Drugs / ribitol
ribitol
also known as
BBP-418 · 353ZQ9TVDA · Adonit · Adonite · Adonitol · Meso-pentitol · Meso-ribitol · NSC-16868 · +1 more
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | muscular dystrophy, limb-girdle, autosomal recessive 29 | 2026-03-30 | “today announced the submission of its New Drug Application (NDA) to the FDA for oral BBP-418” bridgebio.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial | ||||||
| Phase 2 | NCT04800874 | Feb 2021 → Nov 2026 expected | autosomal recessive limb-girdle muscular dystrophy type 2I | ML Bio Solutions, Inc. | Active not recruiting | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT07678775 | Jun 2026 → Oct 2030 expected | autosomal recessive limb-girdle muscular dystrophy type 2I | ML Bio Solutions, Inc. | Active not recruiting | Met primary |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT05775848 Comparator | May 2023 → Jul 2027 expected | autosomal recessive limb-girdle muscular dystrophy type 2I | ML Bio Solutions, Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 16
| Date | Issuer | Release |
|---|---|---|
| 2026-05-27 | Eidos Therapeutics, a BridgeBio company | Regulatory BridgeBio Announces FDA Acceptance and Priority Review of NDA for BBP-418 for LGMD2I/R9 bridgebio.com ↗
today announced the FDA has accepted for filing its New Drug Application (NDA) with Priority Review for oral BBP-418 for the treatment of individuals living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). |
| 2026-05-07 | Eidos Therapeutics, a BridgeBio company | Regulatory BridgeBio Reports First Quarter 2026 Financial Results and Corporate Updates bridgebio.com ↗ |
| 2026-03-30 | Eidos Therapeutics, a BridgeBio company | Regulatory BridgeBio Submits NDA to FDA for BBP-418 for Individuals Living with LGMD2I/R9 bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a biopharmaceutical company focused on developing medicines for genetic conditions, today announced the submission of its New Drug Application (NDA) to the FDA for oral BBP-418 for the treatment of individuals living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). |
| 2026-03-11 | Eidos Therapeutics, a BridgeBio company | Results BBP-418 Demonstrates Consistent Efficacy and Favorable Safety Profile in Phase 3 FORTIFY Interim Analysis in LGMD2I/R9 bridgebio.com ↗ |
| 2026-03-04 | Eidos Therapeutics, a BridgeBio company | Results BridgeBio to Present Additional Data from the Phase 3 FORTIFY Trial at the 2026 MDA Clinical & Scientific Conference bridgebio.com ↗
Interim Analysis from Ongoing Phase 3 FORTIFY Study of BBP-418 for Patients with LGMD2I/R9 Meets Efficacy Endpoints |
| 2026-01-12 | Eidos Therapeutics, a BridgeBio company | Results BridgeBio Announces Commercial Progress, Program Updates, and 2026 Milestones at the 44th Annual J.P. Morgan Healthcare Conference bridgebio.com ↗ |
| 2025-10-27 | Eidos Therapeutics, a BridgeBio company | Results BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study bridgebio.com ↗ |
| 2024-06-18 | Eidos Therapeutics, a BridgeBio company | Regulatory bridgebio pharma surpasses interim analysis enrollment target and receives u.s. fda rare pediatric disease designation for bbp-418, a potential treatment for limb-girdle muscular dystrophy type 2i/r9 (lgmd2i/r9) bridgebio.com ↗
Rare Pediatric Disease Designation for BBP-418 highlights that LGMD2I/R9 is a rare disease with serious manifestations, which primarily impacts children, and if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher |
| 2023-10-09 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma shares positive long-term data from an ongoing phase 2 study, which support the potential use of glycosylated alpha-dystroglycan (⍺dg) levels as a surrogate endpoint in limb-girdle muscular dystrophy type 2i/r9 (lgmd2i/r9) bridgebio.com ↗
The new long-term data remains consistent with earlier data from the Phase 2 study showing a well-tolerated safety profile and encouraging preliminary efficacy. |
| 2023-05-04 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma reports first quarter 2023 financial results and business update bridgebio.com ↗
Shared preliminary findings on novel bioassay measuring glycosylated alpha-dystroglycan (αDG) in patients with limb-girdle muscular dystrophy type 2I (LGMD2I) and 15-month results from ongoing Phase 2 study of BBP-418 for LGMD2I; continuing to progress towards 2023 initiation of a global Phase 3 registrational clinical trial of BBP-418 for LGMD2I |
| 2023-03-21 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma shares preliminary findings on novel bioassay measuring glycosylated alpha-dystroglycan (⍺dg) in patients with limb-girdle muscular dystrophy type 2i (lgmd2i) bridgebio.com ↗
BridgeBio also shared 15-month results from its ongoing Phase 2 study, which showed a doubling of glycosylated ⍺DG in LGMD2I patients treated with BBP-418 |
| 2022-10-14 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents 12-month results from phase 2 study of bbp-418 in limb-girdle muscular dystrophy type 2i (lgmd2i) bridgebio.com ↗ |
| 2022-05-05 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma reports first quarter 2022 financial results and business update bridgebio.com ↗
Released positive data from Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i), which demonstrated a 43% increase in the ratio of glycosylated alpha-dystroglycan (aDG) to total aDG from baseline |
| 2022-03-14 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma announces positive phase 2 data for limb-girdle muscular dystrophy type 2i (lgmd2i) bridgebio.com ↗
today announced positive data from the Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i). |
| 2021-10-12 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma announces progress in its kras portfolio, new gene therapy programs, and updates on advancements across its r&d pipeline targeting genetic diseases and cancers bridgebio.com ↗
BridgeBio will review Phase 1 data in which BBP-418 was shown to be well-tolerated in healthy volunteers. |
| 2021-09-15 | Eidos Therapeutics, a BridgeBio company | Regulatory bridgebio pharma receives fda fast track designation for investigational therapy for the treatment of limb-girdle muscular dystrophy type 2i (lgmd2i) bridgebio.com ↗
today announced that the United States Food and Drug Administration (FDA) granted Fast Track designation for the investigation of BBP-418 as a treatment option for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i). |
All press releases naming this drug 20 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ribitol | “BBP-418 (ribitol)” NCT07678775 ↗1“BBP-418 (ribitol)” NCT05775848 ↗ |
| Known as | 353ZQ9TVDA | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | Adonit | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | Adonite | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | Adonitol | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | BBP-418 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07678775 ↗ |
| Known as | Meso-pentitol | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | Meso-ribitol | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | NSC-16868 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Known as | Ribo-pentitol | ChEMBL registry synonym — accepted as the source's own label CHEMBL3137744 ↗ |
| Modality | Small molecule | — CHEMBL3137744 ↗ |
| Route | Oral | “The granules will be reconstituted in water for oral administration.” NCT05775848 ↗ |