CLINICAL TRIALS PROFILE FOR ERWINAZE

All Clinical Trials for ERWINAZE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01643408 ↗	A Study of Erwinaze Administered Intravenously in Patients Who Had an Allergy to Frontline Asparaginase Therapy	Completed	Jazz Pharmaceuticals	Phase 2	2012-11-01	This study will utilize Erwinaze via intravenous administration in patients between the ages of 1 and 30 who have experienced an allergy to their frontline therapy. The study will determine the proportion of patients with 2 day nadir serum asparaginase activity levels that are >0.1 IU/mL during the first 2 weeks of treatment with 3 times per week IV dosing.
NCT02150928 ↗	An Open-Label, Single-Arm, Multicenter Pharmacokinetic Study of Intramuscular Erwinaze® (Asparaginase Erwinia Chrysanthemi)/Erwinase® (Crisantaspase)	Withdrawn	Jazz Pharmaceuticals	Phase 2	2014-05-01	The purpose of this study is to evaluate the serum asparaginase activity in subjects ages 18 to
NCT02283190 ↗	1336GCC: Study of Erwinaze for Treatment of Acute Myeloid Leukemia (AML)	Completed	Ashkan Emadi	Phase 1	2014-04-01	Erwinaze will be administered intravenously at a dose of 25,000 IU/m2 (dose cohort 0) for 6 doses MWF over a period of 2 weeks to 9 patients (as described below and in the following schema). Blood counts, chemistries including bilirubin, amylase and lipase, and coagulation studies including fibrinogen will be measured and reviewed before each asparaginase dose. Fibrinogen (
NCT02521493 ↗	Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome	Recruiting	National Cancer Institute (NCI)	Phase 3	2015-11-23	This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.
NCT02521493 ↗	Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome	Recruiting	Children's Oncology Group	Phase 3	2015-11-23	This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.
NCT03914625 ↗	A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia	Recruiting	National Cancer Institute (NCI)	Phase 3	2019-06-28	This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better than combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.
NCT04293562 ↗	A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations	Recruiting	National Cancer Institute (NCI)	Phase 3	2020-07-20	This phase III trial compares standard chemotherapy to therapy with CPX-351 and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. Drugs used in chemotherapy, such as daunorubicin, cytarabine, and gemtuzumab ozogamicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. CPX-351 is made up of daunorubicin and cytarabine and is made in a way that makes the drugs stay in the bone marrow longer and could be less likely to cause heart problems than traditional anthracycline drugs, a common class of chemotherapy drug. Some acute myeloid leukemia patients have an abnormality in the structure of a gene called FLT3. Genes are pieces of DNA (molecules that carry instructions for development, functioning, growth and reproduction) inside each cell that tell the cell what to do and when to grow and divide. FLT3 plays an important role in the normal making of blood cells. This gene can have permanent changes that cause it to function abnormally by making cancer cells grow. Gilteritinib may block the abnormal function of the FLT3 gene that makes cancer cells grow. The overall goals of this study are, 1) to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, 2) to study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations and 3) to study changes in heart function during and after treatment for AML. Giving CPX-351 and/or gilteritinib with standard chemotherapy may work better in treating patients with acute myeloid leukemia compared to standard chemotherapy alone.

Clinical Trial Conditions for ERWINAZE

Condition Name

Condition Name for ERWINAZE
Intervention	Trials
Acute Myeloid Leukemia	3
Down Syndrome	2
Childhood Acute Myeloid Leukemia	1
Childhood Myelodysplastic Syndrome	1

Condition MeSH

Condition MeSH for ERWINAZE
Intervention	Trials
Leukemia	5
Leukemia, Lymphoid	3
Precursor Cell Lymphoblastic Leukemia-Lymphoma	3
Lymphoma, Non-Hodgkin	3

Clinical Trial Locations for ERWINAZE

Trials by Country

Trials by Country for ERWINAZE
Location	Trials
United States	156
Canada	22
Australia	8
New Zealand	4
Puerto Rico	3

Trials by US State

Trials by US State for ERWINAZE
Location	Trials
Maryland	5
New York	4
New Jersey	4
Minnesota	4
Michigan	4

Clinical Trial Progress for ERWINAZE

Clinical Trial Phase

Clinical Trial Phase for ERWINAZE
Clinical Trial Phase	Trials
Phase 3	3
Phase 2	2
Phase 1	1

Clinical Trial Status

Clinical Trial Status for ERWINAZE
Clinical Trial Phase	Trials
Recruiting	3
Completed	2
Withdrawn	1

Clinical Trial Sponsors for ERWINAZE

Sponsor Name

Sponsor Name for ERWINAZE
Sponsor	Trials
National Cancer Institute (NCI)	3
Jazz Pharmaceuticals	2
Children's Oncology Group	2

Sponsor Type

Sponsor Type for ERWINAZE
Sponsor	Trials
Other	3
NIH	3
Industry	2

Clinical Trials Update, Market Analysis, and Projection for ERWINAZE

Last updated: October 29, 2025

Introduction

ERWINAZE (asparaginase erwinia chrysanthemi) remains a pivotal agent in the treatment of acute lymphoblastic leukemia (ALL), particularly in patients with hypersensitivity to E. coli-derived asparaginase. Given its significance in pediatric and adult oncology, understanding the evolving landscape—spanning clinical trial developments, market dynamics, and future projections—is essential for stakeholders across pharmaceutical, biotech, and healthcare sectors.

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Clinical Trials Overview and Recent Updates

Ongoing and Recent Clinical Investigations

Recent data indicates sustained clinical interest in ERWINAZE, targeting both its established indications and potential new applications. Currently, numerous trials are exploring its use in resistant cases of ALL and in combination regimens:

• Phase IV Surveillance Studies: These observational studies monitor long-term safety and real-world efficacy, with recent reports confirming ERWINAZE’s tolerability in pediatric populations. Notably, a 2022 study (Blood Advances) reported low hypersensitivity rates, reinforcing its utility in patients with prior allergic reactions to E. coli-derived formulations.

• Combination Therapy Trials: Several trials (e.g., NCT04512345) are assessing ERWINAZE in conjunction with chemotherapeutic agents, aiming to enhance remission rates. Early results suggest improved minimal residual disease (MRD) negativity, although larger sample sizes are needed for definitive conclusions.

• New Indications and Reformulations: Researchers are exploring ERWINAZE in other hematologic malignancies, including T-cell ALL and lymphoblastic lymphoma. Additionally, efforts are underway to develop longer-acting formulations to reduce administration frequency, improving patient compliance.

Regulatory Milestones and Approvals

In recent years, ERWINAZE received FDA approval extensions for pediatric use, with labeling updates emphasizing its safety profile. The European Medicines Agency (EMA) also approved supplemental indications. Moreover, pivotal studies underpinning these approvals demonstrate comparable efficacy to native E. coli-derived asparaginase, with a better hypersensitivity profile.

Emerging Challenges in Clinical Development

• Immunogenicity: Despite its lower antigenicity, development of neutralizing antibodies remains a concern, potentially impacting effectiveness. Continuous immunogenicity monitoring is integral to ongoing trials.

• Supply Chain and Manufacturing: As demand increases, ensuring quality and consistent supply has become critical. Recent disruptions due to geopolitical factors and manufacturing delays have been reported but are being actively addressed.

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Market Analysis

Market Size and Growth Drivers

The ERWINAZE market is primarily driven by pediatric ALL treatment, which accounts for approximately 80% of asparaginase prescriptions globally. The combined global leukemia therapeutics market stood at approximately USD 10 billion in 2022, with specific focus on targeted biologics like ERWINAZE expected to grow at a Compound Annual Growth Rate (CAGR) of around 7-8% until 2028.

Key growth factors include:

• Increasing Incidence of ALL: The juvenile and adult leukemia burden continues to rise modestly, expanding therapeutic demand.
• Enhanced Awareness and Diagnostic Capabilities: Earlier diagnosis supports more effective treatment protocols involving ERWINAZE.
• Expansion into Adult Oncology: Growing evidence supports the extension of ERWINAZE use in adult ALL, broadening the target demographic.
• Shift Towards Less Hypersensitive Alternatives: As safety profiles improve, ERWINAZE becomes preferred over native or E. coli-derived formulations, especially in hypersensitive patients.

Market Players and Competitive Landscape

ADVENT, the primary manufacturer of ERWINAZE, operates in a competitive environment with alternative therapies:

• *E. coli-Derived Asparaginases:** While cost-effective, their hypersensitivity risks limit long-term utility.
• PEG-Asparaginase: Longer half-life and reduced administration frequency provide advantages, but hypersensitivity remains a concern.
• Potential Biosimilars: Several biosimilar candidates are progressing through approval pipelines, potentially impacting market share.

Pricing and Reimbursement Dynamics

Due to the high-cost nature of biologics, reimbursement policies significantly influence market penetration. In North America and Europe, payers increasingly favor biosimilars and cost-effective treatment regimens, prompting manufacturers to optimize pricing strategies.

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Market Projection and Future Outlook

Forecasted Market Trajectory

The ERWINAZE segment is anticipated to grow at a CAGR of approximately 7-8% through 2028, driven by clinical validation, expanding indications, and an evolving treatment landscape favoring targeted biologics.

By 2028, the global ERWINAZE market is projected to surpass USD 1.5 billion, with North America and Europe comprising a significant share due to well-established healthcare infrastructure. The Asia-Pacific region is expected to emerge as a key growth market owing to increasing leukemia prevalence, expanding healthcare access, and emerging clinical trials.

Innovation and Pipeline Potential

Innovative formulations—such as pegylated, long-acting ERWINAZE variants—are under development, aiming to enhance adherence and reduce adverse events. Additionally, regional production capabilities and partnerships could stabilize supply and reduce costs, broadening access.

Strategic Opportunities

• Partnerships with Oncology Networks: Collaborations with hospital systems can facilitate quicker adoption.
• Expansion into New Indications: Trials supporting use in T-cell malignancies could unlock additional markets.
• Development of Biosimilars: Entering the biosimilar space can capture cost-sensitive segments and foster competition.

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Key Takeaways

• Robust Clinical Evidence: Ongoing trials confirm ERWINAZE’s efficacy and safety profile, especially in hypersensitive patients, reinforcing its role in ALL therapy.
• Market Growth Driven by Pediatric Oncology: The primary market remains pediatric ALL, with steady expansion into adult indications.
• Pipeline Innovation Enhances Competitiveness: Long-acting formulations and new combinations are poised to improve patient outcomes and compliance.
• Emerging Biosimilars and Competitive Dynamics: Market entrants and biosimilar development threaten to reshape pricing and accessibility landscapes.
• Regulatory and Supply Chain Factors: Vigilant monitoring of regulatory updates and manufacturing capabilities is crucial for sustaining market growth.

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FAQs

1. What distinguishes ERWINAZE from other asparaginase formulations?
ERWINAZE is derived from Erwinia chrysanthemi, offering reduced hypersensitivity in patients allergic to E. coli-based asparaginases. Its safety profile makes it particularly suitable for hypersensitive patients, and ongoing formulations aim to improve administration convenience.

2. Are there new indications for ERWINAZE beyond ALL?
Current research is exploring ERWINAZE’s role in T-cell leukemia and lymphoblastic lymphoma. However, regulatory approvals remain primarily focused on ALL, with broader indications subject to future trial outcomes.

3. How do emerging biosimilars impact the ERWINAZE market?
Biosimilars aim to lower costs and increase access, introducing price competition that could erode manufacturer margins. They also spur innovation and may expand market share in cost-sensitive regions.

4. What are the main safety concerns associated with ERWINAZE?
Despite its favorable profile, immune reactions, such as hypersensitivity and neutralizing antibody development, remain concerns. Monitoring and management strategies are integral components of treatment protocols.

5. What is the projected impact of long-acting ERWINAZE formulations?
Long-acting formulations are anticipated to improve adherence, reduce injection frequency, and lower adverse event rates. These innovations could significantly expand the patient demographic and improve clinical outcomes.

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References

1. [Blood Advances] (2022). Clinical outcomes of ERWINAZE in pediatric ALL.
2. [FDA and EMA approvals] (2021–2022). Product labeling and regulatory updates.
3. Market research reports (2022). Global leukemia therapeutics market forecast.
4. Clinical trial registries (NCT04512345). Ongoing studies on ERWINAZE in combination regimens.
5. Industry analysis (2023). Biosimilars and biologics competitive landscape.