During clinical trials, severe infections reduced from 2 per year to 0.12-0.15 per year post-treatment, while moderate and severe bleeding episodes reduced from 1 to 0.16 per year. According to GlobalData’s Drugs database, Waskyra is just one of 24 cell and gene therapies (CGTs) that are currently approved for treating rare diseases ... During clinical trials, severe infections reduced from 2 per year to 0.12-0.15 per year post-treatment, while moderate and severe bleeding episodes reduced from 1 to 0.16 per year. According to GlobalData’s Drugs database, Waskyra is just one of 24 cell and gene therapies (CGTs) that are currently approved for treating rare diseases in the US. In clinical trials, 51% of patients were surgery free at year one. Of these patients, 14 out of 18 had still undergone zero surgeries after three years. There are now more than 1,500 cell and gene therapies for rare diseases in active clinical development globally. As the rise of CGTs drives the next stage of growth for orphan drugs, developers must confront the complexities of two of the most demanding areas of drug development in order to bring these advanced therapies to small, underserved patient populations. Despite the challenges, the landscape is evolving and maturing, with growing clinical pipelines and new product launches reshaping the outlook for rare disease treatment. Cell and gene therapies are fuelling new possibilities for rare diseases, turning a field defined by unmet need into one of the industry’s most...

Original title: “The rare disease revolution: The state of play for CGTs in orphan diseases | Sharp Services”

As Xenon Pharmaceuticals announced that azetukalner has reached the FDA for focal seizures, the company also disclosed the emergence of psychosis adverse events in psychiatric trials of the drug, triggering a temporary pause.

Original title: “Xenon tumbles on voluntary psychiatry hold, but analysts confident in $2B epilepsy opportunity”

12 new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended 12 medicines for approval at its September 2026 meeting.The committee recommended…, CHMP statistics Key figures from the September 2026 CHMP meeting are represented in the graphic below. © European Medicines Agency, CHMP statistics:…, Posit

Original title: “Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 September 2026”

New format simplifies administration for clinicians

Original title: “Japan approves prefilled syringe for GSK’s shingles vaccine”

The TREM2 stabilizer failed to show efficacy in a Phase 2 trial of patients with early-stage ALS. This development follows two late-stage trial failures reported by Novartis last week, plus the news that the pharma has paused multiple CAR T cell trials following patient deaths.

Original title: “Novartis halts TREM2 targeting ALS program in 4th trial stumble in a month”