Approvals

• The FDA approved five first-in-class therapies during the first half of 2026, targeting obesity, AML, kidney disease, hearing loss, and bladder cancer. • On April 14, Travere Therapeutics received approval for sparsentan (Filspari), the first treatment specifically approved for focal segmental glomerulosclerosis (FSGS), a rare kidney disease. • These approvals are significant as they replace off-label or burdensome parenteral treatments with more accessible options, such as all-oral regimens that allow patients to be treated at home.

• The FDA approved six novel drugs for pediatric patients under 18 in 2026, including Copper histidinate (Zycubo) by Sentynl Therapeutics Inc. • Approved on January 12, 2026, Zycubo is indicated for the management of Menkes disease in children via subcutaneous formulation. • Clinical data from two open-label trials showed that patients starting treatment within four weeks of birth experienced a 78% reduction in the risk of death.

• The US Food and Drug Administration (FDA) approved 22 novel drugs during the first half of 2026, expanding treatment options across various medical specialties. • Key approvals include copper histidinate (formerly CUTX-101) for Menkes disease, supported by two open-label studies involving 129 patients treated with subcutaneous administration for up to 3 years. • The FDA also granted approval for a new treatment targeting mild to moderate Alzheimer's disease (AD) based on phase 3 clinical trial data from both adult and pediatric populations.

• The American Journal of Health-System Pharmacy has outlined the pharmaceutical pipeline for novel drug approvals anticipated between 1Q 2026 and 4Q 2026. • The upcoming pipeline focuses on therapies for rare inherited genetic disorders, immunologic and inflammatory diseases, and advancements in gene therapy and targeted oncology. • This tracking is critical for health-system pharmacists to effectively manage hospital formularies, allocate financial resources, and optimize patient care.

• The FDA extended its review of a supplemental biologics license application (sBLA) submitted by Eisai and Biogen on May 8, 2026. • The application concerns a once-weekly subcutaneous autoinjector starting regimen for lecanemab-irmb (Leqembi Iqlik), used to treat early Alzheimer disease. • This regulatory delay pushes the Prescription Drug User Fee Act (PDUFA) action date to August 24, 2026.

• Regulatory bodies are increasingly integrating real-world evidence (RWE) into the drug approval process to supplement traditional randomized clinical trials. • This shift utilizes real-world data (RWD) sourced from electronic health records, administrative claims databases, disease registries, and digital health tools. • The adoption of RWE allows for a more comprehensive understanding of patient health and healthcare delivery, potentially accelerating decision-making for pharmaceutical companies.

• The FDA approved Icotyde (icotrokinra), developed by Johnson & Johnson Innovative Medicine, on March 17, 2026. • The drug is an interleukin-23 (IL-23) receptor antagonist specifically indicated for patients with moderate-to-severe plaque psoriasis. • This approval provides a new therapeutic option for managing a chronic inflammatory skin condition by targeting specific immune responses.