Field Update
This page tracks the current FKRP pipeline with one rule above all others: dates and evidence level stay visible. Trial status, sponsor-reported updates, regulatory events, and peer-reviewed conclusions are not interchangeable.
Current Programs
Active, not recruiting · Phase 1
FKRP-targeted AAV9 gene-transfer program with long-term follow-up still underway. This is an early safety and biomarker program, not established efficacy.
Status note: ClinicalTrials.gov record NCT05224505 last updated 2025-10-06.
Caution: Use early safety and biomarker language only. The public record does not support claiming proven clinical benefit as of 2026-04-21.
Active, not recruiting · Phase 3
Late-stage oral ribitol program in LGMD2I/LGMDR9. This is the most advanced current FKRP-directed interventional program found in the sweep.
Status note: ClinicalTrials.gov record NCT05775848 last updated 2025-09-29.
Caution: BridgeBio announced an NDA submission on 2026-03-30, but a regulatory filing is not the same as approval or completed trial evidence.
Active, not recruiting · Phase 2
Open-label sequential-dose study that provides important context for how the ribitol program developed before the phase 3 trial.
Status note: ClinicalTrials.gov record NCT04800874 last updated 2025-05-08.
Caution: This remains an ongoing interventional study. Sponsor summaries and phase progression should be kept distinct from final peer-reviewed efficacy conclusions.
Terminated · Phase 3 history
Earlier corticosteroid program in FKRP-related LGMD2I that ended early and now functions mainly as historical trial context.
Status note: ClinicalTrials.gov record NCT03783923 last updated 2022-06-27.
Caution: Useful for understanding endpoint choice and field history, but not a current disease-modifying pipeline signal.
Reading Rules
ClinicalTrials.gov is the most stable source for phase, status, enrollment, and posted update dates. Use it before sponsor marketing language.
Press releases and conference summaries are useful for field awareness, but they should be labeled as sponsor-reported or preliminary.
Peer-reviewed publications carry more weight for interpretation than interim promotional summaries, especially when efficacy claims are involved.
A filing, fast-track designation, or review milestone is not the same as approval and should never be written as if treatment availability is settled.
Current interpretation
As of 2026-04-21, the main active FKRP programs in public records are ATA-100 gene therapy and the BBP-418 ribitol studies. The ribitol track has progressed furthest commercially, including a sponsor-announced NDA filing on 2026-03-30, but that does not collapse trial status, regulatory review, and approval into one event.
Ongoing studies are still ongoing, even when enrollment is closed. That matters for families reading field updates.
The page should keep early signals visible without upgrading them into claims the literature does not yet support.
Selected Sources
Primary source for the current ATA-100 status and trial framing.
Primary source for the current phase 3 ribitol record.
Primary source for the phase 2 ribitol study.
Regulatory filing announcement dated 2026-03-30. This is sponsor communication, not approval.
Useful for field context, but should be labeled as preliminary sponsor-linked reporting.
Historical record showing a terminated FKRP-linked steroid program.
Continue Reading
Evidence Layer
Structured records for current and historical studies, registries, and trial entries.
Audience Guide
Research-facing route through terminology, evidence capture, and current field organization.
Core Reference
Use alongside the pipeline when families ask what still matters clinically while trials evolve.
Practical Support
Practical support layer for registry participation, emergency prep, and care navigation.