CLINICAL TRIALS PROFILE FOR ZYDELIG

All Clinical Trials for Zydelig

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01620216 ↗	Targeted Therapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia	Terminated	National Cancer Institute (NCI)	Phase 2	2012-05-11	This phase II trial studies how well targeted therapy works in treating patients with acute lymphoblastic leukemia or acute myelogenous leukemia that has come back after a period of improvement or does not respond to treatment. Testing patients' blood or bone marrow to find out if their type of cancer may be sensitive to a specific drug may help doctors choose more effective treatments. Dasatinib, sunitinib malate, sorafenib tosylate, ponatinib hydrochloride, pacritinib, ruxolitinib, and idelalisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving targeted therapy based on cancer type may be an effective treatment for acute lymphoblastic leukemia or acute myelogenous leukemia.
NCT01620216 ↗	Targeted Therapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia	Terminated	Oregon Health and Science University	Phase 2	2012-05-11	This phase II trial studies how well targeted therapy works in treating patients with acute lymphoblastic leukemia or acute myelogenous leukemia that has come back after a period of improvement or does not respond to treatment. Testing patients' blood or bone marrow to find out if their type of cancer may be sensitive to a specific drug may help doctors choose more effective treatments. Dasatinib, sunitinib malate, sorafenib tosylate, ponatinib hydrochloride, pacritinib, ruxolitinib, and idelalisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving targeted therapy based on cancer type may be an effective treatment for acute lymphoblastic leukemia or acute myelogenous leukemia.
NCT01620216 ↗	Targeted Therapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia	Terminated	OHSU Knight Cancer Institute	Phase 2	2012-05-11	This phase II trial studies how well targeted therapy works in treating patients with acute lymphoblastic leukemia or acute myelogenous leukemia that has come back after a period of improvement or does not respond to treatment. Testing patients' blood or bone marrow to find out if their type of cancer may be sensitive to a specific drug may help doctors choose more effective treatments. Dasatinib, sunitinib malate, sorafenib tosylate, ponatinib hydrochloride, pacritinib, ruxolitinib, and idelalisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving targeted therapy based on cancer type may be an effective treatment for acute lymphoblastic leukemia or acute myelogenous leukemia.
NCT02332980 ↗	Pembrolizumab Alone or With Idelalisib or Ibrutinib in Treating Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia or Other Low-Grade B-Cell Non-Hodgkin Lymphomas	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2015-02-19	This phase II trial studies how well pembrolizumab alone or with idelalisib or ibrutinib works in treating patients with chronic lymphocytic leukemia or other low-grade B-cell non-Hodgkin lymphomas that have returned after a period of improvement (relapsed) or have not responded to treatment (refractory). Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Idelalisib and ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving pembrolizumab alone or with idelalisib or ibrutinib may be an effective treatment in patients with chronic lymphocytic leukemia or other low-grade B-cell non-Hodgkin lymphomas.
NCT02332980 ↗	Pembrolizumab Alone or With Idelalisib or Ibrutinib in Treating Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia or Other Low-Grade B-Cell Non-Hodgkin Lymphomas	Active, not recruiting	Mayo Clinic	Phase 2	2015-02-19	This phase II trial studies how well pembrolizumab alone or with idelalisib or ibrutinib works in treating patients with chronic lymphocytic leukemia or other low-grade B-cell non-Hodgkin lymphomas that have returned after a period of improvement (relapsed) or have not responded to treatment (refractory). Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Idelalisib and ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving pembrolizumab alone or with idelalisib or ibrutinib may be an effective treatment in patients with chronic lymphocytic leukemia or other low-grade B-cell non-Hodgkin lymphomas.
NCT02439138 ↗	Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia	Terminated	Gilead Sciences	Phase 2	2015-10-01	This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM).
NCT02439138 ↗	Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia	Terminated	Dana-Farber Cancer Institute	Phase 2	2015-10-01	This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM).

Clinical Trial Conditions for Zydelig

Condition Name

Condition Name for Zydelig
Intervention	Trials
Recurrent Small Lymphocytic Lymphoma	4
Recurrent Chronic Lymphocytic Leukemia	4
Waldenstrom Macroglobulinemia	3
Refractory Chronic Lymphocytic Leukemia	2

Condition MeSH

Condition MeSH for Zydelig
Intervention	Trials
Leukemia, Lymphoid	9
Leukemia	8
Leukemia, Lymphocytic, Chronic, B-Cell	8
Lymphoma	7

Clinical Trial Locations for Zydelig

Trials by Country

Trials by Country for Zydelig
Location	Trials
United States	39
Japan	10
France	6
United Kingdom	5
Korea, Republic of	3

Trials by US State

Trials by US State for Zydelig
Location	Trials
Oregon	4
Maryland	3
Washington	2
Massachusetts	2
Minnesota	2

Clinical Trial Progress for Zydelig

Clinical Trial Phase

Clinical Trial Phase for Zydelig
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	6
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for Zydelig
Clinical Trial Phase	Trials
Active, not recruiting	5
Terminated	3
Recruiting	2

Clinical Trial Sponsors for Zydelig

Sponsor Name

Sponsor Name for Zydelig
Sponsor	Trials
National Cancer Institute (NCI)	5
Gilead Sciences	5
OHSU Knight Cancer Institute	3

Sponsor Type

Sponsor Type for Zydelig
Sponsor	Trials
Other	20
Industry	8
NIH	5

Zydelig Market Analysis and Financial Projection

Last updated: April 27, 2026

ZYDELIG (Idelalisib): Clinical Trials Update, Market Analysis, and 5-Year Projection

What is ZYDELIG and who controls its global development and supply?

ZYDELIG is idelalisib, an oral PI3K delta (PI3Kδ) inhibitor used in defined B-cell malignancies and associated clinical settings (labeling depends on jurisdiction and line of therapy). Commercial and regulatory footprint is anchored by the original NDA/launch program and subsequent label updates that narrowed or clarified eligible patient populations.

Commercial reality (global): Idelalisib has moved from launch-era growth into a late-lifecycle, access-constrained portfolio position as oncology practice shifted toward BTK inhibitors and other targeted pathways, and as safety-focused prescribing and reimbursement decisions tightened over time.

What is the clinical-trials state for idelalisib now?

Idelalisib’s clinical development has largely transitioned from late-stage registrational work to smaller, label-support and mechanistic studies, with the bulk of new enrollment activity reduced versus the peak launch years. Trial continuity is concentrated in:

• Post-marketing and label-support studies (where required by regulators or to define long-term risk management)
• Exploratory combination trials in specific biomarker or disease subtypes
• Geography-limited or sponsor-limited follow-on studies, often with enrollment already completed

Key point for forecasting: For market projection, the credible assumption is that incremental clinical expansion is modest versus historical growth, and the near-term commercial trajectory is governed more by competition, safety governance, and payer restrictions than by new winning trials.

What do the major safety and risk-management constraints imply for trial design and uptake?

Idelalisib prescribing is constrained by PI3Kδ class safety liabilities (notably immune-mediated events) and by regulator-driven risk mitigation. This drives:

• stronger monitoring requirements in clinical protocols
• higher discontinuation risk in real-world use
• more conservative payer coverage thresholds

Commercial impact channel: tighter eligibility, clinician selectivity, and payer prior authorization reduce addressable market even when patients exist clinically.

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Clinical Trials Update: Snapshot of Ongoing vs. Completed Activity

Clinical trials are typically cataloged by registries and sponsor-sponsored pipelines. For a market-facing update, the relevant signal is not the existence of trials, but whether they are likely to expand labeled indications, improve safety profile, or displace standard-of-care comparators. For idelalisib, that signal is muted.

How should investors treat the current trial pipeline?

Treat the idelalisib pipeline as label maintenance and niche optimization, not as a franchise restart. The dominant drivers are:

• competition from BTK inhibitors and next-line regimens
• maturing sequencing algorithms in CLL and related B-cell malignancies
• protocol-level safety governance that affects patient retention
• payer and provider comfort shaped by historic risk signals

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Market Analysis: Where ZYDELIG sits in oncology spending

What patient segments drive idelalisib use?

Idelalisib’s demand has historically been tied to:

• relapsed/refractory B-cell malignancies where PI3Kδ inhibition provides efficacy after prior therapies
• specific clinical scenarios where the regimen matches guideline niches and label language

In practice, idelalisib uptake depends on:

• prior exposure patterns (especially in CLL)
• willingness to manage immune-mediated adverse events
• payer criteria that often restrict coverage to label-eligible populations and line-of-therapy definitions

What are the competitive threats?

Idelalisib faces intense competition from agents that have:

• stronger durability perception in real-world treatment
• simpler administration and fewer monitoring barriers
• entrenched guideline positions

The core competitive set is:

• BTK inhibitors across CLL and related indications
• other targeted pathways that reduce reliance on PI3Kδ inhibitors
• fixed-duration or regimens with established sequencing logic, which can displace PI3K-driven therapy

What does this mean for pricing, contracting, and reimbursement?

For mature and risk-constrained oncology brands:

• net pricing is pressured by payer contracting
• formulary access depends on prior authorization and documentation
• utilization declines often occur faster than headline incidence growth

Idelalisib’s late lifecycle dynamics typically mean volume erosion is not offset by meaningful price increases.

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Market Projection: 5-Year Outlook (2026–2030)

What is the projection framework for ZYDELIG?

A realistic projection uses three layers:

1. Addressable patients based on incidence and line-of-therapy eligible cohorts
2. Utilization fraction shaped by safety governance, guideline positioning, and competition
3. Net sales efficiency influenced by rebates, contracting outcomes, and displacement

Directionally: idelalisib is projected to keep shrinking in net sales as the market consolidates around competing oral targeted agents, with modest drag offset by remaining niche eligible patients.

5-year market projection table (directional, not label-expansion driven)

Because a fully numeric, jurisdiction- and payer-adjusted model requires current net price, current country share, and complete registry trial status, this projection is issued as a market-direction framework rather than a counterfeit precision forecast.

Metric	2026	2027	2028	2029	2030
ZYDELIG net sales trend	Decline	Decline	Decline	Low decline	Stabilize at lower run-rate
Primary demand driver	Remaining label-niche patients	Continued competition displacement	Safety-governed selectivity	Contracting and access stabilization	Reduced eligible pool dominates
Key limiter	BTK-led standard-of-care	Access tightening and switching	Discontinuation and monitoring burden	Further sequencing shift	Late lifecycle attrition

Interpretation for decision-makers: The growth case for idelalisib is weak; the decision is whether the residual franchise is still worth holding in a portfolio versus reallocating resources to next-generation assets.

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Strategic Implications for R&D, BD, and Investment

What actions follow from the clinical and market setup?

• R&D posture: focus on incremental, label-support objectives or combination rationalization that reduces discontinuation risk rather than aiming to restart a franchise.
• BD posture: pursue partnerships where idelalisib’s niche efficacy still aligns with payer-acceptable patient definitions.
• Investment posture: treat ZYDELIG as a late-cycle cashflow asset, not a high-upside clinical story.

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Key Takeaways

• ZYDELIG (idelalisib) sits in a late lifecycle position shaped by competition and safety governance, not by major new clinical expansion.
• The clinical-trials pattern is consistent with label maintenance and niche exploration, with limited expected market expansion.
• Over 2026–2030, the market outlook is downward-to-stabilizing, driven by shrinking eligible populations and continued displacement by competing targeted regimens.

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FAQs

1) Does ZYDELIG have an active registrational growth path right now?

No growth narrative is supported by the current pattern of trial activity; the effective commercial story is driven by access, competition, and safety governance.

2) What most determines real-world utilization for idelalisib?

Prior-therapy line eligibility, prescriber risk tolerance, monitoring burden, and payer authorization requirements.

3) Which competitor class most pressures idelalisib demand?

BTK inhibitors and other targeted oral regimens with stronger guideline entrenchment and operational convenience.

4) What is the dominant market risk for the next 5 years?

Further formulary restriction and patient switching as sequencing algorithms consolidate around competing therapies.

5) Where could idelalisib still find residual demand?

In narrow label-eligible niches where PI3Kδ inhibition remains a viable option and where safety governance can be executed reliably.

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Sources

[1] U.S. Food and Drug Administration. “Zydelig (idelalisib) prescribing information.” FDA label documentation.
[2] U.S. National Library of Medicine. ClinicalTrials.gov. Search results for idelalisib clinical trials (current public registry listings).
[3] EMA. European public assessment reports and product information for Zydelig (idelalisib). EMA documentation.
[4] NCCN Guidelines Insights and related guideline publications for CLL and B-cell malignancies (idelalisib position and sequencing context).