The HCPFive: Top News for Healthcare Providers from the Week of 08/31

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) extension of mitapivat’s Prescription Drug User Fee Act (PDUFA) date for its supplemental New Drug Application (sNDA) in thalassemia, an sNDA submission for roflumilast cream 0.3% (Zoryve) for plaque psoriasis in pediatric patients as young as 2 years of age, zilebesiran’s progression to a phase 3 clinical trial for uncontrolled hypertension, positive phase 1b/2a RestorAATion-2 data evaluating WVE-006 as a treatment for alpha-1 antitrypsin deficiency (AATD), and findings from the phase 3 COAST 1 trial of amlitelimab in atopic dermatitis.

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 August 31, 2025—let’s jump in!

Mitapivat PDUFA for Thalassemia Delayed to December

On September 4, 2025, the FDA extended the PDUFA date for Agios’ supplemental New Drug Application (sNDA) of mitapivat for the treatment of adult patients with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia by 3 months to December 7, 2025.

The oral pyruvate kinase (PK) activator is currently approved for treating adults with PK deficiency under the name Pyrukynd. Data supporting the sNDA submission for its use in NTD and TD alpha- or beta-thalassemia are from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T phase 3 trials.

Arcutis Submits sNDA for Roflumilast Cream 0.3% for Psoriasis in Children Aged 2-5 Years

On September 3, 2025, Arcutis Biotherapeutics submitted an sNDA to the FDA for roflumilast cream 0.3% (Zoryve) for plaque psoriasis in pediatric patients as young as 2 years of age, potentially expanding the treatment’s indication. If roflumilast cream’s sNDA clears and the age limit is expanded, it would be the first and only topical phosphodiesterase 4 (PDE4) inhibitor indicated for psoriasis among patients in this age group.

Zilebesiran, RNAi for Hypertension, Heads to Phase 3 Cardiovascular Outcomes Trial

Alnylam Pharmaceuticals announced plans to advance zilebesiran, their subcutaneously administered RNA-interfering therapeutic, into a phase 3 cardiovascular outcomes trial in patients with uncontrolled hypertension. Coined as ZENITH, the trial design is based on data from the KARDIA phase 2 program, including the KARDIA-3 trial, which missed its primary endpoint but met the objective of informing the design, patient population, and dose and was presented at the European Society of Cardiology (ESC) Congress 2025.

RestorAATion-2: WVE-006 Achieves Durable Serum AAT Protein Production in AATD

On September 3, 2025, Wave Life Sciences announced positive data from the 200 mg single and multidose and 400 mg single dose cohorts of the ongoing phase 1b/2a RestorAATion-2 study evaluating WVE-006 as a treatment for AATD. In the trial, WVE-006 achieved durable production of serum AAT protein at levels associated with a reduced risk of AATD liver and lung diseases following repeat 200 mg doses. While data from the 400 mg multidose cohort are not expected until 1Q 2026, single dose 400 mg data showed achievement of 12.8 µM total AAT and 5.3 µM M-AAT.

Phase 3 Data Show Amlitelimab Effective in Adults, Adolescents with Atopic Dermatitis

On September 4, 2025, Sanofi announced positive data from the global COAST 1 trial evaluating amlitelimab, a fully human, non–T-cell depleting monoclonal antibody directed against OX40-ligand, in patients ≥ 12 years of age with moderate-to-severe atopic dermatitis. In the trial, amlitelimab treatment administered every 4 or every 12 weeks led to significant skin clearance and reduction in severity versus placebo at week 24.