Floatz Rating
B57/100
Confidence
Indicative
Sponsor
Ozlem Goker-Alpan
Modality
Small molecule
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
42Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06614894CT.gov An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III | — | Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Mucopolysaccharidosis Type 3
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Ambroxol (this asset) | Ozlem Goker-Alpan | P2/3 | B · 57 |
| Amidase, Sulfoglucosamine Sulf- | Shire | P2 | B |
| Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) | Ultragenyx Pharmaceutical Inc | P2/3 | B |
| Hydroxyurea | Masonic Cancer Center, University of Minnesota | P2 | B |
| Alemtuzumab | Masonic Cancer Center, University of Minnesota | P2 | B |
| Melphalan | Masonic Cancer Center, University of Minnesota | P2 | B |
| Clofarabine | Masonic Cancer Center, University of Minnesota | P2 | B |
| Hgt-1410 | Shire | P2 | CCC |
| Duoc-01 | Joanne Kurtzberg, MD | P1 | CC |
Other indications for Ambroxol
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Parkinson Disease | — | P3 | BB · 65 |
| GBA1-Related Parkinson Disease, Susceptibility | — | P2 | BB · 64 |
| Mucus Inspissation Of Respiratory Tract | — | P3 | BB · 64 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
- Amidase, Sulfoglucosamine Sulf-ShireB
- Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying)Ultragenyx Pharmaceutical IncB
- HydroxyureaMasonic Cancer Center, University of MinnesotaB
- AlemtuzumabMasonic Cancer Center, University of MinnesotaB
- MelphalanMasonic Cancer Center, University of MinnesotaB
Citation
Floatz Terminal. Ambroxol in Mucopolysaccharidosis Type 3. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/ambroxol-mucopolysaccharidosis-type-3
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