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Oxlumo (LUMASIRAN)
Oxlumo (generic name: LUMASIRAN) is a Oligonucleotide drug developed by Alnylam Pharmaceuticals. It is currently FDA-approved (first approved 2020) for Primary hyperoxaluria, type I.
Oxlumo works by binding to and silencing the mRNA that produces the enzyme Hydroxyacid oxidase 1, which is overactive in patients with Primary hyperoxaluria, type I.
At a glance
| Generic name | LUMASIRAN |
|---|---|
| Sponsor | Alnylam Pharmaceuticals |
| Target | Hydroxyacid oxidase 1 (HAO1) mRNA |
| Modality | Oligonucleotide |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2020 |
| Annual revenue | 200 |
Mechanism of action
Lumasiran reduces levels of glycolate oxidase (GO) enzyme by targeting the hydroxyacid oxidase ( HAO1) messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, substrate for oxalate production. As the GO enzyme is upstream of the deficient alanine:glyoxylate aminotransferase (AGT) enzyme that causes PH1, the mechanism of action of lumasiran is independent of the underlying AGXT gene mutation. OXLUMO is not expected to be effective in primary hyperoxaluria type (PH2) or type (PH3) because its mechanism of action does not affect the metabolic pathways causing hyperoxaluria in PH2 and PH3.
Approved indications
- Primary hyperoxaluria, type I
Common side effects
- Injection site reaction
- Abdominal pain
- Erythema
- Pruritus
- Swelling
Key clinical trials
- BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
- A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PHASE3)
- A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (PHASE3)
- Lumasiran in Hyperoxalaemic Patients on Haemodialysis (PHASE2)
- A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (PHASE3)
- A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels (PHASE2)
- An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1 (PHASE2)
- Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
Patents
| Patent | Expiry | Type |
|---|---|---|
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Oxlumo CI brief — competitive landscape report
- Oxlumo updates RSS · CI watch RSS
- Alnylam Pharmaceuticals portfolio CI
Frequently asked questions about Oxlumo
What is Oxlumo?
How does Oxlumo work?
What is Oxlumo used for?
Who makes Oxlumo?
What is the generic name of Oxlumo?
When was Oxlumo approved?
What development phase is Oxlumo in?
What are the side effects of Oxlumo?
What is Oxlumo's annual revenue?
What does Oxlumo target?
Related
- Target: All drugs targeting Hydroxyacid oxidase 1 (HAO1) mRNA
- Manufacturer: Alnylam Pharmaceuticals — full pipeline
- Therapeutic area: All drugs in Metabolic
- Indication: Drugs for Primary hyperoxaluria, type I
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing