The Austin, TX-based biotech told Endpoints News it plans to submit an application to the FDA in April that would seek approval for its drug, Aqneursa. Known generically as N-acetyl-L-leucine, Aqneursa was first approved in 2024 to treat neurological symptoms related to another rare disease called Niemann-Pick disease type C. There are currently no approved treatments specifically for ataxia-telangiectasia. Seventy-three patients with the disorder were enrolled as part of the Phase 3 trial, an

Novartis has announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation to ianalumab, a treatment for Sjögren’s disease, the second most prevalent rheumatic autoimmune condition. Ianalumab is a fully human monoclonal antibody that targets B-cells and inhibits their activation and survival by blocking B-cell activating factor receptor (BAFF-R). Novartis intends to submit ianalumab for regulatory approval globally in early 2026. If approved, it would re

Jan. 22, 2026 -- Mabwell (688062.SH), an innovation-driven biopharmaceutical company with a fully integrated industry chain, announced that the first patient has been dosed in a clinical trial in the U.S. of its independently developed novel CDH17-targeting antibody-drug conjugate (ADC) (R&D code: 7MW4911) for the treatment of advanced colorectal cancer and other advanced gastrointestinal tumors. The Phase I/II study (NCT07216560) represents the first-in-human (FIH) study of 7MW4911 in the Unit

Jan. 22, 2026 -- Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical company focused on advancing innovative therapies for cancer patients with significant unmet needs, today announced positive interim results from the open-label pharmacokinetic (PK) cohort of its ongoing CLEER-001 clinical trial evaluating HT-001 in cancer patients receiving EGFR inhibitor therapy. In the open-label PK cohort, 100% of evaluable patients achieved clinical response by Week 6, accompanied b

January 21, 2026 – 6pm CET - OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnemo: OSE), today acknowledges the announcement by its partner Veloxis Pharmaceuticals, Inc. that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to pegrizeprument (VEL-101) for the prevention of organ rejection in patients undergoing liver transplantation. Pegrizeprument (also known as VEL-101) is a novel investigational immunomodulatory monoclonal antibody fragment originally discovere

Jan. 21, 2026 -- BPGbio, Inc ., a leading biology-first, AI-powered clinical-stage biopharma company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead candidate, BPM31510, for the treatment of primary oenzyme Q10 (CoQ10) deficiency (PCQD). Primary CoQ10 deficiency is an ultra-rare, life-threatening mitochondrial disorder affecting fewer than one in 100,000 individuals. The condition severely impairs cellular energy product

January 21, 2026 Athos Therapeutics, Inc. ("Athos"), an AI software and clinical stage biotechnology company pioneering the development of precision small molecule therapeutics for patients with immune-mediated diseases, announced today that it has been granted US FDA authorization to commence an international Phase 2 clinical trial of ATH-063 in patients with moderately to severely active, biologic-refractory ulcerative colitis. In addition to the main efficacy and safety assessment goals of

January 21, 2026 -- Rosemont Pharmaceuticals Announces License Agreement with Fidelity Biopharma to Market ONTRALFY™ (tizanidine oral solution) for Treatment of Spasticity in Adults. Product will be available nationwide in February Rosemont Pharmaceuticals has announced a license agreement with Fidelity BioPharma Company to market ONTRALFY™ (tizanidine oral solution 2 mg/5 mL) in the United States. Product will be available nationwide in February. ONTRALFY is the first FDA approved oral solut

-Initiation of phase 1 portion of the trial planned in 2H 2026 -Phase 2 portion of the trial will initiate development of the elraglusib tablet in specific indications, including refractory melanoma and additional target solid tumor and hematologic cancers -Program builds on early clinical evidence of monotherapy activity observed in immune checkpoint inhibitor (CPI)-refractory metastatic melanoma in a previously completed trial Jan. 21, 2026 -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Act

– No objections raised by FDA to advancement of pilavapadin into Phase 3 development utilizing two-arm registrational studies of 10 mg daily dose compared to placebo – Jan. 21, 2026 -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced the successful completion of the End-of-Phase 2 (EOP2) Meeting with the U.S. Food and Drug Administration (FDA) for pilavapadin in diabetic peripheral neuropathic pain (DPNP). During the meeting, the FDA raised no objections to the advancement of pilav