Oxlumo (lumasiran) / Alnylam, Royalty, Novo Nordisk - LARVOL DELTA

Kidney Function and Isolated Kidney Transplant Outcomes in Primary Hyperoxaluria Type 1 Treated with Long-Term Lumasiran (WTC 2025) - P2, P3 | "Lumasiran treatment for PH1 resulted in minimal annual eGFR decline over 30 to 60 months in patients with chronic kidney disease and effectively lowered plasma oxalate to allow for iKT, rather than liver-kidney transplantation, in some patients with end-stage kidney disease."

Chronic Kidney Disease • Nephrology • Transplantation

The Evolving Role of Liver Transplantation as Enzyme Replacement Therapy in the Era of RNA Nanotherapies. (PubMed, Am J Transplant) - "A lower proportion of patients were waitlisted for LT due to ATTR/PH in the post-FDA approval era, and these patients were also sicker. Changes in patient selection criteria in context of availability of less invasive strategies may have resulted in sicker patients being listed later in their disease course."

Journal • Amyloidosis • Cardiac Amyloidosis • Nephrology • Solid Organ Transplantation • Transplantation

Updates on Pharmacological Therapy for Urolithiasis (UAA 2025) - "In primary hyperoxaluria (PH), novel RNA interference (RNAi) agents like lumasiran and nedosiran significantly lower urinary oxalate levels, offering promising alternatives for patients unresponsive to pyridoxine...Xanthine oxidase inhibitors (allopurinol, febuxostat) are reserved for hyperuricemic patients...Medical expulsive therapy (MET) with alpha-blockers (tamsulosin) remains effective for distal ureteral stones (5–10 mm), reducing time to expulsion and need for surgery...Operative times vary, with suction PCNL often being faster for large stones (47–82 min) but requiring fluoroscopy, while suction RIRS avoids tract-related risks but may necessitate staged procedures for stones >2 cm. Cost-effectiveness analyses favor suction PCNL due to fewer retreatments, though RIRS reduces radiation exposure."

Infectious Disease • Nephrology • Renal Calculi • Urolithiasis

ILLUMINATE-C: A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (clinicaltrials.gov) - P3 | N=21 | Completed | Sponsor: Alnylam Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Nephrology

Suction PCNL vs Suction RIRS? Do We Have a Winner (UAA 2025) - "In primary hyperoxaluria (PH), novel RNA interference (RNAi) agents like lumasiran and nedosiran significantly lower urinary oxalate levels, offering promising alternatives for patients unresponsive to pyridoxine...Xanthine oxidase inhibitors (allopurinol, febuxostat) are reserved for hyperuricemic patients...Medical expulsive therapy (MET) with alpha-blockers (tamsulosin) remains effective for distal ureteral stones (5–10 mm), reducing time to expulsion and need for surgery...Operative times vary, with suction PCNL often being faster for large stones (47–82 min) but requiring fluoroscopy, while suction RIRS avoids tract-related risks but may necessitate staged procedures for stones >2 cm. Cost-effectiveness analyses favor suction PCNL due to fewer retreatments, though RIRS reduces radiation exposure."

Infectious Disease • Nephrology • Renal Calculi • Urolithiasis

OXLUMO庐, (lumasiran) for the Treatment of Primary Hyperoxaluria (PH1): Case Studies (WTC 2025) - "Sponsored by Alnylam"

Case study • Clinical • Nephrology

The Transformative Impact of RNA Nanomedicine on Need for Solid Organ Transplantation (WTC 2025) - "In conclusion, our study reports important findings of a decreased proportion of ATTR and PH patients who were waitlisted for LT, coupled with an increase in non-liver SOT following FDA approval of patisiran and lumasiran. Physicians may have waitlisted sicker patients in the post-FDA approval era given availability of RNA nanotherapies. The increased proportion of Black patients waitlisted for SOT in the post-FDA approval era may reflect healthcare access and socioeconomic disparities."

Amyloidosis • Cardiac Amyloidosis • Genetic Disorders • Nephrology • Solid Organ Transplantation • Transplantation

Lumasiran at birth changes the trajectory of primary hyperoxaluria type 1: same disease, different outcomes in two affected siblings. (PubMed, J Nephrol) - "No adverse events occurred. In this report, early treatment with lumasiran, coupled with hyperhydration and supportive therapy, was able to ensure the absence of primary hyperoxaluria type 1 symptoms throughout the 24 months of follow-up."

Journal • Metabolic Disorders • Nephrology

OXLUMO (lumasiran injection) Now Reimbursed in Canada For the Treatment of Primary Hyperoxaluria Type 1 (PH1) in Pediatric and Adult Patients (Canada Newswire) - "Alnylam Canada ULC is pleased to announce that OXLUMO (lumasiran) is now funded across Canada through both public and private plans. OXLUMO is an RNA interference (RNAi) therapeutic administered via subcutaneous injection, indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients....The positive recommendation for reimbursement was supported by results of the ILLUMINATE clinical studies, including ILLUMINATE-A: a randomized, double-blind, placebo-controlled clinical study in patients six years and older with PH1, ILLUMINATE-B: a single-arm clinical study in patients less than six years of age with PH1 and ILLUMINATE-C: a single-arm trial in patients of all ages with advanced PH1, including patients on dialysis."

Reimbursement • Rare Diseases

Plasma Glycolate Levels Contribute to Drive the Decision of Isolated Kidney Transplantation in Dialyzed Patients with End-Stage Kidney Disease due to Primary Hyperoxaluria Type 1 Treated with Lumasiran: A Case Report. (PubMed, Case Rep Nephrol Dial) - "The novelty of our case lies in the methods we adopted to evaluate the lumasiran efficacy during dialysis prior listing for IKT. Specifically, we decided to list the patient for IKT according to the high plasma levels of glycolate reached after lumasiran treatment together with the significant reduction of the pOx."

Journal • Chronic Kidney Disease • Genetic Disorders • Metabolic Disorders • Nephrology • Renal Calculi • Renal Disease • Transplantation

Administration of lumasiran in a child with infantile oxalosis undergoing chronic peritoneal dialysis: A case report (PubMed, Nephrol Ther) - "Once the patient reached a sufficient body weight to safely initiate hemodialysis (HD), she was switched to HD; POx levels remained around 100 µmol/L, despite an intensive HD regimen. This is one of the first reported cases of lumasiran therapy during PD in an infant with PH1; even with this dialysis modality, POx levels can be decreased to a certain extent."

Journal • Chronic Kidney Disease • Nephrology • Renal Disease

Long-Term Efficacy and Safety in the 60-Month, Phase 3 ILLUMINATE-B Trial of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ERA 2025) - P3 | "In this final analysis of long-term data from ILLUMINATE-B, infants and young children with PH1 had reductions in UOx and POx that were sustained over 60 months of lumasiran treatment. Kidney function was stable throughout long-term treatment. Medullary NC grade improved in most patients, and kidney stone event rates were low."

Clinical • P3 data • Chronic Kidney Disease • Dermatology • Genetic Disorders • Immunology • Metabolic Disorders • Nephrology • Pain • Pediatrics • Renal Calculi • Urolithiasis • Urticaria

Improvement of the diagnostic delay of primary hyperoxaluria (ERA 2025) - "Since then, the development of new treatments, such as lumasiran (an RNA interference therapy), has increased awareness of the disease within the nephrology community... The diagnostic delay for PH has been reduced by half over the past five years, driven by advancements in treatment and improved knowledge and awareness of the disease. This may facilitate earlier management and limit complications with appropriate therapies."

Genetic Disorders • Nephrology • Pediatrics

Rescue of renal function following novel RNA Interference therapy (Lumasiran) for catastrophic recurrent PrimaryHyperoxaluria type 1 (PH1) in a kidney transplant (UKKW 2025) - No abstract available

Nephrology • Transplantation

Differential Protein Precipitation-Based GalNAc-siRNA Sample Preparation with LC/MS Method Development Workflow in Plasma. (PubMed, Anal Chem) - "This approach achieved a lower limit of quantification in the single-digit ng/mL range for four FDA-approved GalNAc-siRNAs (Givosiran, Lumasiran, Inclisiran, and Vutrisiran) and a major Givosiran metabolite, AS(N-1)3'. The applicability of this approach was successfully demonstrated by analyzing plasma samples from an in vivo rat study involving three molecules (Givosiran, Givosiran AS(N-1)3', and Inclisiran). This method is straightforward, robust, highly sensitive, and cost-effective and should be readily adaptable for the bioanalysis of diverse GalNAc-siRNAs and, potentially, for late-stage sample analyses."

Journal

Treatment preferences among individuals with primary hyperoxaluria type 1 (PH1): a real-world study. (PubMed, Orphanet J Rare Dis) - "This study shows that patients with PH1 value treatments that are convenient and fit their lifestyle."

Journal • Real-world evidence • Genetic Disorders • Nephrology

Recent Progression and Treatment Approaches for the Kidney Stone Management. (PubMed, Curr Drug Targets) - "To implement more effective treatments, it is necessary to identify and develop a targeted therapy for the druggable targets. Various such druggable targets have been reported such as osteopontin which has come out as a protein with various functions including involvement in the inhibition of crystal adherence to the renal epithelium. Another such target is vitamin D and nephrocalcin."

Journal • Nephrology • Renal Calculi • Urolithiasis • SPP1

First Quarter 2025 and Recent Significant Business Highlights (Businesswire) - "Achieved global net product revenues for AMVUTTRA and ONPATTRO for the first quarter of $310 million and $49 million, respectively, representing 36% growth compared to Q1 2024...Achieved global net product revenues for GIVLAARI and OXLUMO for the first quarter of $67 million and $42 million, respectively, representing 8% growth compared to Q1 2024."

Sales • Amyloidosis • Cardiomyopathy • Genetic Disorders • Renal Disease

Real-Life Data of 2-Year Lumasiran Use in the DAILY-LUMA Cohort. (PubMed, Kidney Int Rep) - P | "Tolerance was good with no severe side effects; injection site reactions, abdominal pain, and headaches were the main adverse events. DAILY-LUMA is the largest cohort of patients receiving lumasiran in real life, confirming its safety and efficacy at 2 years."

Journal • Metabolic Disorders • Nephrology • Pain • Transplantation

Primary Hyperoxaluria Type 1 Management: Phase 3 ILLUMINATE Clinical Trial Data and Pediatric Case Review (PAS 2025) - "This will be closed by a Q&A session. Learning Objectives: Detail the pathophysiology of PH1 and the burden of PH1 Review the ILLUMINATE clinical trial program study duration, patient populations, lumasiran dosing regimen, and recent data Summarize data from the ILLUMINATE clinical trial program and a real-world patient case, which demonstrates the long-term efficacy and acceptable safety profile of lumasiran in pediatric patients with PH1"

Clinical • P3 data • Review • Nephrology • Pediatrics

Kidney Function and Isolated Kidney Transplant Outcomes in Primary Hyperoxaluria Type 1 Treated with Long-term Lumasiran (PAS 2025) - No abstract available

Nephrology • Transplantation

Long-term Efficacy and Safety of Lumasiran in Patients with Primary Hyperoxaluria Type 1 in a Final Analysis of the ILLUMINATE-A Trial (PAS 2025) - No abstract available

Clinical • Nephrology

Hidden in CAKUT: Post-Transplant Diagnosis of Primary Hyperoxaluria Type 1 and Rescue Management Using Lumasiran. (PubMed, Pediatr Transplant) - "This case underscores the importance of maintaining a high index of suspicion for PH1 in patients with congenital anomalies of the kidney and urinary tract (CAKUT) or unexplained end-stage kidney disease (ESKD) cases, even post-transplantation. It also demonstrates the potential efficacy of lumasiran in managing PH1 post-transplantation when combined with intensive hemodialysis and supportive care. However, more studies with prolonged follow-up periods are necessary to establish the long-term efficacy and safety of lumasiran in the treatment of PH1 without the requirement for liver transplantation."

Journal • Chronic Kidney Disease • Hepatology • Metabolic Disorders • Nephrology • Pediatrics • Transplantation

The efficacy and safety of RNA interference for the treatment of primary hyperoxaluria: a systematic review and meta-analysis. (PubMed, Clin Kidney J) - "Furthermore, high-dose and long time-duration RNAi therapy may have a better clinical effect, and acceptable safety. The efficacy of RNAi combined with hemodialysis seems to be promising in PH treatment."

Journal • Retrospective data • Review • Genetic Disorders • Hepatology • Nephrology • Renal Disease

Long-term effects of lumasiran on kidney stones and nephrocalcinosis in patients with primary hyperoxaluria type 1 (AUA 2025) - "Long-term lumasiran treatment was associated with low occurrence of kidney stone–related AEs in the Phase 2 OLE study and low KSE rates plus decreases in NC grade in ILLUMINATE-A and ILLUMINATE-B. These are encouraging and clinically relevant outcomes that are consistent with the degree of urinary oxalate reduction while on lumasiran."

Clinical • Metabolic Disorders • Nephrology • Pediatrics • Renal Calculi