AbbVie is eyeing regulatory filings in 2027 for the skin disease drug lutikizumab after clearing multiple endpoints in a late-stage trial, but the results have disappointed some analysts, raising questions about its commercial prospects.

Original title: “AbbVie’s skin disease drug lessens abscess burden in Phase 3 but falls below expectations”

U.S. regulators have accepted a new submission for Vyglxia, offering the chance at a turnaround for another rare disease medicine spurned by previous FDA leadership.

Original title: “Biohaven’s previously rejected rare disease drug to get fresh FDA review”

Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.

Original title: “Biohaven shares pop as FDA accepts resubmission for ataxia drug with adcomm expected”

Brepocitinib is on a roll. After winning approval for an autoimmune skin disease, Roivant Sciences subsidiary Priovant’s drug hit all primary and secondary endpoints in a pair of Phase 3 non-infectious uveitis trials, leading analysts to say the candidate “looked superior to Humira.”

Original title: “Roivant's TYK2/JAK inhibitor reduces need for steroids in inflammatory eye disease”

Results support advancement into phase 2 trials

Original title: “Sitryx reports phase 1b data for SYX-5219”

The pilot will switch approximately six adults from standard cysteamine therapy for seven days; topline data are anticipated in Q1 2027, assuming timely start. Jul 13, 2026 Thiogenesis Receives FDA Rare Pediatric Disease Designation for TTI-0102 for the Treatment of Leigh Syndrome · Jun 17, 2026 Thiogenesis Appoints Dr. Gilad Aharon to Its Board of Directors · Apr 16, 2026 Thiogenesis Therapeutics to Present at 2026 Bloom Burton & Co. Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQB: TTIPF) is a clinical-stage biopharmaceutical company with operations based in San Diego, CA. The Company is publicly traded on the TSX Venture Exchange and in the U.S. on the OTCQB. Thiogenesis is developing sulfur-containing prodrugs that act as precursors to previously approved thiol-active compounds, with the potential to treat serious pediatric diseases with unmet medical needs. Actual results may differ materially due to risks including regulatory actions; the FDA's expectation that, before a registrational trial, the Company establish an optimal dose range, generate sufficient clinical and nonclinical safety data to support chronic administration, and reach agreement with the FDA on an adequate and well-controlled trial design, which may require additional studies, time and expense; delays in study start-up or enrollment; the fact that the study is conducted by an independent investigator and institution; the small size, open-label single-arm design and short treatm The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language. ... Thiogenesis Therapeutics (TTIPF) received FDA clearance on October 16, 2026, to study TTI-0102 in patients with nephropathic cystinosis.

Original title: “FDA clears an application to study a once-daily powder for a rare kidney disease”

/PRNewswire/ -- A new clinical trial funded by Breakthrough T1D recently launched and is testing islet cell transplantation paired with Eledon Pharmaceuticals'... 21, 2026 /PRNewswire/ -- A new clinical trial funded by Breakthrough T1D recently launched and is testing islet cell transplantation paired with Eledon Pharmaceuticals' investigational immunosuppression therapy tegoprubart in people living with type 1 diabetes (T1D) and chronic kidney disease (CKD). The first participant in the trial received an islet cell transplant, marking the first time individuals with T1D and CKD have been included in islet transplant trials. Nearly one in three people living with T1D develop CKD, which increases the risk of kidney failure and other T1D complications. Treatment options for these individuals have remained limited, and until now, potential kidney toxicity related to standard immunosuppressant regimens used in transplants has kept people with T1D and reduced kidney function out of islet cell transplant studies. "Advancing promising islet cell research that is a next step toward durable and scalable cures for type 1 diabetes is a key priority for Breakthrough T1D," said Esther Latres, Ph.D., Breakthrough T1D senior vice president, Research. "We're proud to support this trial that expands cell therapy research and access to people living with T1D and chronic kidney disease for the first time." "This first transplant represents a milestone towards expanding islet cell therapy to a broader population of people living with type 1 diabetes, including those with kidney disease who have historically been excluded from these studies," said Piotr Witkowski, M.D., Ph.D., director of the Pancreatic and Islet Transplant Program at the University of Chicago and principal investigator of the trial. "By replacing tacrolimus with tegoprubart, we hope to protect both the transplanted islets and our patients' kidney function, and show that more people with T1D can safely benefit from this therapy." The multi-center trial is currently enrolling 10 participants at the University of Chicago and the University of Miami. Eligible participants must be 18-70 years old, diagnosed with T1D for more than five years with onset before age 40, living with CKD at stage 1, 2, or 3a, and have impaired awareness of hypoglycemia. This new trial builds on the current Breakthrough T1D-funded study testing tegoprubart with deceased-donor islets transplants in people with severe hypoglycemic events or hypoglycemia unawareness.

Original title: “Breakthrough T1D Funded Clinical Trial Testing Islet Cell Transplantation with Eledon's Tegoprubart Expands to Include People with Type 1 Diabetes and Kidney Disease”

Hibsago is viewed as a potential “functional cure” for chronic infections and an important part of the British drugmaker’s goal of reaching £40 billion in annual revenue by 2031.

Original title: “GSK, Ionis win US approval for first-of-its-kind hepatitis B drug”

Shortly after winning a clearance in the skin disease dermatomyositis, brepocitinib posted results suggesting it might also be a “best-in-disease” medicine for an inflammatory eye condition, one analyst said.

Original title: “Roivant, continuing ascent, claims another victory for rare disease drug”

Vaccination and Disease-Modifying Therapies: Impact of Ocrelizumab vs Fingolimod on Preexisting Vaccine Seropositivity and Vaccine-Preventable Infections in Pediatric Patients With MS in OPERETTA 2 (#P1415). Presented at: MSToronto2026 10th Joint ACTRIMS-ECTRIMS Meeting; 22 October 2026; Toronto, Canada. [8] Butzkueven H, et al. Ocrelizumab Maintains Clinical ... Vaccination and Disease-Modifying Therapies: Impact of Ocrelizumab vs Fingolimod on Preexisting Vaccine Seropositivity and Vaccine-Preventable Infections in Pediatric Patients With MS in OPERETTA 2 (#P1415). Presented at: MSToronto2026 10th Joint ACTRIMS-ECTRIMS Meeting; 22 October 2026; Toronto, Canada. [8] Butzkueven H, et al. Ocrelizumab Maintains Clinical and Safety Outcomes in Older Patients (>55 Years) with Progressive Multiple Sclerosis: 2-Year Results from the CONSONANCE Study (#P1426). Reduction of chronic active lesions (Late-breaking oral #O137): In the first prospective demonstration of a therapeutic effect on chronic active lesions in MS Phase III clinical trials, data show fenebrutinib reduced the volume of slowly expanding lesions (SELs) by up to 30% (p<0.001) in RMS (over 96 weeks) and PPMS (over 120 weeks), along with a 62% (p=0.001) reduction in accumulation of paramagnetic rim lesions (PRLs) in RMS, versus active comparators. Across its Diagnostics and Pharmaceutical divisions, Roche focuses on areas including oncology, neurology, cardiovascular and metabolic diseases, ophthalmology, infectious diseases and immunology with the aim of providing real and positive change for patients, the people they love and the professionals who care for them. Genentech in the United States is a fully owned subsidiary in the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, a major innovator in the Japanese therapeutic antibody market. Enspryng reduces risk of attack and is associated with improved recovery Exploratory analyses of the pivotal METEOROID trial suggest that in addition to significantly lowering the risk of relapse (AAN 2026), Enspryng was associated with improved recovery and reduced disability progression, which is critical to prevent permanent damage to the optic nerves, spinal cord or brain in myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). These findings reinforce Enspryng’s potential to become the first FDA-approved treatment for MOGAD, with a decision in the U.S.

Original title: “Roche presents new pivotal trial data across broad neuroimmunology portfolio at MSToronto2026 - BioSpace”

Hibsago’s approval—a functional cure for certain patients with a hepatitis B infection—further validates Ionis Pharmaceuticals’ antisense platform, which has already generated two other new drug approvals this year.

Original title: “GSK, Ionis win FDA nod for hepatitis B functional cure that controls virus after treatment”

A Kerala government hospital distributed generic SMA medicine bearing a clinical trial label. The switch has raised concerns about regulatory approval, bioequivalence and parental consent. The issue is particularly sensitive because SMA is a rare genetic disorder that progressively weakens muscles and can be fatal in its more severe forms. Treatment can improve survival and slow disease progression, but the cost of medicines has historically put them beyond the reach of many Indian families. Kerala has been an exception. Since 2022, it has been the only Indian state to provide free SMA therapy to eligible children, and currently around 200 patients are estimated to benefit from the programme. It was expanded into the Kerala United Against Rare Diseases (KARE) programme in 2023, providing approved medicines and specialised care to children with SMA under the age of 12. The programme uses a hub-and-spoke model to coordinate treatment and has established a patient registry to help monitor beneficiaries. The generic had the potential to substantially reduce the cost of treatment and make public funding programmes more sustainable. This week, Sree Avittom Thirunal (SAT) Hospital in Thiruvananthapuram started distributing generic risdiplam. Parents of the patients, however, noticed that the packaging carried the words “Physician Sample” and “For Clinical Trials Only”, prompting fears that their children were being given an experimental drug instead of the medicine they had been receiving. It modifies the processing of SMN2 messenger RNA, helping the body produce more survival motor neuron (SMN) protein. A deficiency of this protein causes the progressive muscle weakness associated with the disease. Treatment is most effective when started early, particularly in infancy, but older patients can also benefit.

Original title: “Kerala SMA treatment row: Natsmart's clinical trial label raises concerns - India Today”

Results support combination strategy for influenza vaccines

Original title: “Osivax reports phase 2b data for OVX836”