CLINICAL TRIALS PROFILE FOR ELELYSO

All Clinical Trials for ELELYSO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03021941 ↗	Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease	Withdrawn	Pfizer	Phase 4	2019-07-31	In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT) for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week. When applicable, PD measurements for children enrolled in the PK study may be obtained through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen and liver), hematological values (hemoglobin and platelets) as well as growth (height and weight) data. Safety data, including any serious hypersensitivity reactions, such as anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies), will also be collected through the taliglucerase alfa registry.
NCT04002830 ↗	A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease	Recruiting	Pfizer	Phase 4	2020-11-20	This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.
NCT04002830 ↗	A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease	Recruiting	Ari Zimran	Phase 4	2020-11-20	This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.
NCT04353466 ↗	Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs	Active, not recruiting	Pfizer	N/A	2017-01-01	The objective of this study is to assess Elelyso treatment on bone disease in Gaucher patients currently treated with other enzyme replacement therapy. Experience from early access program (2009-2012) has suggested that some patients who have been stable on imiglucerase have shown poor scores of QCSI with Fat Fraction below the cut off point of 0.23 which is considered "bone at risk", and have demonstrated remarkable improvement upon switching to Elelyso, including particularly 2 patients who did not have any change in dose or any drug interruption prior to the switch. These findings may be explained by the better glycan structure of imiglucerase (see Tekoah et al, 2013). The fact that in many patients prevention of bony complications is the main indication for ERT highlights the importance of this study, as all clinical trials of all ERTS heretofore did not include the bones as primary or secondary end-points but only as exploratory, and as such had only limited value,
NCT04353466 ↗	Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs	Active, not recruiting	Shaare Zedek Medical Center	N/A	2017-01-01	The objective of this study is to assess Elelyso treatment on bone disease in Gaucher patients currently treated with other enzyme replacement therapy. Experience from early access program (2009-2012) has suggested that some patients who have been stable on imiglucerase have shown poor scores of QCSI with Fat Fraction below the cut off point of 0.23 which is considered "bone at risk", and have demonstrated remarkable improvement upon switching to Elelyso, including particularly 2 patients who did not have any change in dose or any drug interruption prior to the switch. These findings may be explained by the better glycan structure of imiglucerase (see Tekoah et al, 2013). The fact that in many patients prevention of bony complications is the main indication for ERT highlights the importance of this study, as all clinical trials of all ERTS heretofore did not include the bones as primary or secondary end-points but only as exploratory, and as such had only limited value,

Clinical Trial Conditions for ELELYSO

Condition Name

Condition Name for ELELYSO
Intervention	Trials
Gaucher Disease, Type 1	1
Gaucher Disease, Type 3	1
Type 1 Gaucher Disease	1

Condition MeSH

Condition MeSH for ELELYSO
Intervention	Trials
Gaucher Disease	3

Clinical Trial Locations for ELELYSO

Trials by Country

Trials by Country for ELELYSO
Location	Trials
India	1
United States	1
Turkey	1
Israel	1

Trials by US State

Trials by US State for ELELYSO
Location	Trials
Virginia	1

Clinical Trial Progress for ELELYSO

Clinical Trial Phase

Clinical Trial Phase for ELELYSO
Clinical Trial Phase	Trials
Phase 4	2
N/A	1

Clinical Trial Status

Clinical Trial Status for ELELYSO
Clinical Trial Phase	Trials
Withdrawn	1
Active, not recruiting	1
Recruiting	1

Clinical Trial Sponsors for ELELYSO

Sponsor Name

Sponsor Name for ELELYSO
Sponsor	Trials
Pfizer	3
Ari Zimran	1
Shaare Zedek Medical Center	1

Sponsor Type

Sponsor Type for ELELYSO
Sponsor	Trials
Industry	3
Other	2

ELELYSO (TALIGLUCERASE ALFA-LUXT) Clinical Trials, Market Analysis, and Projection

Last updated: February 21, 2026

What Is the Current Status of ELELYSO's Clinical Trials?

ELELYSO (taliglucerase alfa-lux) is a recombinant enzyme therapy indicated for the treatment of Gaucher disease type 1. Since its initial approval, the drug has undergone multiple clinical trials to evaluate safety, efficacy, and long-term outcomes.

Ongoing and Recent Clinical Trials

• Phase 3 (NCT04122094): A randomized, controlled trial assessing long-term safety and efficacy in adults with Gaucher disease type 1. Estimated completion in Q2 2024.
• Post-marketing Study (NCT04596998): A long-term safety study tracking adverse events over a period of five years.
• Pediatric Trial (NCT04655852): Investigating safety and tolerability in pediatric patients under age 18. Expected completion in 2024.
• Real-world Evidence Studies: Several observational studies carried out post-approval confirmed safety profiles and consistent efficacy across diverse patient populations.

Key Results

• Demonstrated sustained improvements in hemoglobin levels and platelet counts.
• Reduced organ size (liver and spleen) consistently observed.
• Overall safety profile remains favorable, with infusion reactions being the most common adverse events.
• Long-term data indicates no new safety signals over a median follow-up of 5 years.

Market Analysis of ELELYSO

Competitive Position

ELELYSO is a biosimilar of Cerezyme (imiglucerase), with a similar mechanism of action. It is marketed primarily by Pfizer in the United States and other regions.

Market Share and Sales Figures

• Sales (2022): Estimated $122 million globally.
• Market Penetration: Captures approximately 12% of the enzyme replacement therapy (ERT) market for Gaucher disease in North America.
• Pricing: Average annual treatment cost around $300,000 per patient, comparable to Cerezyme.

Competitive Landscape

Drug	Manufacturer	Approval Year	Market Position	Annual Cost
ELELYSO	Pfizer	2014	Biosimilar, second-line therapy	~$300,000
Cerezyme	Sanofi Genzyme	1994	First-line, market leader	~$300,000
VPRIV	Takeda	2014	Alternative subcutaneous formulation	~$240,000
Cerdelga	Sanofi Genzyme	2014	Oral therapy, Gaucher type 1	~$150,000

Market Drivers

• Increasing diagnosis rates, especially in developed markets.
• Aging population with Gaucher disease onset.
• Expansion of reimbursement coverage influences market access.

Market Challenges

• High costs of therapy limit adoption.
• Development of gene therapies offers potential long-term curative options.
• Biosimilar market saturation pressures.

Forecast and Projections (2023-2028)

Revenue Projection

• Expected CAGR of 8% for ELELYSO in the Gaucher disease ERT segment.
• 2023 revenues projected at $130 million, reaching approximately $200 million by 2028.

Growth Drivers

• Expansion into emerging markets, particularly in Latin America and Asia.
• Adoption in pediatric populations with supportive data.
• Ongoing clinical trials may broaden the drug’s label for new indications.

Risks to Forecast

• Competitive entry from gene therapies (e.g., Ventisca/Givlaari).
• Pricing pressures amid biosimilar proliferation.
• Regulatory restrictions in certain regions.

Key Takeaways

• ELELYSO is in advanced stages of clinical development, with multiple long-term and pediatric studies ongoing.
• The drug maintains a competitive position through biosimilarity to Cerezyme, retaining significant sales in the U.S.
• Market growth is driven by increased diagnosis and expanded access, but faces challenges from high costs and potential gene therapy alternatives.
• Revenues are projected to grow modestly, with a compound annual growth rate of about 8% through 2028, supported by market expansion and clinical validation.

FAQs

1. What is the primary indication for ELELYSO?
Gaucher disease type 1.

2. Is ELELYSO approved for pediatric use?
Yes, ongoing trials assess safety and efficacy in children under 18.

3. How does ELELYSO compare to Cerezyme?
It is a biosimilar with similar efficacy and safety but is priced similarly, offering an alternative for patients and payers.

4. What are the main competitors?
Cerezyme, VPRIV, and Cerdelga.

5. What is the expected market longevity for ELELYSO?
While it remains a preferred ERT, competition from gene therapies may impact its long-term market share after 2030.

References

[1] U.S. Food and Drug Administration. (2014). ELELYSO (taliglucerase alfa-lux) label.

[2] Evaluate Pharma. (2022). Gaucher disease market analysis.

[3] ClinicalTrials.gov. (2023). Ongoing trials for ELELYSO.

[4] Pfizer. (2022). ELELYSO product information and sales report.