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Sanfilippo syndrome type A: the approved drugs compared for prescribers
Fayuvi side by side on what each first fill waits on, what the label requires you to monitor and what an insurer asks to see, with FDA decisions, label changes and open trials for sanfilippo syndrome type A.
The sanfilippo syndrome type A prescribing guide
One page per drug with the dispensing gate, prior authorization documentation, dosing, monitoring and interactions, each line sourced.
The drugs side by side
Each cell is a summary of that drug's prescribing guide, which carries the full detail and a source on every line. Insurer thresholds are one public policy shown as an example; plans differ.
| Fayuvirebisufligene etisparvovec-hopf | |
|---|---|
| Label dated | October 1, 2026 |
| Starting dose | Prednisone or prednisolone 1.0mg/kg/day orally, continued for 8 weeks. |
| Dispensing gate | No REMS; administration at an Ultragenyx Qualified Treatment Center after anti-AAV9 total binding antibody titer below 1:100, baseline ALT, AST, GGT, bilirubin, platelets, PT and aPTT, infection control, no vaccines in the prior 30 days, a negative serum pregnancy test where applicable, and prednisone or prednisolone started the day before |
| Required monitoring | LFTs weekly for 2 weeks then every 2 weeks until 2 weeks after the steroid taper; platelets weekly for 4 weeks then monthly for 6 months; TMA, infusion reaction and malignancy vigilance; vector shedding precautions for 3 months |
| Example PA threshold | Cigna IP0849 example: no prior Fayuvi, MPS IIIA by SGSH variants or deficient sulfamidase, anti-AAV9 below 1:100, neurologist, medical geneticist or metabolic disease specialist, weight within 30 days, one lifetime dose with a 90-day approval |
| Support line | UltraCare, 1-888-756-8657, Gene Therapy Guides Monday to Friday 9 AM to 8 PM Eastern for benefits investigation, PA and appeal status, commercial Copay Assistance for drug and administration costs, and information on 501(c)(3) foundations |
- Label dated
- October 1, 2026
- Starting dose
- Prednisone or prednisolone 1.0mg/kg/day orally, continued for 8 weeks.
- Dispensing gate
- No REMS; administration at an Ultragenyx Qualified Treatment Center after anti-AAV9 total binding antibody titer below 1:100, baseline ALT, AST, GGT, bilirubin, platelets, PT and aPTT, infection control, no vaccines in the prior 30 days, a negative serum pregnancy test where applicable, and prednisone or prednisolone started the day before
- Required monitoring
- LFTs weekly for 2 weeks then every 2 weeks until 2 weeks after the steroid taper; platelets weekly for 4 weeks then monthly for 6 months; TMA, infusion reaction and malignancy vigilance; vector shedding precautions for 3 months
- Example PA threshold
- Cigna IP0849 example: no prior Fayuvi, MPS IIIA by SGSH variants or deficient sulfamidase, anti-AAV9 below 1:100, neurologist, medical geneticist or metabolic disease specialist, weight within 30 days, one lifetime dose with a 90-day approval
- Support line
- UltraCare, 1-888-756-8657, Gene Therapy Guides Monday to Friday 9 AM to 8 PM Eastern for benefits investigation, PA and appeal status, commercial Copay Assistance for drug and administration costs, and information on 501(c)(3) foundations
FDA decisions for sanfilippo syndrome type A
From the Trial Friend FDA decision calendar, which records each date from the sponsor's or the FDA's own announcement.
- September 17, 2026UX111 (Fayuvi, rebisufligene etisparvovec), BLA resubmission, UltragenyxApproved
Recent label changes
What changed in each label revision, from the FDA's approval letters and the labels' own revision lists.
- October 2026Fayuvi. The label was posted on DailyMed with an October 2026 revision date. The sections compared for this page match the September 2026 package insert, with no boxed warning and no contraindications. FDA label
- September 2026Fayuvi. In the approval letter, Ultragenyx committed to studies supporting an FDA-authorized AAV9 antibody test, with completion due December 31, 2028. FDA letter
- September 2026Fayuvi. The FDA approved Fayuvi for Sanfilippo syndrome type A on September 17th. Ultragenyx called it the first FDA-approved treatment for Sanfilippo syndrome type A and a standard full approval. Ultragenyx press release
- July 2025Fayuvi. The FDA's approval letter says a March 2026 resubmission answered its earlier action letter of July 7, 2025, which did not approve the first application. FDA letter
Open trials
Questions prescribers ask about Sanfilippo syndrome type A
Does Fayuvi require a REMS?
No. Fayuvi has no REMS. Its prescribing guide lists what does gate the first fill and its source.
What monitoring does Fayuvi require?
LFTs weekly for 2 weeks then every 2 weeks until 2 weeks after the steroid taper; platelets weekly for 4 weeks then monthly for 6 months; TMA, infusion reaction and malignancy vigilance; vector shedding precautions for 3 months.
What do insurers require before covering Fayuvi?
This is one public insurer policy, an example rather than every plan. Cigna IP0849 example: no prior Fayuvi, MPS IIIA by SGSH variants or deficient sulfamidase, anti-AAV9 below 1:100, neurologist, medical geneticist or metabolic disease specialist, weight within 30 days, one lifetime dose with a 90-day approval.
Which support program starts Fayuvi patients on therapy?
UltraCare, 1-888-756-8657, Gene Therapy Guides Monday to Friday 9 AM to 8 PM Eastern for benefits investigation, PA and appeal status, commercial Copay Assistance for drug and administration costs, and information on 501(c)(3) foundations.
A reference for licensed professionals, checked on November 9, 2026. Labels and plan policies change; the label on DailyMed and the plan's current policy govern. Not medical advice. For patients: the Sanfilippo syndrome type A page and the first 90 days pages for Fayuvi.