The HCPFive: Top News for Healthcare Providers from the Week of 12/07

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 approvals of omidubicel-onlv (Omisirge) for the treatment of severe aplastic anemia, etuvetidigene autotemcel (Waskyra) for Wiskott-Aldrich syndrome, gepotidacin (Blujepa) for patients with uncomplicated urogenital gonorrhea, trofinetide (Daybue Stix) for oral solution for Rett syndrome, and berotralstat (Orladeyo) oral granules for patients with hereditary angioedema (HAE).

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 December 7, 2025—let’s jump in!

FDA Approves Omidubicel-onlv (Omisirge) As First Cellular Severe Aplastic Anemia Therapy

On December 8, 2025, the FDA approved Ayrmid’s omidubicel-onlv (Omisirge) for the treatment of severe aplastic anemia, making it the first hematopoietic stem cell transplant therapy for this indication. The treatment addresses the limitations of umbilical cord blood as a source including delayed hematopoietic recovery and increased infections and provides additional graft options for patients with severe aplastic anemia who need hematopoietic stem cell transplant.

FDA Approves Etuvetidigene Autotemcel (Waskyra) For Wiskott-Aldrich Syndrome

On December 9, 2025, the FDA approved etuvetidigene autotemcel (Waskyra), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome. It is indicated for pediatric patients ≥ 6 months of age and adults with Wiskott-Aldrich syndrome who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation is appropriate and no suitable human leukocyte antigen-matched related stem cell donor is available.

FDA Approves Gepotidacin as Oral Treatment of Uncomplicated Urogential Gonorrhea

On December 11, 2025, the FDA approved GSK’s gepotidacin as an oral option for adult and pediatric patients ≥ 12 years of age weighing ≥ 45 kg who have limited or no alternative options for the treatment of uncomplicated urogenital gonorrhoea caused by susceptible strains of Neisseria gonorrhoeae. The decision follows the FDA approval of gepotidacin earlier this year as an oral treatment for female adult and paediatric patients ≥ 12 years of age weighing ≥40 kg with uncomplicated urinary tract infection.

FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome

On December 12, 2025, the FDA approved Acadia Pharmaceuticals’ trofinetide (Daybue Stix) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients ≥ 2 years of age. The new formulation is expected to deliver the same efficacy and safety profile of trofinetide oral solution, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their trofinetide treatment.

FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE

On December 12, 2025, the FDA approved BioCryst’s berotralstat (Orladeyo) oral granules for patients with HAE aged 2 – 11 years. The decision expands upon berotralstat’s 2020 FDA approval for the prevention of HAE attacks in adults and children aged ≥ 12 years.

References

1. Brooks A. FDA Approves Omidubicel-onlv (Omisirge) As First Cellular Severe Aplastic Anemia Therapy. HCPLive. December 8, 2025. Accessed December 12, 2025. https://www.hcplive.com/view/fda-approves-omidubicel-onlv-omisirge-first-cellular-severe-aplastic-anemia-therapy

2. Brooks A. FDA Approves Etuvetidigene Autotemcel (Waskyra) For Wiskott-Aldrich Syndrome. HCPLive. December 9, 2025. Accessed December 12, 2025. https://www.hcplive.com/view/fda-approves-etuvetidigene-autotemcel-waskyra-wiskott-aldrich-syndrome

3. Smith T. FDA Approves Gepotidacin as Oral Treatment of Uncomplicated Urogential Gonorrhea. HCPLive. December 11, 2025. Accessed December 12, 2025. https://www.hcplive.com/view/fda-approves-gepotidacin-oral-treatment-uncomplicated-urogential-gonorrhea

4. Brooks A. FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome. HCPLive. December 12, 2025. Accessed December 12, 2025. https://www.hcplive.com/view/fda-approves-new-powder-formulation-of-trofinetide-daybue-for-rett-syndrome

5. Derman C. FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE. HCPLive. December 12, 2025. Accessed December 12, 2025. https://www.hcplive.com/view/fda-approves-oral-berotralstat-children-aged-2-11-years-hae