FDA flurry: approvals & alerts
Several regulatory moves landed this week — Denali scored its first FDA approval for a pediatric genetic therapy, Narsoplimab‑wuug won approval for transplant‑associated thrombotic microangiopathy, and the FDA issued an early alert over tubing resin changes in certain B. Braun dialysis bloodlines. Those concurrent approvals and safety notices show how fast clinical wins and compliance risks can reshape medtech commercial plans and procurement conversations. (whtc.com) (pharmacytimes.com) (aha.org)
Denali’s AVLAYAH (tividenofusp alfa‑eknm) received accelerated FDA approval on March 25, 2026 for the neurologic manifestations of Hunter syndrome (MPS II). (finance.yahoo.com) The label limits use to pediatric patients started while presymptomatic or symptomatic and weighing at least 5 kg before advanced neurologic impairment, and the approval was granted on the basis of reduced cerebrospinal‑fluid heparan sulfate (CSF HS) as a surrogate endpoint with continued approval contingent on a confirmatory trial. (healio.com) The FDA awarded Denali a Rare Pediatric Disease Priority Review Voucher with the approval, and company management discussed the decision and commercial plans on a March 25 conference call where they flagged the PRV as a transferable asset the company may monetize. (marketbeat.com) Omeros’ YARTEMLEA (narsoplimab‑wuug) won FDA approval on Dec. 24, 2025 for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in adults and children aged 2 years and older. (businesswire.com) The agency’s decision for narsoplimab was supported by single‑arm clinical data plus expanded‑access experience (28 evaluable trial patients plus 19 from an expanded access program were cited by FDA reviewers), making YARTEMLEA the first approved lectin‑pathway (MASP‑2) inhibitor for TA‑TMA. (onclive.com) The FDA issued an Early Alert on March 25, 2026 for certain B. Braun Streamline Airless System and B3 Low Volume hemodialysis bloodlines after preliminary testing found a tubing‑resin change caused small air bubbles to adhere inside the arterial bloodline segment between the patient connector and the blood pump. (aha.org) B. Braun has sent an Urgent Medical Device Correction recommending those affected bloodline sets be used only if no alternative is available, and the FDA’s MedWatch notice urged customers to follow the firm’s corrective actions while evaluations continue. (content.govdelivery.com)
