Last reviewed · How we verify
Sialic Acid-Extended Release
Sialic Acid-Extended Release is a Small molecule drug developed by Hadassah Medical Organization. It is currently in Phase 2 development. Also known as: aceneuramic acid, UX001.
Sialic Acid-Extended Release is a small molecule intervention being studied for various myopathies, including Hereditary Inclusion Body Myopathy and GNE Myopathy. It is being investigated as a potential treatment in clinical trials, with the goal of evaluating its safety and efficacy in patients with these conditions.
-
Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Sialic Acid-Extended Release |
|---|---|
| Also known as | aceneuramic acid, UX001 |
| Sponsor | Hadassah Medical Organization |
| Modality | Small molecule |
| Phase | Phase 2 |
Approved indications
Common side effects
Key clinical trials
- Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (PHASE3)
- Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (PHASE3)
- A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment (PHASE2)
- An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (PHASE2)
- Efficacy of Sialic Acid GNE Related Thrombocytopenia (PHASE2)
- A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (PHASE2)
- Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Sialic Acid-Extended Release CI brief — competitive landscape report
- Sialic Acid-Extended Release updates RSS · CI watch RSS
- Hadassah Medical Organization portfolio CI
Frequently asked questions about Sialic Acid-Extended Release
What is Sialic Acid-Extended Release?
Who makes Sialic Acid-Extended Release?
Is Sialic Acid-Extended Release also known as anything else?
What development phase is Sialic Acid-Extended Release in?
Related
- Manufacturer: Hadassah Medical Organization — full pipeline
- Also known as: aceneuramic acid, UX001
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing