Updated weekly · 13 upcoming catalysts
Upcoming FDA Prescription Drug User Fee Act target action dates for US-listed biotech and pharma sponsors. Sponsor, ticker, drug, indication, regulatory pathway, and clinical context — the catalysts that drive biotech stock moves.
Indication: Pediatric type 1 and type 2 diabetes (ages 4-17)
sBLA would make Afrezza the first needle-free mealtime insulin for children; Phase 3 INHALE-1 missed HbA1c noninferiority but showed satisfaction/weight advantages.
Indication: Relapsed/refractory mantle cell lymphoma (MCL) after prior BTK inhibitor therapy
Next-gen BCL2 inhibitor with FDA Breakthrough Therapy designation; NDA backed by Phase 1/2 BGB-11417-201.
Indication: 1st-line unresectable/metastatic triple-negative breast cancer (TNBC) in patients ineligible for PD-1/PD-L1 therapy
TROPION-Breast02 showed 23.7-mo vs 18.7-mo median OS; major label expansion for TROP2 ADC franchise.
Indication: ESR1-mutated, ER+/HER2- advanced or metastatic breast cancer after prior endocrine therapy
First oral PROTAC ER degrader; VERITAC-2 showed 5.0-mo vs 2.1-mo PFS vs fulvestrant. Would validate PROTAC modality commercially.
Indication: Nicotine dependence / smoking cessation in adults
ORCA Phase 3 showed superior abstinence vs placebo; first new smoking cessation therapy in ~20 years if approved.
Indication: PET imaging of somatostatin receptor-positive neuroendocrine tumors
PDUFA extended 3 months from Mar 29 to Jun 29 for additional CMC review; not tied to safety/efficacy.
Indication: Expanded: hemophilia A/B patients 6+ with inhibitors and pediatric (6-11) without inhibitors
Expansion sBLA would make HYMPAVZI the first non-factor option for kids 6-11; FDA pushed decision from Q1 to Jun 29.
Indication: Plaque psoriasis in children ages 2-5
Would make roflumilast the first topical PDE4 inhibitor labeled for plaque psoriasis down to age 2.
Indication: AML, ALL, MDS requiring allogeneic stem cell transplant
First allogeneic cell therapy of its kind; Precision-T hit cGvHD-free survival endpoint. FDA extended PDUFA 3 months (CMC amendment).
Indication: Duchenne muscular dystrophy (DMD) cardiomyopathy
CRL-resubmission after FDA lifted prior complete response letter; HOPE-3 Phase 3 hit primary PUL v2.0 plus LVEF endpoint.
Indication: Essential thrombocythemia (ET) in adults
Label expansion from polycythemia vera into ET; SURPASS-ET showed 42.9% vs 6.0% mELN response vs anagrelide.
Indication: TKI pre-treated locally advanced or metastatic ROS1-positive NSCLC
CNS-penetrant, TRK-sparing ROS1-selective inhibitor designed to overcome G2032R resistance; ARROS-1 data-backed.
Indication: Alexander disease (AxD) in children and adults
First disease-modifying therapy for ultra-rare fatal GFAP neurodegenerative disease; pivotal met primary gait-speed endpoint.
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