The HCPFive: Top News for Healthcare Providers from the Week of 01/04

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 (FDA) approvals of caplacizumab-yhdp (Cablivi) for pediatric patients ≥ 12 years of age with acquired thrombotic thrombocytopenic purpura (aTTP) and Fibrinogen (Human) Lyophilized Powder for Reconstitution (Fibryga) for fibrinogen replacement in patients with acquired fibrinogen deficiency, Priority Review designations for a BLA atacicept for the treatment of adults with IgA nephropathy (IgAN) and an sBLA for teplizumab-mzwv to expand the age indication from ≥8 years to ≥1 year, aiming to delay the onset of stage 3 type 1 diabetes, and Breakthrough Therapy Designation for pemvidutide for the treatment of metabolic dysfunction-associated steatotic liver disease (MASH).

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 January 4, 2025—let’s jump in!

FDA Approves Caplacizumab-yhdp (Cablivi) as First Pediatric aTTP Therapy

On January 5, 2026, the FDA approved Sanofi’s caplacizumab-yhdp (Cablivi) for injection to treat pediatric patients ≥ 12 years of age with aTTP in combination with plasma exchange and immunosuppressive therapy. The decision builds upon caplacizumab’s initial 2019 approval for adults with aTTP and was supported by efficacy data from a retrospective chart review study of 30 patients 2-18 years of age showing 80% of patients achieved clinical remission. Of note, the approval made caplacizumab the first treatment for pediatric aTTP.

Pemvidutide Gets Breakthrough Therapy Designation for MASH

On January 5, 2026, the FDA granted Breakthrough Therapy Designation (BTD) to Altimmune’s pemvidutide for the treatment of MASH based on 24-week data from the phase 2b IMPACT trial demonstrating statistically significant MASH resolution without worsening of fibrosis and early and substantial improvements in liver fat and non-invasive tests (NITs) of fibrosis and hepatic inflammation.

Altimmune has aligned with the FDA on the parameters for a planned registrational phase 3 trial of pemvidutide for MASH patients with moderate to advanced liver fibrosis evaluating multiple pemvidutide doses over a 52-week treatment period and incorporating biopsy-based endpoints to support a potential accelerated approval and the use of AIM-MASH AI Assist, the first AI pathology tool qualified by the FDA for use in MASH clinical trials.

FDA Grants Priority Review Of Biologics License Application For Atacicept In IgA Nephropathy

On January 7, 2026, the FDA granted priority review to Vera Therapeutics’ Biologics License Application (BLA) for atacicept for the treatment of adults with IgAN. The BLA submission was supported by data from the phase 3 ORIGIN 3 trial’s prespecified interim analysis showing the trial met its primary endpoint for reduction in proteinuria at week 36, with a 46% reduction compared to baseline and a 42% reduction compared to placebo (P <.0001).

FDA Approves 2-Gram Presentation of Fibryga for Acquired Fibrinogen Deficiency

On January 7, 2026, the FDA approved a 2-gram presentation of Octapharma’s Fibrinogen (Human) Lyophilized Powder for Reconstitution (Fibryga) for fibrinogen replacement in patients with acquired fibrinogen deficiency. Initially approved as a 1-gram single-dose bottle in 2024, Fibryga is the first and only virus-inactivated, human plasma-derived fibrinogen concentrate approved for AFD in the US. The updated kit also contains 100 mL of Water for Infusion and continues to feature the nextaro reconstitution device.

FDA Accepts for Priority Review sBLA for Teplizumab for Children With Stage 2 T1D

On January 5, 2026, the FDA accepted for priority review the supplemental biologics license application (sBLA) for Sanofi’s teplizumab-mzwv to expand the age indication from ≥8 years to ≥1 year, aiming to delay the onset of stage 3 type 1 diabetes. The sBLA submission was based on positive interim one-year data from the ongoing PETITE-T1D phase 4 study, a 2 year single-arm, open-label, multicenter study of children <8 years with stage 2 T1D.