An FDA decision, a weekly pen, and Medicare’s one window

Europe’s drug regulator committee recommended approval on September 17 for gefurulimab (brand name Klygefa), a weekly injection you give yourself with a pre-filled pen or syringe, as an add-on treatment for adults with AChR-positive generalized MG. Then at the Scientific Session, a neurologist breaking down the trial results noted that 3 people on the drug needed an MG-related hospital stay versus 12 on placebo. The company says it’s also under review in the U.S., but no U.S. decision date has been announced.

This is the one-day research meeting where next year’s patient news gets its start. The agenda covered things like using a breathing-support device to monitor respiratory muscle weakness, what a “meaningful improvement” actually looks like on patient-reported scales, treatment results in seronegative MG, and several CAR-T cell therapy talks.

Regeneron’s cemdisiran is a quarterly injection (four times a year) for adults with AChR-positive generalized MG. If approved, it would be the first siRNA — a newer kind of medicine that turns down production of a protein involved in the complement system — for MG. In the main trial, people on it improved about 2.3 points more than placebo on the MG-ADL scale after 24 weeks. The FDA is reviewing it on an accelerated (priority) timeline. Nothing is approved yet, so this is a “watch this space,” not a “ask for it Monday.”

Per trade reporting on CMS’s final announcement, the 2027 Part D deductible rises from $615 to $700 and the yearly out-of-pocket cap rises from $2,100 to $2,400. One catch for us: the cap applies to Part D drugs, not Part B drugs, so ask your pharmacy or infusion center which bucket your treatment falls into. There’s also a Medicare Prescription Payment Plan that spreads your drug costs into monthly installments. Worth a half hour with the plan finder before December 7.

If open enrollment season has you doing math you didn’t sign up for, MGFA’s assistance page (and its Food Support Program) is worth bookmarking before you need it.

Flu season is here and this is the plain-language version of a question that comes up every fall. It explains that most vaccines are considered safe for people with MG, that live vaccines (like the nasal flu spray) should be avoided if you’re on immunosuppressants, and that inactivated flu and COVID shots are recommended. Your neurologist still gets the final say on timing and which shots are right for you.

Cartesian Therapeutics reported on September 29 that people who got a second round of its mRNA-based CAR-T therapy (Descartes-08) improved by an average of 6.6 points on the MG-ADL scale a year later, with no cytokine release syndrome or neurotoxicity reported in that group. The big test is the Phase 3 AURORA trial — about 100 AChR-positive patients getting six weekly outpatient infusions without chemotherapy first. Results are expected in early 2027. This is company-reported data from a small group, so read it as “promising,” not “proven.”

A new analysis of a China-based Phase 3 trial (114 adults) found about 42% of people on telitacicept hit minimal symptom expression — an MG-ADL score of 0 or 1 — at some point over 48 weeks, and once they got there they stayed there most of the time. This is a post-hoc analysis reported by the company, and the drug isn’t approved in the U.S. yet; the global trial I mentioned last month reads out in the first half of 2027.