FDA Eases Biosimilar Rules to Cut Drug Costs
Summary:
The FDA has announced new guidance to speed up biosimilar approvals by removing the requirement for human trials comparing biosimilars with their branded biologic counterparts. The agency estimates this change could save developers up to $100 million per product and bring lower-cost biologics to patients faster. The policy reflects a major step toward increasing competition in the biologics market, where biosimilars currently hold less than 20% market share due to high development costs. Industry groups hailed the move as a “watershed moment” for improving access and affordability in U.S. healthcare.
HHS Doubles AI Funding to Accelerate Childhood Cancer Research
Summary:
The U.S. Department of Health and Human Services (HHS) has announced a major boost in pediatric cancer research funding – doubling support for the Childhood Cancer Data Initiative (CCDI) at the NIH from $50 million to $100 million. The expansion follows a new Executive Order, “Unlocking Cures for Pediatric Cancer with Artificial Intelligence,” signed by President Trump. The initiative aims to harness AI and large-scale health data to advance diagnostics, treatments, and prevention strategies for childhood cancers. HHS, NIH, and NCI leaders emphasized that this effort will use cutting-edge AI to uncover causes, predict risks earlier, and improve outcomes for young patients.
Pediatric cancer remains the leading disease-related cause of death among U.S. children, with incidence rising over 40% since 1975 – making this funding surge a significant step toward accelerating cures through data-driven science.
Intellia Halts Phase 3 CRISPR Trials Over Liver Safety Concern
Summary:
Intellia Therapeutics has paused dosing and screening in two phase 3 trials of its CRISPR therapy nex-z after a patient in the ATTR-cardiomyopathy study was hospitalized for severe liver toxicity. The event triggered regulatory stopping criteria, leading to a temporary halt as investigators assess the cause. The setback sent Intellia shares down 44%, delaying progress toward potential competition with Alnylam, BridgeBio, and Pfizer in the ATTR market.
BridgeBio Scores Phase 3 Win in Rare Calcium Disorder
Summary:
BridgeBio Pharma’s drug encaleret achieved strong phase 3 results in patients with autosomal dominant hypocalcemia type 1 (ADH1), normalizing calcium levels in 76% of patients vs. just 4% on standard care. The company plans to file for FDA approval in 2026, with additional trials for chronic and pediatric hypoparathyroidism next year. Analysts project $1B peak sales and call the result a “clear win” that cements BridgeBio’s path as a multi-product biotech.
Merck’s Welireg Succeeds in Two Major Kidney Cancer Trials
Summary:
Merck reported phase 3 wins for its kidney cancer drug Welireg, showing it improved outcomes both after surgery (when paired with Keytruda) and in relapsed patients (with Lenvima). The results could significantly expand Welireg’s use and generate over $6B in potential revenue, strengthening Merck’s portfolio ahead of Keytruda’s 2028 patent loss.
