The agency’s “plausible mechanism” framework targets genome editing and RNA-based approaches, and outlines how sponsors can build substantial evidence using clinical data, supported by disease biology, natural-history data, and target confirmation, when randomized trials are not feasible.
On February 23, 2026, the FDA approved Loargys (pegzilarginase-nbln) from Immedica Pharma for hyperargininemia in adults and pediatric patients 2 years and older with Arginase 1 Deficiency, in conjunction with dietary protein restriction. ARG1-D affects an estimated 250 people in the U.S.A. Immedica Pharma’s CEO, Anders Edvell,
On February 20, 2026, the FDA approved Bysanti (milsaperidone) from Vanda Pharmaceuticals (VNDA, Nasdaq) for schizophrenia and for manic or mixed episodes associated with bipolar I disorder in adults. Vanda describes Bysanti as a new chemical entity atypical antipsychotic that is bioequivalent to iloperidone across the therapeutic dosing spectrum, enabling
FDA agreed to review Moderna’s amended mRNA influenza vaccine application after issuing a refusal-to-file letter. The revised approach splits review by age, with accelerated approval in adults 65 and older contingent on a confirmatory post-marketing study.
The FDA approved labeling changes for six menopausal hormone therapy products, removing certain risk statements from the boxed warning. The agency said the action follows a literature review initiated in November 2025.
On February 12, 2026, the FDA approved Adquey (difamilast 1%) ointment from Acrotech Biopharma for the topical treatment of mild to moderate atopic dermatitis in adults and pediatric patients aged 2 years and older. The commercial question centers on where difamilast can earn durable share against entrenched topicals. Mechanistically, difamilast
A phase III study of more than 2,000 patients found post-surgical ctDNA detection was associated with a four- to six-fold increase in recurrence risk, according to Guardant Health.
The FDA approved Novocure’s Optune Pax for adults with locally advanced pancreatic cancer through the PMA pathway. The decision was based on a randomized, controlled study with follow-up up to five years.
Series D backs global rollout of automated IDMO factories as Cellares targets lower costs, higher throughput, and commercial scale.
Affinia Therapeutics secured FDA acceptance of its AFTX-201 IND, enabling initiation of the Phase 1/2 UPBEAT study in BAG3-associated dilated cardiomyopathy with safety and tolerability as key endpoints.
ImmunityBio has launched a Phase 2 outpatient trial evaluating a chemotherapy-free combination of off-the-shelf CD19-targeted NK cells, ANKTIVA, and rituximab in relapsed or refractory indolent B-cell lymphomas.
February 12, 2026 is the expected record date for the spin-off of Atrium Therapeutics, which will hold Avidity's early-stage precision cardiology programs.
Piramal Pharma Solutions has commercialized a tablet-in-capsule drug delivery capability following a five-month technology transfer between its Ahmedabad development facility and its commercial manufacturing site in Pithampur, India.
AstraZeneca has shifted its U.S. listing to ordinary shares on the New York Stock Exchange as it targets $80 billion in annual revenue by 2030, alongside announcing major investment and licensing commitments in China.
