Regulatory Authority Actions

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.

The EC has approved AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan ADC for 1st-line metastatic TNBC, which showed a 5.0-month overall survival benefit over chemotherapy.

Kelun-Biotech's sac-TMT has gained NMPA acceptance for a new first-line indication application in advanced triple-negative breast cancer.

FDA has granted priority review to Johnson & Johnson's Rybrevant Faspro for head and neck cancer, backed by 42% ORR in the OrigAMI-4 trial.

Allogene's cema-cel has received FDA RMAT and fast track designations for first-line consolidation therapy in high-risk large B-cell lymphoma.

FDA staff have questioned RP1's melanoma trial data ahead of a July 30, 2026 advisory committee vote, causing Replimune shares to fall 30%.

AbelZeta has received FDA RMAT clearance for a registrational phase 2 trial of its bispecific CAR-T therapy, C-CAR168, in refractory lupus nephritis.

Scholar Rock’s apitegromab is awaiting a US manufacturing facility resolution, which may impact the therapy’s European review, for which clinical assessment has been completed.

The EC has approved AbbVie's Boey (trenibotulinumtoxinE), the first serotype E neurotoxin for glabellar lines, with faster onset than current options.