response rate in platinum-resistant ovarian cancer, tripling pembrolizumab's historical rate alone.

Melanoma patients who failed anti-PD-1 therapy gain a new option after Replimune's oncolytic virus clears FDA on its third try.

The European Commission has approved an expanded indication for Bavarian Nordic's IMVANEX (MVA-BN) mpox and smallpox vaccine, extending eligibility down to children 2 to less than 12 years old, based on phase 2 data showing an immune response and safety profile comparable to adults.

Backed by an 84.3% drop in RSV hospitalizations, Enflonsia could extend protection to high-risk infants still vulnerable in their second season.

The FDA's approval of mFLUSIVA (mRNA-1010) extends Moderna's lipid nanoparticle platform to a new indication, with broad implications for biopharmaceutical manufacturing flexibility and regulatory precedent.

Nonhealing wounds precede 80% of diabetes-related amputations, and BIOxHEAL is now cleared to test whether it can change that in a controlled trial.

FDA has granted fast track designation to Aravax's PVX108, a peptide immunotherapy for peanut allergy, ahead of phase 2 data.