FKRP Reference Public rare-disease knowledge base

A clearer path through rare-disease information.

Menu

Field Update

Therapeutic Pipeline

These selected FKRP programs show how clinical development is progressing. Each entry separates the trial record from dated sponsor updates, so study status, preliminary results, and regulatory review can be read in context.

Gene therapyRibitolTrial statusRegulatory context

Program snapshots

Selected programs and their evidence status

Active, not recruiting · Phase 1

ATA-100 gene therapy

FKRP-targeted AAV9 gene-transfer study with six participants. The record lists primary completion in February 2025 and estimated overall completion in September 2029 for long-term follow-up.

Status note: ClinicalTrials.gov NCT05224505, last updated 2025-10-06 and checked 2026-09-05, lists Phase 1 and active, not recruiting.

Caution: Atamyo's 2025-04-03 update describes dose finding within a broader phase 1b/2b program. Its early reported findings do not establish clinical benefit or change the Phase 1 label of this registry record.

Primary source Internal record

Active, not recruiting · Phase 1/2

AB-1003 gene therapy

Placebo-controlled, sequential-dose FKRP gene-therapy study in adults. The current record lists nine actual participants and long-term follow-up.

Status note: ClinicalTrials.gov NCT05230459, updated 2026-08-26 and checked 2026-09-05, lists ages 18 to 65 and active, not recruiting.

Caution: The registry has no posted results and no expanded-access program. Study locations and continued follow-up do not imply enrollment or access.

Primary source Internal record

Active, not recruiting · Phase 3

BBP-418 phase 3 FORTIFY

Randomized, placebo-controlled oral ribitol study in LGMD2I/LGMDR9. BridgeBio has reported positive 12-month interim findings while longer follow-up continues.

Status note: ClinicalTrials.gov NCT05775848, last updated 2025-09-29 and checked 2026-09-05, lists Phase 3 and active, not recruiting. Estimated completion is July 2027.

Caution: BridgeBio's 2026-08-10 update reiterates FDA acceptance and Priority Review on 2026-05-27, with a 2026-11-27 target action date. It describes launch as conditional on FDA approval. A target date is not a guarantee of approval or availability.

Primary source Internal record

Active, not recruiting · Phase 2

BBP-418 phase 2 study

Open-label sequential-dose study that provides important context for how the ribitol program developed before the phase 3 trial.

Status note: ClinicalTrials.gov NCT04800874, updated 2026-07-21 and checked 2026-09-05, lists 14 participants and estimated completion on 2026-11-01.

Caution: The record remains active, not recruiting, with no posted results. Its small open-label design limits efficacy conclusions; progression to Phase 3 does not itself demonstrate benefit.

Primary source Internal record

Terminated · Phase 3 history

Deflazacort history

Earlier corticosteroid program in FKRP-related LGMD2I that ended early and now functions mainly as historical trial context.

Status note: ClinicalTrials.gov NCT03783923, last updated 2022-06-27 and checked 2026-09-05, includes posted results and an early-termination explanation.

Caution: Eleven participants enrolled, but only one had an evaluable week-26 change for the primary stair-climb outcome. This terminated study does not establish routine benefit.

Primary source Internal record

Reading Rules

How this site handles moving targets without overstating them

Trial record

ClinicalTrials.gov records report study phase, status, eligibility, and dates submitted by study teams. Check the last update because records can lag later announcements.

Sponsor update

Sponsor releases can provide more recent developments. Their dates and source matter, and interim results can be available while a trial continues.

Peer-reviewed evidence

Full methods, outcome data, and limitations allow closer appraisal of benefit and harm. A press release and a publication are different kinds of evidence.

Regulatory event

FDA explains that Priority Review changes the review timetable without lowering the evidence standard for approval. A filing or review milestone does not settle treatment availability.

Current interpretation

Read each milestone alongside its source date.

On 2026-09-05, the linked records continued to list ATA-100, AB-1003, and both BBP-418 studies as active, not recruiting. BridgeBio's 2026-08-10 update described BBP-418 as under regulatory review with potential launch after FDA approval. These sources do not establish approved treatment access, and this page does not assess individual eligibility.

Use exact datesSeparate sponsor from peer reviewPriority Review is not approval
Status discipline

Write “active, not recruiting” literally

Follow-up can continue after enrollment closes and after interim results are announced. Listed sites do not mean a study is accepting new participants.

Future-study statement

Sponsor intent is not an open pediatric study

BridgeBio stated on 2026-08-10 that it intends to begin under-12 BBP-418 studies in the first half of 2027. No enrollment, access, approval, or future protocol is inferred from that statement.

Evidence discipline

Biomarker change is not the same as clinical benefit

Biomarker findings need to be considered alongside function, safety, study design, and follow-up when interpreting potential benefit.

Selected Sources

Reference trail for this page

BridgeBio second-quarter 2026 update

Sponsor update dated 2026-08-10 reiterates the May acceptance and November target date, discusses interim results, and states an intention to begin under-12 studies in the first half of 2027. This is not enrollment, access, approval, or a peer-reviewed appraisal.

Continue Reading

Use these routes to go deeper

Evidence Layer

Trials and Studies

Structured records for current and historical studies, registries, and trial entries.

Audience Guide

For Researchers

Research-facing route through terminology, evidence capture, and current field organization.

Core Reference

Monitoring and Care

Use alongside the pipeline when families ask what still matters clinically while trials evolve.

Practical Support

Resources

Practical support layer for registry participation, emergency prep, and care navigation.