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Stay updated with the latest healthcare breakthroughs, including FDA actions and new topline clinical trial data, in this week’s essential news roundup.
Welcome to The HCPFive, your go-to roundup for the latest healthcare news and breakthroughs, curated specifically for busy healthcare professionals.
Each week, we highlight 5 key developments or headlines from healthcare that you need to know — whether it's a cutting-edge treatment, regulatory updates, or innovations shaping the future of medicine. This week's top stories include a prespecified interim analysis of the phase 3 I-CAN trial showing ravulizumab reduced proteinuria in IgA nephropathy as early as week 10, 52-week REZOLVE-AA extension data demonstrating deepening hair regrowth responses with rezpegaldesleukin in severe alopecia areata, a third consecutive positive phase 3 readout for tozorakimab in COPD from the MIRANDA trial, topline results from the phase 3 HIBISCUS trial showing etavopivat met both co-primary endpoints in sickle cell disease, and FDA accelerated approval of lunsotogene parvec-cwha (Otarmeni) as the first gene therapy for OTOF-related genetic hearing loss.
With The HCPFive, you'll get the essential takeaways to stay informed and ahead of the curve. Here's your quick dive into the top stories for the week of April 19, 2026 — let's jump in!
A prespecified interim analysis of the phase 3 I-CAN trial found that ravulizumab (Ultomiris), a long-acting C5 complement inhibitor administered intravenously every 8 weeks, met its primary endpoint of statistically significant and clinically meaningful proteinuria reduction based on 24-hour urine protein creatinine ratio at week 34, with rapid effects observed as early as week 10, in adults with IgA nephropathy at risk of progression. Alexion/AstraZeneca plans to seek accelerated approval in key markets while the trial continues to its week 106 eGFR endpoint, with full data to be presented at a forthcoming medical meeting.
New data from the blinded 16-week extension of the phase 2b REZOLVE-AA study, announced April 20, showed that 29% to 31% of patients in the low- and high-dose rezpegaldesleukin arms who had not yet reached a SALT ≤20 response by week 36 achieved new responses during the extension, with SALT ≤20 rates across the full study population reaching 25.8% and 27.6% in the low- and high-dose groups, respectively, versus 6.7% with placebo at 52 weeks. Rezpegaldesleukin, a first-in-class IL-2 pathway agonist designed to selectively expand regulatory T cells, was well tolerated through 52 weeks, with 94% of extension patients completing treatment and no discontinuations due to adverse events, supporting Nektar's plans to advance the drug into phase 3 development in severe alopecia areata.
AstraZeneca announced April 20 that tozorakimab met its primary endpoint in the phase 3 MIRANDA trial, demonstrating statistically significant and clinically meaningful reductions in moderate-to-severe COPD exacerbations compared with placebo in former smokers and in the overall population — including current smokers across all eosinophil levels and lung function severity stages — with patients receiving 300 mg every 2 weeks rather than the 4-week interval used in OBERON and TITANIA. The result marks the third consecutive positive phase 3 readout in the LUNA program, further establishing tozorakimab as a potential first-in-class IL-33–targeting biologic for COPD independent of eosinophil-driven disease subtypes; full numeric data remain pending presentation at a medical meeting.
Topline results from the phase 3 HIBISCUS trial, announced April 20, showed that once-daily oral etavopivat 400 mg — an investigational pyruvate kinase-R activator — reduced vaso-occlusive crisis events by 27% and delayed time to first crisis to 38.4 weeks versus 20.9 weeks on placebo, while 48.7% of treated patients achieved a hemoglobin increase of more than 1 g/dL at week 24 compared with 7.2% on placebo. Etavopivat, which holds Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the US, met both co-primary endpoints in 385 patients aged 12 years and older with sickle cell disease on background standard of care, and Novo Nordisk plans to file for initial regulatory approval in the second half of 2026.
The FDA granted accelerated approval on April 23 to lunsotogene parvec-cwha (Otarmeni; Regeneron), the first gene therapy for OTOF-related hearing loss and the first approved gene therapy to restore a neurosensory function, based on phase 1/2 CHORD trial data in which 80% of 20 evaluable pediatric patients achieved a primary hearing threshold endpoint at 24 weeks, with 42% reaching normal hearing thresholds at 48 weeks. The single-dose AAV vector therapy is delivered via intracochlear infusion under general anesthesia and indicated for patients with molecularly confirmed biallelic OTOF variants, preserved outer hair cell function, and no prior cochlear implant in the treated ear; Regeneron announced the drug will be provided at no cost to eligible US patients, though procedure-related costs may still apply.
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