Hematology Weekly News - June 30th 2025

🔬🩸 Hematology Updates: Sarclisa Advances, Ancestry Insights in T-ALL, and Next-Gen Hemophilia Breakthroughs

From CHMP approvals and FDA fast tracks to durable gene therapy data and first-in-class sickle cell trials—this week’s hematology update brings forward the most pivotal developments shaping blood cancer and rare disease care.

💡 Key Highlights This Week:

✅ Sanofi’s Sarclisa receives CHMP recommendation for front-line use in transplant-eligible multiple myeloma

🧬 Major study shows genetic ancestry influences T-ALL outcomes—NOTCH1 benefits vary by population

⚡ Syndax’s Revuforj granted FDA Priority Review for relapsed/refractory mutant NPM1 AML

🧪 Schrödinger’s SGR-1505 earns Fast Track status for Waldenström’s macroglobulinemia

💊 Enterome’s EO2463 hits 60 percent complete response rate in indolent non-Hodgkin lymphoma

🔁 BeOne’s BRUKINSA tablet formulation backed by CHMP for easier use and lower environmental impact

🧫 Roche’s NXT007 achieves zero treated bleeds at highest doses in hemophilia A trial

🩺 Pfizer’s HYMPAVZI cuts bleeding by 93 percent in patients with hemophilia A or B with inhibitors

🧬 BioMarin’s ROCTAVIAN delivers five-year data showing sustained efficacy in severe hemophilia A

💊 Cellarity initiates Phase 1 trial of CLY-124, an oral globin-switching therapy for sickle cell disease with greater than 20 percent fetal hemoglobin induction and no cytotoxicity

🎯 Whether you’re tracking precision biomarkers, gene therapies, or next-gen bispecifics—this weekly roundup delivers insights you can use across hematologic R&D and strategic planning.

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