As the year draws to a close, the U.S. Food and Drug Administration (FDA) has made significant strides in its mission to promote public health through its regulatory decisions. For many, the end of the year translates into a whirlwind of final tasks before the holiday season, and the FDA is no exception. A glimpse back at December reveals a month characterized by groundbreaking approvals that promise to reshape treatment options for various conditions, coupled with some notable setbacks.

Notable Firsts in Drug Approvals

December witnessed an array of firsts, underscoring the FDA’s commitment to advancing healthcare solutions. Eli Lilly’s Zepbound made history as the first FDA-approved drug treatment for obstructive sleep apnea, a prevalent condition traditionally managed through medical devices. Clinical trials revealed that treatment with Zepbound not only led to weight reductions—a key risk factor for sleep apnea—but also resulted in improved breathing. This marks a significant addition to Lilly’s rapidly growing portfolio of metabolic medications.

Also noteworthy was the approval of Ionis Pharmaceuticals’ olezarsen, now branded as Tryngolza. A promising option for patients with familial chylomicronemia syndrome—a rare inherited metabolic disorder—Tryngolza will be pivotal for individuals who have long relied on restrictive diets to manage their condition. This marks Ionis’s maiden solo venture into commercializing a product following prior collaborations with larger firms.

The landscape for trauma care expanded with Humacyte’s FDA approval of Symvess, a bioengineered blood vessel designed to restore blood flow in limb trauma cases where traditional vein grafting is not feasible. This innovative regenerative medicine option provides hope for patients facing limb loss.

Advancements in Immunology and Rare Diseases

Regulatory decisions in the field of immunology also stood out, with Organon’s Vtama receiving approval for treating atopic dermatitis in adults and children, expanding on its prior indication for plaque psoriasis. Furthermore, Galderma’s nemolizumab, a targeted therapy for moderate-to-severe atopic dermatitis, received FDA approval, marking a significant advancement for patients struggling with this chronic condition.

In the realm of rare diseases, Neurocrine Biosciences introduced Crenessity, an innovative treatment for congenital adrenal hyperplasia, while Novo Nordisk bolstered its rare disease portfolio with Alhemo, aimed at reducing bleeding episodes in hemophilia patients. Vertex Pharmaceuticals also made headlines with the approval of Alyftrek, a new cystic fibrosis therapy that addresses additional mutations not covered by existing treatments.

Cancer Therapy Developments

The cancer landscape saw significant regulatory milestones with the FDA granting accelerated approvals to several new therapies. Merus Therapeutics’ zenacutuzumab is now approved for advanced non-small cell lung cancer and pancreatic adenocarcinoma, targeting a unique genetic signature. AstraZeneca’s Imfinzi expanded its indications to include limited-stage small cell lung cancer—a pivotal development for patients in this challenging therapeutic space.

Additionally, Xcovery Holdings received approval for Ensacove, a treatment targeted at advanced non-small cell lung cancer with ALK mutations. Pfizer’s Braftovi also secured accelerated approval for metastatic colorectal cancer, marking a significant advance in targeted cancer therapy, while BeiGene’s Tevimbra was approved for use in gastric cancers, solidifying its position in the oncology market.

Challenges and Setbacks

While the month was rife with accomplishments, it was not without its challenges. Astellas Pharma faced rejection from the FDA for an alternative dosing regimen for Izervay, a treatment for geographic atrophy, limiting its competitive edge. Furthermore, Intercept Pharmaceuticals encountered significant hurdles with its liver disease treatment Ocaliva, facing both safety alerts and European market challenges.

The FDA’s rejection of Zealand Pharma’s glepaglutide for short bowel syndrome and Johnson & Johnson’s complete response letter regarding an injectable version of Rybrevant highlight the complexities and rigorous standards of the regulatory process.