Key Takeaway
FORTIFY is listed as active, not recruiting. The sponsor has reported interim results and regulatory review, but a trial record and an application under review do not establish treatment availability.
Trial and Study Record
BBP-418 (Ribitol) FORTIFY Trial in LGMD2I/LGMDR9
Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9, with trial details and separately dated sponsor-reported regulatory updates.
TrialTherapy DevelopmentTrial ReadinessFamilies
Why this record matters
- The record describes randomized, placebo-controlled treatment, with functional and safety outcomes through 36 months and estimated study completion in July 2027.
- BridgeBio's 2026-08-10 update reports positive 12-month interim findings; these sponsor-reported results are separate from the registry, which has no posted results.
- That sponsor update reiterates FDA acceptance and Priority Review on 2026-05-27 with a 2026-11-27 target action date and describes launch as conditional on FDA approval.
- ClinicalTrials.gov marks this study as having no expanded-access program. That field describes this record; it is not a general promise about future access policy.
- BridgeBio stated on 2026-08-10 that it intends to initiate studies in people younger than 12 in the first half of 2027. This is sponsor intent, not enrollment, access, approval, or a protocol for children.
Eligibility and participation notes
- The record specifies ages 12 to 60 years, with EU enrollment restricted to 18 to 60 years, and genetically confirmed, clinically affected LGMD2I/LGMDR9.
- Required weight above 30 kg and ability to complete study procedures, including biopsies; additional exclusions apply.
- The protocol excludes use of ribose or another sugar-alcohol-containing supplement within 90 days before screening.
- These are selected protocol criteria for a study that is not recruiting, not an individual eligibility assessment.
Limits and cautions
- The registry was last updated in September 2025 and can lag sponsor announcements. Ongoing follow-up does not mean interim results are unavailable.
- FDA acceptance and Priority Review are not approval. A target action date is not a guaranteed approval or availability date.
- Listed countries describe study sites, not confirmed places to enroll. Sponsor reporting is not independent clinical appraisal.
- The 90-day supplement exclusion and age limits are criteria from the current non-recruiting study and should not be presented as future pediatric policy.
Primary Sources
Direct links for verification
Primary registry or publication source used for the structured study summary on this page.
Sponsor announcement dated 2026-05-27 with a 2026-11-27 PDUFA target date. Priority Review is not approval.
Sponsor update dated 2026-08-10 reiterates the November target date, describes launch as conditional on FDA approval, and states an intention to begin under-12 studies in the first half of 2027.
Priority Review changes the review timetable, not the evidence standard required for approval.
Related Content
Related routes inside the site
Study Record
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.
Paper Summary
Quantitative Measurement of Glycosylated Alpha-Dystroglycan as a Biomarker for Disease Severity in LGMD2I/R9
Prospective 12-month observational study of muscle-biopsy glycosylated alpha-dystroglycan measurements in genetically confirmed LGMD2I/R9.
Study Record
Study of BBP-418 in Patients With LGMD2I
Phase 2 open-label ribitol study in FKRP-related LGMD2I/LGMDR9 that provides the main early clinical development context for the BBP-418 program.