1) FDA Approves DAYBUE® STIX for Rett Syndrome (12 Dec 2025)
What’s new: The U.S. FDA has given the green light to DAYBUE® STIX (trofinetide) for its oral solution—this is a novel powder formulation of the first-in-class drug for the treatment of Rett syndrome.
Why it matters: It opens up a more flexible treatment option for the patients and the caregivers while safety and efficacy are preserved.
Source: Acadia Pharmaceuticals press release via Business Wire (FDA approval announcement, December 12, 2025). Business Wire
2) FDA Approves First Cellular Therapy for Severe Aplastic Anemia (8 Dec 2025)
What’s new: As per the FDA's press announcement calendar, the FDA on December 8, 2025, cleared the first cellular therapy for severe aplastic anemia, a significant step forward in the treatment of rare hematologic diseases.
Why it matters: Patients with aplastic anemia now have access to a new innovative treatment option with a novel cell-based approach.
Source: FDA press announcements listing (December 8, 2025). U.S. Food and Drug Administration
3) First Antibiotic Approved Under FDA’s CNPV Pilot (9 Dec 2025)
What’s new: An antibiotic consisting of amoxicillin and clavulanate potassium has received the FDA's Commissioner’s National Priority Voucher (CNPV) pilot program's first drug approval, providing a solution for long-standing shortages in critical antibiotic therapies.
Why it matters: This move takes a significant step towards developing a prioritized regulatory pathway to ensure that antibiotics are available domestically.
Source: PharmTech report on FDA CNPV pilot approval (today’s article, Dec 2025). Pharmaceutical Technology
4) Hikma Launches TYZAVAN™ Vancomycin Injection in the U.S. (8 Dec 2025)
What’s new: Hikma Pharmaceuticals has made an announcement regarding the U.S. launch of TYZAVAN™ (vancomycin injection, USP), which is a premixed formulation ready to use and that is intended to make treatment for serious bacterial infections easier.
Why it matters: The new vancomycin injection is the first in the U.S. market to be room-temperature stable, improving hospital readiness and speeding up patient care.
Source: Hikma press release (December 8, 2025). Hikma
5) Mirum Pharmaceuticals to Acquire Bluejay Therapeutics (8 Dec 2025)
What’s new: Mirum Pharmaceuticals has signed a definitive deal with Bluejay Therapeutics, which includes the acquisition of brelovitug, a drug in its last stage of the monoclonal antibodies for chronic hepatitis delta virus (HDV)—a rare liver infection that needs a lot of medical help.
Why it matters: Mirum gets a possible breakthrough rare-disease product added to the pipeline, and they will get the registrational readouts in 18 months at the latest.
Source: Company press release (December 8, 2025). Bluejay Therapeutics
6) EU Reaches Agreement to Overhaul Pharmaceutical Legislation (11 Dec 2025)
What’s new: The European Medicines Agency (EMA) was all for a political agreement which would majorly update EU’s pharmaceutical legislation and the whole process would lead to modernizing the regulatory frameworks and increasing the competitiveness of the industry.
Why it matters: The changes are expected to make the regulatory procedures faster, to increase the period of data and market protection significantly, and to provide stronger innovation incentives for the entire EU.
Source: EMA and PharmTech coverage (Dec. 11, 2025). Pharmaceutical Technology
Summary of Key Themes (8–13 Dec)
✔ New formulations & approvals: DAYBUE® STIX expands Rett syndrome treatment options; antibiotic first under CNPV pilot strengthens U.S. drug supply.
✔ Hospital & infectious disease focus: TYZAVAN™ launch supports readiness against serious bacterial infections.
✔ Rare disease pipeline growth: Mirum + Bluejay deal accelerates HDV therapy development.
✔ Regulatory strategy: FDA’s new acceleration pathway (CNPV) and EU legislative overhaul reflect shifting global frameworks.
