First and only approved treatment indicated for Ataxia-Telangiectasia (A-T) in the United States or anywhere in the world Second FDA-approved indication for AQNEURSA, following previous approval for Niemann-Pick disease type C (NPC) AQNEURSA is commercially available now AQNEURSA provides a long-awaited treatment option for patients living with A-T IntraBio Inc., a leader in the discovery, development, and commercialization of novel therapies for rare and common neurodegenerative diseases, tod

—sNDA Under Review for Full Approval Is Based on Positive Results From Phase 3b ENCORE Study— —Full Approval Could Bring ARIKAYCE to Patients Earlier in Their Disease Course and Would Fulfill Post-Marketing Requirement From FDA— Sept. 21, 2026 -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that the U.S. Food and Drug Administr

Sept. 20, 2026 -- Accro Bioscience Inc. (Accro Bio), a clinical-stage biotechnology company pioneering the development of novel therapeutics for immune mediated diseases, today announced the dosing of their first patient in the Phase IIb clinical trial of AC-101, a selective RIPK2 inhibitor, in patients with moderate-to-severe ulcerative colitis (UC). The Phase IIb study is a multicenter, double-blind, placebo-controlled clinical trial designed to evaluate AC-101 in patients with moderate-to-sev

Eli Lilly is sharpening its competitive edge in the crowded oral SERD arena as the FDA has approved a combination of its Inluriyo with its CDK4/6 inhibitor Verzenio. The label expansion builds on Inluriyo’s existing monotherapy indication, approved nearly a year ago to treat ER-positive, HER2-negative breast cancer that carries ESR1 mutations in previously treated patients. Given the longer progression-free survival (PFS), Jake Van Naarden, president of Lilly Oncology and head of corporate busi

September 17, 2026 – The NationalInstitute of Diabetes and Digestive and Kidney Diseases (NIDDK), a division ofthe National Institutes of Health (NIH), has awarded SignaBlok, Inc., a biopharmaceutical company focused on developing novel peptide antagonists of cell receptors, a Phase I Small Business Innovation Research (SBIR) grant to develop a new therapeutic approach for inflammatory bowel disease (IBD). SignaBlok’s approach to IBD therapy targets TREM-1, a cell-surface receptor that has emer

It is intended for scientific, financial and investor media only. If approved, Jyseleca will be indicated for the treatment of patients with active axial spondyloarthritis (axSpA), both non-radiographic and radiographic forms of the disease, who have responded inadequately to conventional therapy. Despite the availability of multiple treatment options, nearly half of people living with axSpA do not respond adequately to current therapies, and just 10-20% reach inactive disease within 16-24 weeks

Protocol Submitted for IRB Review at a Leading Cancer Research Institution, Advancing the Study Toward Site Initiation Sept. 17, 2026 -- MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA) ("MIRA" or the "Company"), a clinical-stage pharmaceutical company developing novel oral small-molecule therapeutics, today announced that, following review and comments from the U.S. Food and Drug Administration (FDA), the Phase 2a protocol evaluating Ketamir-2 in chemotherapy-induced peripheral neuropathy (CIPN) has

Novartis receives positive CHMP opinion for Cosentyx® in polymyalgia rheumatica (PMR) If approved, Cosentyx (secukinumab) will be first IL-17A inhibitor licensed in Europe for adults with polymyalgia rheumatica (PMR), providing new treatment option after steroids¹ Recommendation based on data showing Cosentyx doubled sustained remission rates and delivered steroid sparing effects vs placebo; safety was consistent with established Cosentyx profile² Despite being a common inflammatory rheumatic di

Silo Pharma Receives U.S. Patent Allowance for SPC-15 PTSD Treatment, Expanding Intellectual Property Portfolio Allowed claims cover compositions and methods for preventing or mitigating stress-induced behavioral responses Sept. 18, 2026 -- Silo Pharma (Nasdaq: SILO) (“Silo” or “the Company”), a developmental-stage biopharmaceutical company focused on novel therapeutics and drug delivery systems with a wholly owned managed AI agent platform, today announced that the United States Patent and Tr

FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A Ultragenyx’s UltraCare® program will support access, and commercial product is expected to be available to ship to Qualified Treatment Centers within 30-60 days FAYUVI marks the second gene therapy approval, and sixth FDA approval overall, for Ultragenyx The Company received a Priority