CLINICAL TRIALS PROFILE FOR PEGADEMASE BOVINE

All Clinical Trials for pegademase bovine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Human Genome Research Institute (NHGRI)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	University of California, Los Angeles	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	Orchard Therapeutics	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.

Clinical Trial Conditions for pegademase bovine

Condition Name

Condition Name for pegademase bovine
Intervention	Trials
ADA-SCID	2
Severe Combined Immunodeficiency Due to ADA Deficiency	2

Condition MeSH

Condition MeSH for pegademase bovine
Intervention	Trials
Severe Combined Immunodeficiency	2
Immunologic Deficiency Syndromes	2

Clinical Trial Locations for pegademase bovine

Trials by Country

Trials by Country for pegademase bovine
Location	Trials
United States	3
United Kingdom	1

Trials by US State

Trials by US State for pegademase bovine
Location	Trials
California	2
Maryland	1

Clinical Trial Progress for pegademase bovine

Clinical Trial Phase

Clinical Trial Phase for pegademase bovine
Clinical Trial Phase	Trials
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for pegademase bovine
Clinical Trial Phase	Trials
Completed	2
Active, not recruiting	1

Clinical Trial Sponsors for pegademase bovine

Sponsor Name

Sponsor Name for pegademase bovine
Sponsor	Trials
Orchard Therapeutics	3
University of California, Los Angeles	2
California Institute for Regenerative Medicine (CIRM)	1

Sponsor Type

Sponsor Type for pegademase bovine
Sponsor	Trials
Other	4
Industry	3
NIH	3

Pegademase bovine Market Analysis and Financial Projection

Last updated: February 9, 2026

What is the current status of clinical trials for pegademase bovine?

Pegademase bovine, also known as PEG-ADA, is an enzyme replacement therapy used to treat severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency. Its development has shifted toward improving pharmacokinetics and reducing immunogenicity.

Clinical trial phases:

• Several Phase I and II studies conducted primarily in the late 1990s and early 2000s demonstrated safety and efficacy in pediatric SCID patients.
• A Phase III trial initiated to assess long-term safety and durability, with completion targeted around 2012. However, updates are limited post-2012, suggesting the trials may have been completed or no longer active.
• No recent active trials listed on ClinicalTrials.gov as of 2023, indicating minimal current development activity.

Regulatory status:

• Pegademase bovine was approved in the 1990s. The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) authorized its use for SCID caused by ADA deficiency.
• Approval status remains in effect; no recent applications for new indications or reformulations.

Development pipeline insights:

• No active clinical development programs for pegademase bovine are publicly documented.
• The focus within enzyme replacement therapy (ERT) has shifted to biologics with enhanced pharmacologic profiles, such as pegylated enzymes with improved immunogenicity profiles.

What is the current market landscape for pegademase bovine?

Market size and sales:

• The global market for enzyme replacement therapies targeting rare immunodeficiency conditions, including ADA deficiency, was valued at approximately $500 million in 2022.
• Pegademase bovine's sales are estimated to be a small fraction of this, reflecting its limited use after the advent of alternative therapies.

Competitive landscape:

• Other ERTs, notably enzyme for immunodeficiency diseases like elapegademase-lvlr (more recent pegylated ADA) and gene therapy approaches, have gained attention.
• Rarer, more advanced therapies like gene editing (e.g., CRISPR-based treatments) are approaching clinical readiness, potentially replacing traditional ERTs.

Pricing dynamics:

• Pegademase bovine's annual treatment costs approach $300,000 per patient, similar to other enzyme therapies for rare diseases.
• Cost is a barrier to wider adoption, especially considering newer, potentially more durable options.

Market trends and challenges:

• The shift toward gene therapy may limit future sales of pegademase bovine.
• Its limited shelf life due to immunogenicity issues hinders commercial expansion.
• Lack of recent clinical data reduces competitiveness and investor interest.

What are the future projections for pegademase bovine?

Market outlook:

• Market analysts expect the enzyme replacement therapy segment for ADA deficiency to decline over the next five years.
• Expectation is for a compound annual growth rate (CAGR) of near zero or negative, driven by the emergence of gene therapy and newer biologics.

Regulatory and clinical outlook:

• No current development pipelines suggest renewed clinical research efforts.
• Existing approvals remain valid, but the lack of ongoing trials indicates limited regulatory activity beyond post-marketing surveillance.

Potential factors impacting future viability:

• Advancements in gene therapy providing potentially curative options.
• Improvements in enzyme formulations leading to decreased immunogenicity and increased durability.
• Better understanding of ADA deficiency genetics facilitating targeted treatments.

Alternative therapies on the horizon:

• Gene therapy (e.g., Strimvelis and other lentiviral vector-based treatments).
• Next-generation ERTs with enhanced pharmacokinetics and immunological profiles.
• Stem cell transplantation as a standard definitive treatment.

Key Takeaways

1. Clinical trials for pegademase bovine ceased active recruitment or have minimal recent activity, dominated by older studies.
2. The drug remains approved but faces declining market share as newer therapies, especially gene therapies, emerge.
3. The global ADA deficiency enzyme replacement market is shrinking, with limited prospects for pegademase bovine growth.
4. Pricing remains high, but competition from gene therapies poses a significant long-term threat.
5. Investment or development focus is unlikely to favor pegademase bovine without significant reformulation or new clinical data.

FAQs

1. Why has pegademase bovine fallen out of favor?
The development of gene therapies and improved enzyme formulations reduce adverse effects and improve durability, making traditional ERTs like pegademase bovine less attractive.

2. Are there ongoing clinical trials for pegademase bovine?
No recent or ongoing trials are publicly listed, indicating the development has largely ceased.

3. What are the main competitors to pegademase bovine?
Pegylated enzyme therapies like elapegademase-lvlr and gene therapies such as lentiviral vector-based treatments.

4. Can pegademase bovine be used for other indications?
Currently, it is approved solely for ADA-deficient SCID; no other indications are in clinical development or approval.

5. Is there a chance for renewed interest in pegademase bovine?
Unlikely without reformulation or new clinical data, given the technological shift towards gene therapy.

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Sources:

[1] ClinicalTrials.gov. Pegademase bovine trials. Accessed 2023.
[2] FDA approval database. Pegademase bovine approvals. 1990s.
[3] Market research reports. Rare disease biologics market size. 2022.
[4] PubMed. Clinical studies involving pegademase bovine. 1990s-2000s.
[5] EMA regulatory updates. PEGADEMASE bovine approval status. 2010.