pharma · TTR-mediated Amyloidosis · Hypertension · ALNY
Alnylam Netherlands B.V.
Alnylam Netherlands is a pharma organization headquartered in Cambridge, USA. It trades on NYSE under ticker ALNY. Primary therapeutic focus areas include TTR-mediated Amyloidosis, Hypertension, Transthyretin Amyloidosis
Phase 2 · small molecule · PH1
Lumasiran (OXLUMO) is a small-molecule therapeutic developed by Alnylam Netherlands B.V. for Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production leading to kidney stone formation and progressive renal failure. The program, identified as ALN-GO1-002, is currently in
Internal code ALN-GO1-002
Lumasiran (OXLUMO) is a small-molecule therapeutic developed by Alnylam Netherlands B.V. for Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production leading to kidney stone formation and progressive renal failure. The program, identified as ALN-GO1-002, is currently in Phase 2 development with a latest milestone recorded on 25 April 2024. Lumasiran is administered via subcutaneous injection and targets the underlying metabolic dysfunction in PH1.
Notably, lumasiran sodium has already achieved FDA approval under the brand name OXLUMO (NDA 214103), indicating that clinical development has progressed beyond the Phase 2 stage documented in this profile. The approval represents a significant regulatory milestone for Alnylam in the rare disease space. The company is pursuing a monotherapy strategy without disclosed partnerships, focusing on addressing the substantial unmet medical need in PH1 patients who currently have limited treatment options.
The program's advancement to approval status reflects successful demonstration of efficacy and safety in clinical trials. The most recent milestone update in April 2024 suggests ongoing development activities, though specific details of this milestone are not yet disclosed. Lumasiran's approval marks an important therapeutic advance for PH1 patients, offering a novel mechanism to reduce oxalate production and potentially slow or halt disease progression.
Primary Hyperoxaluria Type 1 is a rare, life-threatening genetic disorder with severe unmet medical needs. Patients suffer from recurrent kidney stones, progressive chronic kidney disease, and systemic oxalate deposition (secondary hyperoxaluria), often progressing to end-stage renal disease requiring dialysis or transplantation. Current treatment options are limited to supportive care with pyridoxine (vitamin B6) in responsive patients and dietary management, leaving many patients without effective disease-modifying therapies. The disease affects both pediatric and adult populations, with significant morbidity and mortality if untreated.
Lumasiran's approval addresses a critical therapeutic gap by offering the first disease-modifying treatment targeting the root cause of oxalate overproduction. The market relevance is substantial given the orphan disease designation and the severe consequences of untreated PH1. The subcutaneous route of administration offers practical advantages for chronic management compared to oral alternatives.
Competitively, lumasiran enters a landscape with limited approved therapies specifically targeting PH1 pathophysiology. The program's advancement to approval demonstrates clinical efficacy and safety sufficient for regulatory acceptance. Commercial significance is driven by the rare disease population, potential for long-term chronic therapy, and the absence of curative alternatives short of liver transplantation. The orphan drug designation provides market exclusivity and pricing flexibility, supporting commercial viability despite the small patient population.
Drug Class: Small-molecule therapeutic for rare metabolic disease
Modality: Small molecule
Route of Administration: Subcutaneous injection
Indication: Primary Hyperoxaluria Type 1 (PH1)
Brand Name: OXLUMO
International Nonproprietary Name (INN): Lumasiran sodium
Mechanism of Action: Not yet disclosed in available facts
Molecular Target: Not yet disclosed in available facts
Related Therapies: Pyridoxine (vitamin B6) for PH1 management; liver transplantation for definitive but invasive treatment
First Approval: FDA approval achieved (NDA 214103); specific approval date not disclosed in facts
Patent Status: Not yet disclosed in available facts
Also known as: AGXT primary hyperoxaluria, PH1, glycolic aciduria, peroxisomal alanine-glyoxylate aminotransferase deficiency, primary hyperoxaluria caused by mutation in AGXT, primary hyperoxaluria type I
Prevalence: Point prevalence: 1-9 / 1 000 000 (Netherlands) — source: Orphanet, validated.
A rare disorder of glyoxylate metabolism characterized by the accumulation of oxalate due to a deficiency of the peroxisomal hepatic enzyme L-alanine: glyoxylate aminotransferase (AGT). Clinical presentation is variable, ranging from occasional symptomatic nephrolithiasis to nephrocalcinosis and end-stage renal disease with systemic involvement.
ClinicalTrials.gov lists 10 registered studies for Primary Hyperoxaluria Type 1 (AACT aggregate).
Phase breakdown: NA (4), PHASE2 (3), PHASE3 (2), PHASE1 (1)
Common investigational therapies:
Disease data sourced from MONDO Disease Ontology (MONDO:0009823), Orphanet — primary hyperoxaluria type 1, NCT02795325, NCT02830009, NCT03067142, NCT03655223, NCT04152200, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, Open Targets Platform (CC BY 4.0).
Phase 2 ongoing
Lumasiran Phase 2 development for PH1 ongoing as of profile documentation.
Latest milestone
Most recent program milestone recorded on 25 April 2024; specific details not yet disclosed.
FDA approval
Lumasiran sodium (OXLUMO) approved by FDA under NDA 214103; specific approval date not disclosed in facts.
The competitive landscape for PH1 therapeutics is limited, reflecting the orphan disease status and historically restricted treatment options. Lumasiran competes indirectly with supportive care approaches including pyridoxine (vitamin B6) therapy for responsive patients and dietary oxalate restriction, neither of which addresses the underlying genetic defect.
Within the documented competitor set, Diacomit (stiripentol, Biocodex) is listed as a Phase 3 competitor, though stiripentol is primarily indicated for seizure disorders and its relevance to PH1 treatment is unclear from available facts. Placebo is noted as a comparator in clinical trials rather than a true therapeutic competitor. Tango Therapeutics' TNG908 program is in Phase 2 development; its mechanism and indication are not disclosed, limiting competitive assessment.
Lumasiran's advancement to FDA approval positions it as a first-mover or early-mover in disease-modifying PH1 therapy. The small-molecule modality and subcutaneous administration distinguish it from potential future competitors in development. The orphan drug designation provides market exclusivity, reducing near-term competitive pressure. Future competition may emerge from other genetic or metabolic approaches targeting oxalate production or elimination, but no such programs are documented in the current competitive set.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| Diacomit 250 mg hard capsules, Matched Placebo will consist in caspules similar to the 250 and 500 mg capsules of stiripentol but with no active ingredient, Diacomit 500 mg hard capsules | Biocodex | small_molecule | phase_3 |
| Placebo | Alnylam Netherlands B.V. | small_molecule | phase_3 |
| TNG908, TNG908, TNG908, TNG908, MIDAZOLAM | Tango Therapeutics | small_molecule | phase_2 |
| Lumasiran | Alnylam Netherlands B.V. | small_molecule | phase_2 |
| LUMASIRAN SODIUM | — | Hydroxyacid oxidase 1 mRNA RNAi inhibitor | Approved |
| NEDOSIRAN SODIUM | — | L-lactate dehydrogenase A chain mRNA RNAi inhibitor | Phase 2 |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
United States (FDA): Lumasiran sodium (OXLUMO) has achieved FDA approval under NDA 214103. Sponsor listed as ALNYLAM PHARMS INC. Specific approval date and indication details are not yet disclosed in available facts.
European Medicines Agency (EMA): Regulatory status not yet disclosed in available facts.
Pharmaceuticals and Medical Devices Agency (PMDA, Japan): Regulatory status not yet disclosed in available facts.
National Medical Products Administration (NMPA, China): Regulatory status not yet disclosed in available facts.
Orphan Drug Designation: Not yet disclosed in available facts, though orphan status is typical for rare disease indications such as PH1.
Regulatory Pathway: FDA approval via standard NDA pathway; accelerated or breakthrough designation status not yet disclosed.
Lumasiran (OXLUMO) is used to treat Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production in the body, leading to kidney stones, kidney damage, and systemic oxalate deposition.
Yes, lumasiran sodium (OXLUMO) has received FDA approval under NDA 214103, sponsored by ALNYLAM PHARMS INC. The specific approval date is not disclosed in available facts.
Lumasiran is administered via subcutaneous injection, allowing for outpatient or home-based chronic therapy.
Lumasiran is developed and manufactured by Alnylam Netherlands B.V., with the FDA-approved product marketed under the brand name OXLUMO by ALNYLAM PHARMS INC.
The specific mechanism of action of lumasiran is not yet disclosed in available facts, though it is indicated for PH1, suggesting it targets the metabolic pathway responsible for oxalate overproduction.
The specific molecular target of lumasiran is not yet disclosed in available facts.
Lumasiran's development included the trial NCT03350451; however, detailed trial design, results, and endpoints are not yet disclosed in available facts.
Lumasiran has progressed beyond Phase 2 and has achieved FDA approval, indicating successful Phase 3 completion and regulatory review.
No partner or licensee is disclosed for lumasiran; development and commercialization are being pursued by Alnylam Netherlands B.V. independently.
Lumasiran is indicated for Primary Hyperoxaluria Type 1 (PH1), a rare genetic metabolic disorder affecting oxalate production and excretion.
Current PH1 management relies primarily on supportive care with pyridoxine (vitamin B6) in responsive patients and dietary modification. Lumasiran represents a novel disease-modifying approach; other competitors in development are not well-characterized in available facts.
Lumasiran targets patients with Primary Hyperoxaluria Type 1, a rare orphan disease affecting both pediatric and adult populations; the exact prevalence and patient population size are not disclosed in available facts.
Regulatory status with the EMA, PMDA (Japan), and NMPA (China) is not yet disclosed in available facts.
The internal code for the lumasiran program is ALN-GO1-002, used by Alnylam for program tracking and identification.
The most recent program milestone was recorded on 25 April 2024; specific details of this milestone are not yet disclosed in available facts.
The brand name for lumasiran sodium is OXLUMO, approved and marketed by ALNYLAM PHARMS INC.
Lumasiran → Drug → Target → Indication → Company → Trials → Competitors
Strategic Implications: Alnylam's approval of lumasiran represents successful execution of a rare disease strategy focused on genetic/metabolic disorders. The company's decision to pursue monotherapy development without disclosed partnerships suggests confidence in the program's commercial potential and internal capability to manage development and commercialization. The Phase 2 designation in this profile may reflect earlier documentation; FDA approval indicates Phase 3 completion and regulatory success.
Competitive Implications: Lumasiran's approval establishes a new standard of care for PH1 patients, potentially displacing supportive-care-only approaches. The limited competitive set suggests a first-mover advantage in disease-modifying therapy. Future competitors targeting PH1 via alternative mechanisms (e.g., hepatic gene therapy, alternative small molecules, or biologics) may emerge, but lumasiran's approval and market presence create a high bar for entry.
Future Catalysts: Label expansion to additional PH1 subtypes or related hyperoxaluria conditions; long-term efficacy and safety data; real-world evidence in commercial use; potential pediatric indication expansion; international regulatory approvals (EMA, PMDA, NMPA); combination therapy exploration; and patient access/reimbursement milestones.
Expected Milestones: Post-approval pharmacovigilance and safety monitoring; commercial launch and uptake data; potential Phase 4 studies; regulatory submissions in additional geographies; and expansion of clinical evidence base in diverse patient populations.
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Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.