OXLUMO Continues Upward Market Momentum, Reinforcing RNAi Leadership in Primary Hyperoxaluria Treatment | DelveInsight

The market outlook for OXLUMO reflects a positive trajectory in both market performance and therapeutic potential for treating primary hyperoxaluria type 1. The drug generated approximately USD 167 million in global net product revenues for the full year of 2024, reflecting a 29% increase compared to the previous year.

/EIN News/ -- New York, USA, Feb. 25, 2025 (GLOBE NEWSWIRE) -- OXLUMO Continues Upward Market Momentum, Reinforcing RNAi Leadership in Primary Hyperoxaluria Treatment | DelveInsight

The market outlook for OXLUMO reflects a positive trajectory in both market performance and therapeutic potential for treating primary hyperoxaluria type 1. The drug generated approximately USD 167 million in global net product revenues for the full year of 2024, reflecting a 29% increase compared to the previous year.

DelveInsight’s “OXLUMO Market Size, Forecast, and Market Insight Report” highlights the details around OXLUMO, which is a glycolate oxidase expression inhibitor. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of OXLUMO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Alnylam Pharmaceuticals’ OXLUMO (lumasiran) Overview

OXLUMO (lumasiran) is an RNA interference (RNAi) therapy designed to target hydroxyacid oxidase 1 (HAO1), the gene responsible for encoding glycolate oxidase (GO). By silencing HAO1 and reducing GO enzyme levels, OXLUMO helps prevent the production of oxalate, a key contributor to the development of primary hyperoxaluria type 1 (PH1).

This treatment leverages Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enhances subcutaneous administration by improving potency, durability, and safety. OXLUMO is given as a subcutaneous injection once a month for the first three months, followed by quarterly maintenance doses starting one month after the last loading dose. For patients under 10 kg, monthly dosing continues. A healthcare professional must administer the injections.

In the ILLUMINATE-A trial, OXLUMO significantly reduced urinary oxalate levels compared to placebo, with most patients achieving normal or near-normal levels. The ILLUMINATE-B Phase III pediatric study confirmed a similar efficacy and safety profile. Additionally, the ILLUMINATE-C study demonstrated notable reductions in plasma oxalate in patients with advanced PH1. Across all three trials, the most common treatment-related side effect was injection site reactions (ISRs).

OXLUMO achieved approximately USD 167 million in global net product revenue for the full year of 2024, marking a 29% increase from the previous year. Revenue for OXLUMO is expected to grow significantly in 2025, driven by greater awareness of PH1 and expanded reimbursement options. This growth is further supported by strong clinical outcomes, demonstrating substantial reductions in urinary oxalate levels—key to disease management. Additionally, Alnylam Pharmaceuticals continues to advance research and pursue regulatory approvals to strengthen OXLUMO’s therapeutic profile, reinforcing its leadership in PH1 treatment despite rising competition.

Drug Name	OXLUMO
Molecule type	Small interfering RNA
Developer	Alnylam Pharmaceuticals
Marketed indication	Primary hyperoxaluria type 1 (PH1)
Mechanism of action	Glycolate oxidase expression inhibitor
Route of administration	Subcutaneous

Learn more about OXLUMO projected market size for primary hyperoxaluria @ OXLUMO Market Potential 

Primary Hyperoxaluria is a rare genetic disorder resulting from defects in glyoxylate metabolism. It is primarily inherited in an autosomal recessive manner and is categorized into three types based on the specific gene mutations involved: Type 1 (AGXT) (Primary Hyperoxaluria-I), Type 2 (GRHPR) (Primary Hyperoxaluria-II), and Type 3 (HOGA1) (Primary Hyperoxaluria-III). In 2023, the total prevalent population of primary hyperoxaluria across the 7MM was approximately 12,000, with projections indicating a growth rate of around 1% CAGR over the study period (2020–2034).

Before the introduction of OXLUMO and RIVFLOZA, treatment options were largely limited to off-label supportive therapies, along with kidney and liver transplantation. In severe cases, the preferred approach is isolated kidney transplantation for Primary Hyperoxaluria-II, whereas Primary Hyperoxaluria-I often requires a combined liver-kidney transplant.

The global burden of primary hyperoxaluria has been rising over the years, with the United States reporting the highest number of diagnosed prevalent cases among the seven major markets. Despite its high prevalence in the US, the condition is frequently misdiagnosed, resulting in a smaller treatable patient population relative to the total number of affected individuals, which ultimately influences the market size of primary hyperoxaluria treatments. As per DelveInsight analysis, the primary hyperoxaluria treatment market size in the 7MM was approximately USD 316 million in 2023.

Discover more about the primary hyperoxaluria market in detail @ Primary Hyperoxaluria Market Report

Emerging Competitors of OXLUMO

Several drugs, including Stiripentol (Biocodex), Reloxaliase (Allena Pharmaceutical), and Oxabact (OxThera), were being developed for the treatment of primary hyperoxaluria. However, their development has either been discontinued or no recent progress has been reported.

Currently, various preclinical approaches are being explored, primarily focusing on orally administered formulations designed to degrade oxalate in the gut. These formulations aim to lower oxalate levels in the intestinal tract, making them particularly suitable for treating secondary hyperoxaluria. However, they may also play a supportive role in primary hyperoxaluria treatment. This potential is reinforced by a study in which primary hyperoxaluria patients undergoing dialysis showed symptom improvement and reduced plasma oxalate levels following long-term Oxalobacter therapy.

Several investigational treatments for primary hyperoxaluria have demonstrated promising results in preclinical studies but have yet to reach clinical application. These include enzyme replacement therapy (ERT), CRISPR–Cas9-based gene editing combined with ERT, and inflammasome inhibition.

Stiripentol (Diacomit), developed by Biocodex, is an anticonvulsant used as adjunct therapy with Clobazam and Valproic acid for epilepsy. It received orphan drug designation from the European Commission in June 2020 and from the US FDA in February 2021 for the treatment of primary hyperoxaluria. In May 2019, Biocodex initiated a Phase II trial to assess Stiripentol as monotherapy for primary hyperoxaluria, which was completed in March 2021. However, no recent updates on its development have been reported.

Reloxaliase (ALLN-177), developed by Allena Pharmaceuticals, had reached Phase II clinical trials for primary hyperoxaluria and received orphan drug designation from both the US FDA and the European Commission. However, its development was halted after Allena Pharmaceuticals ceased operations in June 2023 due to liquidation.

Similarly, Oxabact (OC5-DB-02), a bimodal enteric biotherapy developed by OxThera containing a lyophilized formulation of Oxalobacter formigenes—a nonpathogenic, oxalate-degrading bacterium—was being investigated for primary hyperoxaluria. However, its pivotal Phase III trial was terminated in 2021.

Among other emerging treatments, Arbor Biotechnologies is developing ABO-101, a novel type V CRISPR–Cas system, for primary hyperoxaluria. The company is currently preparing to file an Investigational New Drug (IND) application to advance into clinical trials.

Additionally, YolTech Therapeutics has developed YOLT-203, an innovative in vivo gene-editing therapy targeting primary hyperoxaluria type 1. Utilizing its proprietary YolCas12TM CRISPR/Cas gene-editing platform, YOLT-203 is delivered via lipid nanoparticles to target liver cells and correct mutations in the AGXT gene. In August 2024, YolTech announced the administration of the first patient dose in an Investigator-Initiated Trial (IIT), marking YOLT-203 as the world's first in vivo gene-editing therapy for PH1.

To know more about the number of competing drugs in development, visit @ OXLUMO Market Positioning Compared to Other Drugs

Key Milestones of OXLUMO

• In October 2022, Alnylam Pharmaceuticals, announced that the FDA had approved a label expansion for OXLUMO (lumasiran), The approval indicated that OXLUMO was authorized for the treatment of PH1 to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in both pediatric and adult patients. This approval was based on positive efficacy and safety results from the ILLUMINATE-C Phase III study of OXLUMO in patients with severe renal impairment, including those on hemodialysis.
• In November 2020, Alnylam Pharmaceuticals announced that the US FDA approved OXLUMO (lumasiran) for subcutaneous use to treat PH1, marking it as the first therapy for lowering urinary oxalate levels, and the European Commission also granted marketing authorization for PH1 treatment across all age groups.
• In April 2020, Alnylam Pharmaceuticals and Dicerna granted each other a non-exclusive cross-license to their respective intellectual property related to lumasiran and Dicerna’s nedosiran.
• OXLUMO was reviewed by the FDA under Priority Review and had previously received Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease Designations. OXLUMO also received Priority Medicines (PRIME) Designation from the European Medicines Agency (EMA), along with Orphan Designation in the European Union. Additionally, Lumasiran was granted Accelerated Assessment by the EMA.

Discover how OXLUMO is shaping the primary hyperoxaluria treatment landscape @ OXLUMO Primary Hyperoxaluria

OXLUMO Market Dynamics

OXLUMO, a first-in-class RNA interference (RNAi) therapy for primary hyperoxaluria type 1, specifically reduces oxalate production, addressing a critical unmet medical need. Its targeted approach aligns with the growing trend toward personalized medicine, enabling more effective and tailored treatments based on genetic profiles. 

Regulatory incentives such as orphan drug designations, fast-track approvals, and financial support encourage its development and commercialization, while minimal direct competition strengthens its market positioning and pricing power. Clinical evidence supporting OXLUMO’s efficacy in reducing oxalate levels may drive physician adoption and patient preference, further supported by increased genetic testing and awareness leading to earlier diagnoses. 

However, as PH1 is a rare disease, the total addressable market remains limited, with premium pricing posing affordability challenges, particularly in regions with restricted insurance coverage. Economic fluctuations, shifting healthcare policies, and evolving gene therapy and small-molecule treatments could introduce competitive pressures, while limited familiarity among healthcare providers and underdeveloped reimbursement mechanisms in low- and middle-income countries may hinder broader adoption.

Dive deeper to get more insight into OXLUMO’s strengths & weaknesses relative to competitors @ OXLUMO Market Drug Report

Table of Contents

1	Report Introduction
2	OXLUMO: Alnylam Pharmaceuticals
2.1	Product Overview
2.2	Other Development Activities
2.3	Clinical Development
2.4	Clinical Trials Information
2.5	Safety and Efficacy
2.6	Product Profile
2.7	Market Assessment
2.7.1	The 7MM Analysis
2.7.1.1	Cost Assumptions and Rebate
2.7.1.2	Pricing Trends
2.7.1.3	Analogue Assessment
2.7.1.4	Launch Year and Therapy Uptake
2.7.2	The United States Market Analysis
2.7.3	EU4 and the United Kingdom Market Analysis
2.7.3.1	Germany
2.7.3.2	France
2.7.3.3	Italy
2.7.3.4	Spain
2.7.3.5	UK
2.7.4	Japan Market Analysis
2.8	Market Drivers
2.9	Market Barriers
2.10	SWOT Analysis
3	Key Cross of Marketed Competitors of OXLUMO
4	Key Cross of Emerging Competitors of OXLUMO

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