Osilodrostat
An orphan drug for Cushing's syndrome — the imidazole acetic acid backbone on routine supply
Osilodrostat — project profile
- Therapeutic area
- Endocrine (Cushing's syndrome)
- Mechanism
- 11β-hydroxylase (CYP11B1) inhibitor blocking the final step of cortisol synthesis
- Commercial status
- Marketed (US 2020 · EU 2020 · China 2024)
- Originator
- Recordati Rare Diseases
- Brand name
- Isturisa
Why this project, right now
Only publicly disclosed facts: regulatory approvals, clinical data, supply-chain moves
- ✓The FDA approved Isturisa in March 2020 for adults with Cushing's disease for whom pituitary surgery is not an option or has not been curative; in April 2025 the indication was expanded to endogenous hypercortisolemia in Cushing's syndrome.
- ✓The EU authorised it in January 2020 and China in September 2024 — all three major markets are live, so demand is steady rather than spiky.
- ✓As an orphan drug the patient population is small but treatment is long-term: volumes per product are modest, yet supply interruption is barely tolerable — a solid base load for long-term contracts.
- ✓The skeleton is small (imidazole acetic acid plus a chiral amine) with a well-defined starting material, where China-based supply wins clearly on both cost and lead time.
Public regulatory and clinical milestones
| Date | Event |
|---|---|
| 2020-01 | EU authorises Isturisa |
| 2020-03-06 | FDA approves Isturisa for Cushing's disease |
| 2024-09 | Approved in China |
| 2025-04 | FDA expands the indication to endogenous hypercortisolemia in Cushing's syndrome |
Key intermediate list
1 key intermediates
| CAS No. | Chemical name | Fragment role | Action |
|---|---|---|---|
| 3251-69-2 | 4-Imidazoleacetic acid hydrochloride | Imidazole acetic acid backbone starting material (hydrochloride salt) Process note：A commodity-grade backbone — price and lead time can be locked under an annual framework volume |
How we support this project
A routinely supplied item — available under an annual framework agreement with locked pricing and lead time
CoA plus assay and related-substance methods provided; customer audits and supplier qualification filings supported
Starting-material justification and process descriptions available for your formulation registration
Common capabilitiesThe same delivery capability backs every campaign project
- ✓Route design and process development: hands-on scale-up experience with fluorinated, chiral, boronate and heterocyclic fragments at partner plants
- ✓Scale continuity: gram-scale validation → hundred-gram → kilogram → commercial, without a route change
- ✓Analytics: chiral ee, related substances, residual solvents, elemental impurities and genotoxic-impurity assessment to ICH conventions
- ✓Registration support: DMF feasibility assessment, starting-material justification, stability study design
- ✓Confidentiality: customer projects run under NDA; no cross-disclosure between plants and end customers
Supply scope and compliance note
Most of the target drugs above are originator products still under patent. The intermediates listed here are supplied for research use, and for markets where the patent has expired or a valid licence is in place. The buyer is responsible for confirming patent status and registration requirements in the target market and for the resulting compliance obligations. Third-party brand names and trademarks belong to their respective owners and are referenced here only as a technical cross-reference.
Intermediate lists (CAS and chemical names) are taken from the Division II 2026 Key Projects source sheet. Regulatory and clinical information comes from originator press releases and public FDA material. Fragment roles are neutral descriptions derived from the chemical structure and do not represent the originator patent route. Chinese drug names are common industry transliterations, not official registered names.
Data version 2026-09