New data shows nearly three‑year median overall survival

Original title: “Amivantamab regimen delivers longest survival in EGFR Ex20ins lung cancer”

CVMP opinions on veterinary medicinal products The Committee adopted a positive opinion for a marketing authorisation for Cirbloc, for the active immunisation of pigs from…

Original title: “Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 8-10 September 2026”

Amir Hefni, Chief Executive Officer ... EMERALD trial marks a significant milestone for Resolution Therapeutics and reflects our progress toward a potential new treatment paradigm for end-stage liver disease. Patients with this condition face very limited options beyond transplantation and symptom management, and RTX001 has the potential to offer a differentiated, transformational therapeutic approach. We are working with urgency to advance the clinical program and ... Amir Hefni, Chief Executive Officer of Resolution Therapeutics, said: “Dosing the final patient in the EMERALD trial marks a significant milestone for Resolution Therapeutics and reflects our progress toward a potential new treatment paradigm for end-stage liver disease. Patients with this condition face very limited options beyond transplantation and symptom management, and RTX001 has the potential to offer a differentiated, transformational therapeutic approach. We are working with urgency to advance the clinical program and bring this therapy closer to the patients who need it.” 11, 2026 (GLOBE NEWSWIRE) -- Resolution Therapeutics Limited (“Resolution” or “Company”), a clinical-stage biopharmaceutical company pioneering Regenerative Macrophage Therapy (RMT) in inflammatory and fibrotic diseases, today announced that the final patient has been treated in EMERALD, the Company’s Phase I/II clinical trial evaluating its engineered RMT, RTX001, in patients with end-stage liver disease (ESLD) and a history of decompensated liver cirrhosis. Interim data are expected in the fourth quarter of 2026. Liver transplantation remains the only disease-modifying treatment option for patients with ESLD. RTX001 is engineered with IL-10 and MMP-9 to enhance the anti-inflammatory and anti-fibrotic effects of RMT, with the aim of improving patient outcomes and durability of response in end-stage liver disease. RTX001 is administered via peripheral IV requiring no pre-conditioning, bridging therapy, or lymphodepletion; the outpatient infusion takes less than 10 minutes. The manufacturing process was developed with commercial-scale production in mind, and ongoing process refinements have reduced cost-of-goods by 40% to date. In pre-clinical studies, RTX001 has demonstrated no significant safety signals and did not require prolonged monitoring. Its multimodal mechanism acts on the disease microenvironment, creating potential across a broad range of chronic inflammatory and fibrotic diseases. ... Resolution Therapeutics is a clinical-stage biopharmaceutical company focused on pioneering Regenerative Macrophage Therapy in inflammatory and fibrotic diseases.

Original title: “Resolution Therapeutics completes enrollment in Phase I/II EMERALD clinical trial evaluating RTX001 in end-stage liver disease - BioSpace”

Satralizumab also produced significant ... rescue therapy compared with placebo.² The trial's safety profile was consistent with more than a decade of satralizumab clinical trial and post-approval experience in neuromyelitis optica spectrum disorder (NMOSD).¹ · MOGAD is a rare autoimmune disease in which the ... Satralizumab also produced significant improvements on key secondary measures, including a 66% reduction in annualized relapse rate, a 79% reduction in annualized rate of active MRI lesions, and a 73% reduction in need for rescue therapy compared with placebo.² The trial's safety profile was consistent with more than a decade of satralizumab clinical trial and post-approval experience in neuromyelitis optica spectrum disorder (NMOSD).¹ · MOGAD is a rare autoimmune disease in which the immune system mistakenly attacks the optic nerves, brain, or spinal cord, causing unpredictable attacks that can lead to vision loss, confusion, muscle weakness, and disability. Satralizumab cut MOGAD relapse risk 68% in a phase 3 trial, positioning it as the first potential treatment for this rare disease. ... granted priority review to a supplemental biologics license application for Roche’s satralizumab (Enspryng) for treating myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), Roche announced September 10, 2026. If approved, satralizumab would become the first and only disease-modifying therapy for MOGAD, a rare autoimmune disease of the central nervous system with no currently approved treatments, according to the company. Priority review advances satralizumab toward potential first approved disease-modifying therapy for MOGAD, a rare CNS demyelinating disorder associated with relapses that can cause cumulative, irreversible neurologic disability. The trial's most common adverse events occurring more frequently with satralizumab than placebo included injection-related reactions, influenza, arthralgia, back pain, sinusitis, and diarrhea, though rates of treatment interruption were low and similar between groups.² · Roche. US FDA grants priority review for Roche's Enspryng for MOGAD, an autoimmune disease with no approved treatments.

Original title: “FDA Grants Priority Review to Roche's Satralizumab for MOGAD | BioPharm International”

Announced mere weeks after Biohaven sold off rights to the therapy, opakalim, the halt affects a study some analysts believe is necessary to support a potential approval.

Original title: “FDA places partial hold on Biohaven’s epilepsy drug”

The enrollment pause comes after BHV-7000 failed to show efficacy in a midstage depression trial late last year. The news is another regulatory hit for Biohaven after the FDA declined to approve its spinocerebellar ataxia treatment in November 2025.

Original title: “Biohaven takes another hit as FDA pauses enrollment in pivotal trials for embattled epilepsy drug”

Guidance offers first alternative to transplant‑based first‑line care

Original title: “NICE backs ibrutinib regimen for untreated mantle cell lymphoma”

Unmet need and orphan-drug potential: Rare mTOR-driven disorders may provide focused development opportunities, particularly for patients who experience progression or intolerance with established therapies. Successful clinical development could support orphan-drug incentives and adoption within specialized treatment ... Unmet need and orphan-drug potential: Rare mTOR-driven disorders may provide focused development opportunities, particularly for patients who experience progression or intolerance with established therapies. Successful clinical development could support orphan-drug incentives and adoption within specialized treatment centers. Key Topics Covered 1. Report Introduction 2. Sapanisertib Overview in potential indication like Bladder cancer, Cancer, Endometrial cancer, Fallopian tube cancer, Glioblastoma, Neuroendocrine tumours, Ovarian cancer, Peritoneal cancer, Precursor cell lymphoblastic leukaemia-lymphoma, Soft tissue sarcoma, Thyroid cancer, and Urogenital cancer 2.1. Product Detail 2.2. Sapanisertib Clinical Development 2.2.1. Sapanisertib Clinical studies 2.2.2. Sapanisertib Clinical trials information 2.2.3. Safety and efficacy 2.3. Other Developmental Activities 2.4. Product Profile 3. Sapanisertib Competitive Landscape (Marketed Therapies) 4. Competitive Landscape (Late-stage Emerging Sapanisertib Therapies) 5. Sapanisertib Market Assessment 5.1. Sapanisertib Market Outlook in potential indications 5.2. 7MM Analysis 5.2.1. Sapanisertib Market Size in the 7MM for potential indications 5.3. Country-wise Market Analysis 5.3.1. Sapanisertib Market Size in the United States for potential indications 5.3.2. Sapanisertib Market Size in Germany for potential indications 5.3.3. Sapanisertib Market Size in France for potential indications 5.3.4. Sapanisertib Market Size in Italy for potential indications 5.3.5. Sapanisertib Market Size in Sp Forecast assumptions consider eligible patient populations, clinical development probability, potential launch timing, treatment duration, pricing, reimbursement, competitive intensity and anticipated uptake. Competitive intelligence includes pipeline analysis, comparison with existing mTOR inhibitors and assessment of emerging therapies that could influence market penetration.

Original title: “Sapanisertib Market Size Analysis and Sales Forecast 2020-2034: Commercial Outlook Hinges on Late-Stage Success”

Enspryng also provided significant ... rescue therapy use.1 · The safety profile in the METEOROID study was consistent with data from more than a decade of Enspryng clinical trial and post-approval experience in neuromyelitis optica spectrum disorder (NMOSD).1 · MOGAD is a rare autoimmune disease where the ... Enspryng also provided significant improvements across key secondary measures, including annualised relapse rate, MRI lesion activity and rescue therapy use.1 · The safety profile in the METEOROID study was consistent with data from more than a decade of Enspryng clinical trial and post-approval experience in neuromyelitis optica spectrum disorder (NMOSD).1 · MOGAD is a rare autoimmune disease where the immune system mistakenly attacks parts of the central nervous system.2,3 It can cause sudden attacks on the optic nerves, brain and spinal cord, which can lead to vision loss, confusion, muscle weakness and disability.2-6 If approved, Enspryng would be the first and only disease-modifying therapy for MOGAD (myelin oligodendrocyte glycoprotein antibody-associated disease) Filing acceptance is based on the Phase III METEOROID study showing that Enspryng reduced the risk of relapses by 68%1 · MOGAD is a rare disease of the central nervous system characterised by unpredictable attacks of the optic nerves, spinal cord or brain2,3 Roche is the majority shareholder in Chugai Pharmaceutical, a major innovator in the Japanese therapeutic antibody market. ... All trademarks used or mentioned in this release are protected by law. References [1] Levy M, et al. Safety and efficacy of satralizumab in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD): Results from the Phase 3 METEOROID trial. Basel, 10 September 2026 - Roche (SIX: RO, ROP; OTCQX: RHHBY) announced today that the U.S. Food and Drug Administration (FDA) has granted Priority Review to a supplemental Biologics License Application (sBLA) for Enspryng® (satralizumab) for the treatment of myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). The filing acceptance is based on positive results from the Phase III METEOROID study, which showed that Enspryng provided consistent, clinically meaningful improvements for people living with MOGAD.1 This marks the second FDA Priority Review for Enspryng, following the one granted for thyroid eye disease (TED) in June 2026.

Original title: “U.S. FDA grants Priority Review for Roche’s Enspryng for MOGAD, an autoimmune disease with no approved treatments”

Some rare cancers may have genetic changes that are being studied in clinical trials. Learn more about these trials and who’s eligible for them. Smaller studies can make it hard to collect enough data about how well a treatment works. “Healthcare providers, patients, and families often didn’t know what trials existed for rare cancers,” Dr. Weigel said. “Someone who wanted to join a clinical trial might have to travel long distances to places with expertise in those conditions.” · New approaches to cancer research are expanding clinical trial opportunities for some people with rare cancers. These include tumor-agnostic therapies and basket trials. This study design allows people with rare cancers and those with more common cancers to take part in the same clinical trial. If the treatment is shown to be safe and effective, it can be approved to treat several different cancers with that genetic change. “Tumor-agnostic basket trials have transformed cancer care and accelerated new targeted therapies,” said Dr. Every new cancer medication must go through clinical trials before it can be approved for use in the United States. Clinical trials are studies that involve people. They test whether new medicines, procedures, devices, and other treatments are safe and work as intended. “Clinical trials help cancer researchers develop new therapies faster,” said Brenda J. Not every rare cancer has a genetic change that can be targeted with current therapies, so basket trials may not be an option. “Your cancer care team can help determine whether you’re eligible to join a basket trial. If you are, they should be able to help you enroll in the study or connect you to the right people,” Dr. Weigel said. You can also find support with clinical trials matching through ACS ACTS (Access to Clinical Trials & Support).

Original title: “Are There Clinical Trials for Rare Cancers? | American Cancer Society”

The treatment improved his condition but did not eliminate the disease. He eventually learned about an investigational CAR-T cell therapy. CAR-T therapy involves collecting a patient's immune cells and modifying them so they can recognize and attack targeted cells. Lomonaco was able to enroll in a clinical trial ... The treatment improved his condition but did not eliminate the disease. He eventually learned about an investigational CAR-T cell therapy. CAR-T therapy involves collecting a patient's immune cells and modifying them so they can recognize and attack targeted cells. Lomonaco was able to enroll in a clinical trial and received the treatment at The James Cancer Hospital at The Ohio State University. He was diagnosed with AL amyloidosis, a rare disease that causes abnormal proteins to build up in the body's organs The treatment Lomonaco received is not yet widely available. He said the clinical trial closed shortly after he underwent the procedure, leaving other people with the disease without the same option. After about a year of tests and visits to several doctors, Michael Lomonaco was diagnosed with AL amyloidosis, a rare disease that causes abnormal proteins to build up in the body's organs. In Lomonaco's case, the disease was affecting his heart. (provided) ... LOVELAND, Ohio (WKRC) - A Loveland man who once struggled to walk from his car to his desk without stopping to catch his breath is now back to exercising after an experimental treatment for a rare disease.

Original title: “Loveland man credits experimental treatment with putting rare disease in remission”

Remission rates observed in a Phase 2 trial fell short of Wall Street's expectations, though some analysts believe the company should see better results in late-stage testing.

Original title: “Tyra disappoints investors with bladder cancer data”

September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.

Original title: “Novo’s canned heart disease trials, Novartis’ clinical fails, first approval for Alexander disease”

SMC decision expands options for people with chronic itch

Original title: “Livdelzi accepted in Scotland for treatment of PBC”