sirolimus
Orphan DrugFDA Approved, EMA Approved
Description
Sirolimus is an mTOR inhibitor with immunosuppressive and anti-angiogenic properties. It has orphan drug designation for treatment of vascular anomalies and has been used in managing complex vascular malformations such as those in Cobb syndrome. The medication inhibits abnormal cell proliferation and vascular growth.
Indications & Therapeutic Use
Vascular anomalies, lymphangioleiomyomatosis, organ transplant rejection prophylaxis
Linked Diseases:
Mediastinal fibrosis
J98.5C536136
Global Availability (13 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
sirolimus
| Generic Name | sirolimus |
| Brands | 1 brand available |
| Active Ingredient | sirolimus |
| Drug Class | Vascular anomalies |
| Manufacturer | Pfizer |
| Dosage Forms | Oral tablet 0.5mg/1mg/2mg, oral solution 1mg/mL |
| Medical Code | L04AA10 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 5 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT02110069 |
| Countries | 13 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations14 Validated Nodes