FDA

Ascendis Pharma A/S announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for once-weekly TransCon CNP (navepegritide) in the European Union for the treatment of achondroplasia in children two years of age and older whose epiphyses are not closed.

Actinium Pharmaceuticals, announced three presentations, including an oral presentation and two e-posters, highlighting preclinical data supporting ATNM-400's broad potential at the 39th Annual Congress of the European Association of Nuclear Medicine (EANM), October 17–21, 2026, in Vienna, Austria.

Omeros Corporation announced that the European Medicines Agency’s CHMP issued a negative opinion on its marketing authorization application for narsoplimab in TA-TMA, following re-examination of its original negative opinion from June.

Ionis Pharmaceuticals, Inc announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the indication extension for TRYNGOLZA® (olezarsen) as an adjunct to diet, in combination with other lipid-lowering therapies, for the treatment of adult patients with severe hypertriglyceridemia (sHTG, fasting triglyceride levels of ≥880 mg/dL [≥10 mmol/L]).

Fractyl Health, Inc. announced positive topline six-month results from the Pivotal Cohort of its ongoing REMAIN-1 study (Pivotal Cohort), a 308-participant, multicenter, randomized, double-blind, sham-controlled study of the Revita DMR System (Revita) for weight maintenance in adults with obesity following discontinuation of GLP-1 therapy.

Organon announced that Sebela Women’s Health will present interim results from an ongoing Phase 3 study evaluating MIUDELLA®, a hormone-free, copper intrauterine device (IUD), through 6 years of use at the 2026 Society of Family Planning (SFP) Annual Meeting, taking place October 17 to 19, 2026, in San Francisco, California. In June 2026, Organon exclusively licensed global rights to MIUDELLA from Sebela Women’s Health.

Tango Therapeutics, Inc. announced that the Company will present updated data from a Phase 1/2 study of vopimetostat in combination with daraxonrasib or zoldonrasib in patients with MTAP deleted RAS mutant (MTAPdel/RASmut) metastatic pancreatic ductal adenocarcinoma (PDAC) and non-small cell lung cancer (NSCLC) at the European Society For Medical Oncology (ESMO) Congress 2026, which will be held October 23-27, 2026 in Madrid, Spain.

Regeneron Pharmaceuticals, Inc announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of an alternative dosing schedule for Libtayo® (cemiplimab) across all approved indications.

Tiziana Life Sciences, Ltd. announced that the last patient has begun dosing in its Phase 2 randomized, placebo-controlled clinical trial evaluating intranasal foralumab in patients with early/mild Alzheimer’s disease (AD).

Opus Genetics, Inc. presented clinical findings from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026 in Rio de Janeiro, Brazil.

MacroGenics, Inc announced that the first patient has been dosed in the Phase 1 study of MGC030, a first-in-class antibody-drug conjugate (ADC) directed against an undisclosed target, in patients with advanced solid tumors.

Crescent Biopharma, Inc. announced clinical pipeline momentum, including enrolling patients in cohorts combining CR-001, a PD-1 x VEGF bispecific antibody, plus standard of care chemotherapy regimens in its global Phase 1/2 ASCEND trial, as well as the initiation of additional antibody-drug conjugate (ADC) combination studies with CR-001.

Vogenx, Inc. announced the initiation of EMERGE (VGX-001-013), its Phase 2b clinical trial evaluating mizagliflozin for the treatment of post-bariatric hypoglycemia (PBH).

Revolution Medicines, announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of RASONQUE (daraxonrasib) once-daily oral tablets as a monotherapy indicated for the treatment of adult patients with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy.

Edesa Biotech, Inc. reported that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application for its Phase 2 clinical study of EB06 in vitiligo, enabling Edesa to expand recruitment to the United States.

Actinium Pharmaceuticals, announced that an oral presentation with early results from the first-in-human Phase 1 study of ATNM-400 in metastatic castration-resistant prostate cancer (mCRPC) will be presented at the 39th Annual Congress of the European Association of Nuclear Medicine (EANM), October 17–21, 2026, in Vienna, Austria.

New data analyses from Eli Lilly show patients with moderately to severely active ulcerative colitis (UC) and Crohn's disease treated with Omvoh (mirikizumab-mrkz) achieved multiple treatment goals simultaneously at one year, as measured by comprehensive disease control in UC and corticosteroid-free deep remission in Crohn's disease.

Eupraxia Pharmaceuticals Inc announced that the ongoing RESOLVE study evaluating the safety and efficacy of EP-104GI for the treatment of Eosinophilic Esophagitis ("EoE") has reached its enrollment target.

- Jasper Therapeutics, announced that it has completed enrollment of the IgA nephropathy (IgAN) cohort of its Phase 2 basket study evaluating vensobafusp alfa (KP104), a first-in-class bifunctional biologic that simultaneously and selectively targets both the alternative and terminal complement pathways.

Protara Therapeutics, Inc. announced that it has completed enrollment of the Phase 2b, open-label dose confirmation phase of the THRIVE-3 clinical trial evaluating IV Choline Chloride in patients with intestinal failure who are dependent on parenteral support (PS).

Legend Biotech Corporation announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for LB2501, an investigational in vivo CAR-T therapy designed to generate CD19/CD20 dual-targeting chimeric antigen receptor T (CAR-T) cells directly within the patient, for the treatment of relapsed or refractory B-cell malignancies, including B-cell non-Hodgkin lymphoma.

Palvella Therapeutics, Inc. announced that it will present two posters featuring previously reported results from its Phase 3 SELVA and Phase 2 TOIVA clinical studies of QTORIN™ rapamycin, along with additional new pediatric analyses, at the Pediatric Dermatology Research Alliance (PeDRA) Annual Conference, taking place October 15–17, 2026, in Alexandria, Virginia.

Immunocore Holdings plc announced that the first patient with type 1 diabetes (T1D) has been dosed in the Phase 1 trial (NCT07493122) of IMC-S118AI1.

Krystal Biotech, Inc announced a positive clinical update supporting initial proof of concept for KB113, an intratumoral immunotherapy designed to drive sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) in the tumor microenvironment and promote systemic immune-mediated tumor clearance, in the treatment of basal cell carcinomas (BCCs) in patients with Gorlin syndrome

Cellectar Biosciences, announced that efficacy results from a subset of patients treated with iopofosine I 131 immediately post-Bruton Tyrosine Kinase inhibitor (BTKi) therapy in the company’s Phase 2b CLOVER WaM clinical trial of iopofosine I 131 to treat relapsed or refractory (r/r) Waldenström macroglobulinemia (WM) were presented today at the International Workshop for Waldenström Macroglobulinemia (IWWM) taking place October 14 to October 16, 2026, in Palm Springs, California.

Seres Therapeutics, Inc. announced that preclinical data supporting the development of SER-428, an investigational oral liquid live biotherapeutic product (LBP) designed to reduce the risk of bloodstream infections (BSIs) in medically vulnerable patients following sepsis and antibiotic exposure, will be presented at IDWeek 2026, taking place October 21–24 in Washington, D.C.

Aquestive Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has acknowledged its resubmission of the New Drug Application (NDA) for Anaphylm™ (dibutepinephrine) sublingual film for the treatment of Type I allergic reactions, including anaphylaxis, as complete

Insmed Incorporated a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, will feature the global PALM Clinical Development Program at the American College of Chest Physicians® Annual Meeting 2026 (CHEST 2026), which is investigating treprostinil palmitil inhalation powder (TPIP) in a range of serious pulmonary diseases.