The HCPFive: Top News for Healthcare Providers from the Week of 04/27

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 the US Food and Drug Administration’s (FDA) historic approval of a gene therapy for a rare skin disorder, new treatment options for giant cell arteritis and generalized myasthenia gravis, promising results for semaglutide in MASH, and a global study linking adolescent blood sugar levels to heart risk.

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 27, 2025—let’s jump in!

Interested in oncology news? Check out The OncFive, from our sister publication OncLive.

FDA Approves Pz-cel (Zevaskyn) Gene Therapy for RDEB

On April 29, 2025, the FDA approved Abeona Therapeutics Inc.’s prademagene zamikeracel (pz-cel; Zevaskyn) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Based on positive safety and efficacy data from the pivotal Phase 3 VIITAL study, Pz-cel marked the first autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with the rare, debilitating skin disease.

FDA Approves Upadacitinib, Expanding Treatment for Adults With Giant Cell Arteritis

On April 29, 2025, the FDA approved AbbVie’s once-daily upadacitinib (RINVOQ) 15 mg for the treatment of giant cell arteritis (GCA), offering the potential to taper off steroids and achieve sustained remission. The decision was supported by the results of the pivotal Phase 3 SELECT-GCA clinical trial—46.4% of patients receiving upadacitinib achieved sustained remission from week 12 to week 52, compared with 29.0% of patients receiving placebo.

FDA Approves Nipocalimab Generalized Myasthenia Gravis for Adults, Children

On April 30, 2025, the FDA approved Johnson & Johnson’s nipocalimab-aahu (IMAAVY™) to treat generalized myasthenia gravis (gMG) in adults and children >12 years who are anti-acetylcholine receptor (AChR) or anti-muscular-specific kinase (MuSK) antibody positive.

The approval of this human FcRn blocking monoclonal antibody marked the second agent on the market for both common subtypes of gMG, following the 2023 approval of rozanolixizumab (Rystiggo; UCB) in adults.

Semaglutide Improves Steatohepatitis, Fibrosis in Phase 3 MASH Trial

Results from part 1 of the phase 3 Effect of Semaglutide in Subjects with Non-cirrhotic Non-alcoholic Steatohepatitis (ESSENCE) trial revealed the safety and efficacy of semaglutide for the treatment of patients with metabolic dysfunction–associated steatohepatitis (MASH) and stage 2/3 fibrosis. Published in The New England Journal of Medicine, once-weekly semaglutide 2.4 mg outperformed placebo for resolution of steatohepatitis without worsening of fibrosis and reduction in liver fibrosis without worsening of steatohepatitis.

Related Content: Diabetes Dialogue: Semaglutide for MASH in ESSENCE Trial, With Arun Sanyal, MD

Increased Fasting Blood Glucose Triples Risk of Heart Damage in Adolescents

A recent collaborative study between the US, Switzerland, Australia, Finland, and the UK found that high blood sugar and insulin resistance substantially increase the risk of heart failure in adolescents, particularly females. With follow-up twice occurring between ages 17 and 24, a three-to-five-fold increase was identified in blood glucose and insulin resistance, predicting a matching increase in heart disease risk.