Sunday, 20 September 2026

First Sanfilippo A gene therapy, Azetukalner depression trials paused, Xervyteg GvHD approval refused

1. 01

   FDA approves first gene therapy for Sanfilippo syndrome type A

   The US Food and Drug Administration has approved Fayuvi, the first gene therapy for the rare, progressive neurological condition Sanfilippo syndrome type A. Ultragenyx Pharmaceuticals priced the treatment at $3.95 million. It is the company’s second gene therapy approval since August, with sellable drug review vouchers granted alongside both approvals. Ultragenyx shares rose by double digits following the decision, although analysts do not expect either therapy to become a blockbuster product.

   via BioPharma Dive

2. 02

   Neuropsychiatric events pause azetukalner depression trial recruitment

   Xenon Pharmaceuticals paused enrolment in trials of azetukalner for depression after neuropsychiatric adverse events emerged that had not appeared in earlier depression testing. The company described the unspecified events as mild to moderate, short in duration and reversible, and said they were consistent with the drug’s known safety profile and mechanism. Trials will continue among currently enrolled participants, with data expected in early 2027, while Xenon examines potential dosing modifications. The pause came as the FDA began reviewing azetukalner for focal seizures, with Xenon maintaining confidence in its efficacy and safety profile in epilepsy. Xenon shares fell by as much as 30% following the announcement.

   via BioPharma Dive

3. 03

   EMA confirms refusal of Xervyteg for acute gastrointestinal GvHD

   The European Medicines Agency has confirmed its recommendation to refuse marketing authorisation for Xervyteg after re-examining its initial opinion. The pooled allogeneic faecal microbiota therapy was intended for adults with acute graft-versus-host disease affecting the gut when corticosteroids and ruxolitinib had not worked well enough. Its main study involved 67 adults but lacked a comparison group, was open label and allowed concurrent treatments, making Xervyteg’s effects difficult to distinguish from other factors. The EMA concluded it was still not possible to establish the medicine’s safety, effectiveness or degree of benefit, and that its benefits did not outweigh its risks. MaaT Pharma said the decision would not affect patients currently enrolled or receiving treatment in clinical trials or compassionate use programs.

   via European Medicines Agency Human Medicines

4. 04

   FDA clears Neptune’s Triton robotic system for colon procedures

   The US Food and Drug Administration cleared Neptune Medical’s Triton 1 robotic endoscopy system for diagnostic and therapeutic procedures in the colon. The system aims to provide greater endoscope stability and control during colonoscopy, including screening, surveillance, endoscopic mucosal resection and endoscopic submucosal dissection. In Neptune’s 50-patient CARE 1 study, Triton met its primary endpoints with no adverse events and achieved an adenoma detection rate of 54.2%, compared with an industry threshold of 35%. The company said it would use this generation to expand the evidence base and gather endoscopist feedback to shape its next platform.

   via MedTech Dive

5. 05

   National Guide implementation workshops reach Orange healthcare teams

   Healthcare professionals and community representatives in Orange have met to discuss locally tailored implementation of preventive care for Aboriginal and Torres Strait Islander peoples. The event brought together staff from general practices and Aboriginal Community Controlled Health Organisations as part of 13 National Guide workshops across Australia, delivered by NACCHO and the RACGP. The fourth edition of the guide, released in 2024, provides practical guidance across 21 topics, including child health, preconception and pregnancy care, diabetes, racism and climate change. Orange marked the halfway point of the six-month workshop series, with the next event scheduled for Shepparton on 30 September.

   via RACGP newsGP

6. 06

   New law enables publication of individual medical practitioners’ fees

   The Health Legislation Amendment (Improving Choice and Transparency for Private Health Consumers) Bill 2026 passed this week, allowing the government to publish individual medical practitioners’ fees and likely out-of-pocket costs through the Medical Costs Finder. A separate specialist affordability consultation paper presented six options to curb fees, including caps based on individual MBS items or providers’ annual billing. A referral reform proposal would require GPs to include a link to the Medical Costs Finder on referrals. The department acknowledged that a lower cap based on aggregate billing could increase the risk of providers charging excessive fees entirely outside Medicare. No specialist fee cap has been introduced, with the Medical Costs Finder changes the only guaranteed reform so far.

   via The Medical Republic

MDDR is a news digest for medical professionals. It is not medical advice and should not be relied on for clinical decision-making.