Lonafarnib
Orphan DrugFDA Approved, EMA Approved
Contents
Description
Lonafarnib is a farnesyltransferase inhibitor indicated for treatment of Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies. It is the first approved treatment for these ultra-rare, fatal genetic conditions characterized by premature aging.
Indications & Therapeutic Use
Hutchinson-Gilford Progeria Syndrome, Progeroid Laminopathies
Linked Diseases:
progeria
D011371
Global Availability (5 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Lonafarnib
| Generic Name | lonafarnib |
| Brands | 1 brand available |
| Active Ingredient | lonafarnib |
| Drug Class | Hutchinson-Gilford Progeria Syndrome |
| Manufacturer | Eiger BioPharmaceuticals |
| Dosage Forms | Oral capsules, 50mg and 75mg |
| Medical Code | L01XX65 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 21 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00916747 |
| Countries | 5 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes