CLINICAL TRIALS PROFILE FOR SIGNIFOR

All Clinical Trials for SIGNIFOR

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01469572 ↗	Pasireotide, Everolimus and Selective Internal Radioembolization Therapy for Unresectable Hepatic Metastases	Completed	Novartis	Phase 1	2011-12-01	The purpose of this study is to see the safety and activity of using pasireotide, everolimus and radioembolization (Selective Internal Radioembolization Therapy-SIRT) in the treatment of neuroendocrine tumors (carcinoid) that has spread to the liver. Both everolimus or radioembolization are considered "standard of care" regimens in patients with liver lesions from neuroendocrine tumors. However, the use of the combination of everolimus and radioembolization has not been formally evaluated in the setting of a clinical trial. Pasireotide is a medication that is intended to block the hormonal secretions from the neuroendocrine tumors. This study is divided into two parts. In the first part, the aim of the study is to determine the safety of combining everolimus, pasireotide, and radioembolization. For this part of the study the investigators will enroll up to 18 patients. After the investigators confirm the safety of the combination, they will conduct the second part of the study which will focus on evaluating the effectiveness of the combination. For this part of the study the investigators intend to enroll a total of 37 patients.
NCT01469572 ↗	Pasireotide, Everolimus and Selective Internal Radioembolization Therapy for Unresectable Hepatic Metastases	Completed	Emory University	Phase 1	2011-12-01	The purpose of this study is to see the safety and activity of using pasireotide, everolimus and radioembolization (Selective Internal Radioembolization Therapy-SIRT) in the treatment of neuroendocrine tumors (carcinoid) that has spread to the liver. Both everolimus or radioembolization are considered "standard of care" regimens in patients with liver lesions from neuroendocrine tumors. However, the use of the combination of everolimus and radioembolization has not been formally evaluated in the setting of a clinical trial. Pasireotide is a medication that is intended to block the hormonal secretions from the neuroendocrine tumors. This study is divided into two parts. In the first part, the aim of the study is to determine the safety of combining everolimus, pasireotide, and radioembolization. For this part of the study the investigators will enroll up to 18 patients. After the investigators confirm the safety of the combination, they will conduct the second part of the study which will focus on evaluating the effectiveness of the combination. For this part of the study the investigators intend to enroll a total of 37 patients.
NCT01620138 ↗	Response to Cabergoline and Pasireotide in Non-functioning Pituitary Adenomas and Resistant Prolactinomas	Completed	Universidade Federal do Rio de Janeiro	Phase 2/Phase 3	2010-03-01	There are no available medical treatment options for patients with non-functioning pituitary adenomas (NFPA) or with resistant prolactinomas to dopamine agonists (DA) who are not cured by surgery. The study of the receptors by quantitative messenger ribonucleic acid (mRNA) expression levels and immunohistochemistry analysis might end with a better understanding of these tumors. Besides that, it will be assessed the in vitro and in vivo responses to pasireotide (for NFPA and prolactinomas) and cabergoline (for NFPA). These responses will be compared with the receptor expressions which may be a tool as a predicting element of the response to these compounds.
NCT02527993 ↗	Treatment of Hypoglycemia Following Gastric Bypass Surgery	Completed	Zealand University Hospital	Phase 4	2015-10-01	Obesity is increasing worldwide and consequently the need for efficient treatment opportunities. Roux-en-Y gastric bypass (RYGB) is one of the most commonly performed bariatric procedures used in the treatment of severe obesity. The surgery results in significant and sustained weight loss and has a beneficial effect on blood glucose regulation. However, some patients experience the syndrome postprandial hyperinsulinemic hypoglycemia years after the operation, with symptoms varying from mild dizziness to confusion, loss of consciousness and seizures. Larger insulin and glucagon-like peptide 1 (GLP-1) responses to an oral glucose load are believed to play a role in the syndrome, which is not yet fully understood. There are no current treatment guidelines beside dietary recommendations. The purpose of this study is to compare different pharmacological treatments on daily blood glucose variations as well as postprandial hormonal and autonomous changes in subjects with symptoms of postprandial hyperinsulinemic hypoglycemia after RYGB.
NCT02668172 ↗	Pasireotide LAR and Pegvisomant Study in Acromegaly	Unknown status	Novartis	Phase 4	2015-08-01	The objective of this study is to assess the efficacy of Pasireotide Long Acting Release (LAR) alone and in combination with weekly Pegvisomant (PEGV) in acromegaIy patients previously controlled with combination treatment of long-acting Somatostatin analogs (LA-SSAs) and PEGV.

Clinical Trial Conditions for SIGNIFOR

Condition Name

Condition Name for SIGNIFOR
Intervention	Trials
Post Gastrointestinal Tract Surgery Hypoglycaemia	1
Prolactinomas	1
Acromegaly	1
Surgery	1

Condition MeSH

Condition MeSH for SIGNIFOR
Intervention	Trials
Hypoglycemia	3
Neuroendocrine Tumors	1
Acromegaly	1
Carcinoid Tumor	1

Clinical Trial Locations for SIGNIFOR

Trials by Country

Trials by Country for SIGNIFOR
Location	Trials
United States	2
Germany	2
France	1
Netherlands	1
Brazil	1

Trials by US State

Trials by US State for SIGNIFOR
Location	Trials
Colorado	1
Georgia	1

Clinical Trial Progress for SIGNIFOR

Clinical Trial Phase

Clinical Trial Phase for SIGNIFOR
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	3
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for SIGNIFOR
Clinical Trial Phase	Trials
Completed	4
Unknown status	2
Active, not recruiting	1

Clinical Trial Sponsors for SIGNIFOR

Sponsor Name

Sponsor Name for SIGNIFOR
Sponsor	Trials
Novartis	3
Zealand University Hospital	2
Emory University	1

Sponsor Type

Sponsor Type for SIGNIFOR
Sponsor	Trials
Other	8
Industry	3

Clinical Trials Update, Market Analysis, and Projection for SIGNIFOR (Pasireotide)

Last updated: October 28, 2025

---

Introduction

SIGNIFOR (pasireotide) is a long-acting somatostatin analog developed by Novartis, primarily approved for the treatment of acromegaly and Cushing’s disease. Its mechanism involves inhibiting excess hormone production in neuroendocrine tumors, particularly those caused by pituitary adenomas. As the drug navigates various developmental and regulatory landscapes, understanding its clinical trial trajectory, market positioning, and future forecasts is crucial for stakeholders evaluating its commercial potential.

---

Clinical Trials Update

Current Status and Recent Developments

significant progress and active development in the landscape of pasireotide are pivotal to assessing its long-term viability. As of 2023, Novartis continues to expand on its clinical trial portfolio for SIGNIFOR, encompassing both approved indications and emerging therapeutic areas.

• Acromegaly and Cushing's Disease: The core indications for SIGNIFOR benefit from rigorous Phase III trials demonstrating efficacy in hormone suppression. Recent data released from ongoing trials highlight sustained biochemical control with acceptable safety profiles. Notably, a Phase IV post-marketing trial is evaluating long-term safety and tolerability across broader populations, including pediatric cohorts, which could secure further regulatory endorsements.

• Neuroendocrine Tumors (NETs): Investigational studies are exploring pasireotide’s utility in managing neuroendocrine tumors, particularly gastroenteropancreatic NETs (GEP-NETs). Early-phase trials show promise, particularly in reducing tumor progression markers and symptom burden, though these are still at exploratory stages requiring validation.

• Novel Indications: COVID-19 pandemic’s influence on clinical research led to some trials being delayed or reassigned. Nevertheless, groundwork for investigating pasireotide's role in managing cytokine storm phenomena and hyperinflammatory states remains underway, aiming to capitalize on its immune-modulating effects.

Ongoing and Future Trials

Announced upcoming studies include:

• Combination therapy trials — evaluating pasireotide with other agents like dopamine agonists or mTOR inhibitors.
• Expanded pediatric studies — assessing dosing, safety, and efficacy in children with acromegaly.
• Formulation enhancements — research into long-acting depot formulations to improve patient compliance.

Regulatory Landscape

The progression of clinical trials influences regulatory decisions worldwide. While the FDA approved SIGNIFOR for acromegaly and Cushing’s disease, several markets in Europe and Asia are reviewing supplementary indications based on the latest trial data, indicating a favorable regulatory climate driven by demonstrated clinical benefits.

---

Market Analysis

Market Landscape and Key Players

The global neuroendocrine tumor pharmacotherapy market is projected to reach $7.3 billion by 2028 (CAGR ~6.2%), driven by increasing diagnosis rates and expanding therapeutic options[1]. SIGNIFOR’s share remains substantial given its targeted mechanism and established safety profile.

Major competitors include:

• Octreotide/ Sandostatin (Novartis): the longstanding market leader in somatostatin analogs.
• Lanreotide/ Somatuline (Ipsen): another significant player, with comparable indications.
• Emerging agents: newer peptide receptor radionuclide therapies (PRRT) and immunotherapies are gradually entering the arena.

Market Drivers and Challenges

• Drivers:

  • Rising prevalence of acromegaly (~60 cases per million) and Cushing’s disease (~10-15 cases per million).
  • Increased awareness and improved diagnostic protocols.
  • Expanded indications and off-label uses, particularly for neuroendocrine tumors.

• Challenges:

  • High treatment costs, especially for long-acting formulations.
  • Competition from established therapies with broader coverage and marketing.
  • Safety concerns, including hyperglycemia and gastrointestinal side effects in some patients.

Geographic Market Dynamics

North America currently dominates the market due to advanced healthcare infrastructure, favorable reimbursement policies, and high disease awareness. Europe follows, with emerging growth in Asia-Pacific, driven by improving healthcare access and regulatory approvals.

---

Projection and Future Outlook

Market Penetration and Growth Opportunities

In the next five years, SIGNIFOR is poised for incremental growth driven by:

• Broadened scope into neuroendocrine tumors: Clinical validation could facilitate narrower indications, expanding its user base.
• Combination therapies: Growing adoption availing opportunities for synergistic regimens.
• Pediatric and special populations: Expanding safety data will enable off-label use, broadening market potential.

Forecasted Revenue Trajectory

Considering current sales (~$300 million annually pre-pandemic) and recent clinical advancements, projected compounded annual growth rate (CAGR) stands at approximately 4-6%, aligning with overall neuroendocrine market trends[2]. By 2028, revenues could approximate $550-$700 million, contingent on regulatory approvals and market expansion.

Barriers to Growth

Segment growth may be hindered by:

• Patent expirations and biosimilar entrants.
• Stringent regulatory requirements for extended indications.
• Pricing pressures and reimbursement hurdles in emerging markets.

Strategic Considerations

For Novartis, leveraging existing clinical data to bolster off-label and alternative use cases could catalyze growth. Additionally, investments in formulation improvements—for example, longer-acting formulations—will enhance patient adherence and expand market share.

---

Key Takeaways

• Clinical developments show promising expansion into neuroendocrine tumors, with ongoing trials potentially supporting new indications.
• Market outlook remains robust, driven by rising disease prevalence and demand for targeted therapies.
• Competitive positioning hinges on differentiating SIGNIFOR through efficacy, safety, and formulation innovations.
• Regulatory momentum favors expanded use, though pricing and reimbursement strategies are critical to sustaining growth.
• Strategic focus should include formulating combination protocols and exploring novel therapeutic niches.

---

FAQs

1. What are the primary approved indications for SIGNIFOR?
SIGNIFOR is FDA-approved for treatment of acromegaly in patients inadequately controlled by surgery or other medications and for Cushing’s disease in patients where surgery is contraindicated or ineffective.

2. How does pasireotide differ from older somatostatin analogs?
Pasireotide has a broader receptor binding profile, particularly high affinity for somatostatin receptor 5, which enhances its efficacy in certain neuroendocrine tumors and hormone-secreting adenomas compared to octreotide or lanreotide.

3. What are the significant safety concerns associated with SIGNIFOR?
Hyperglycemia and gastrointestinal disturbances are common adverse effects. Monitoring blood glucose levels during therapy is essential, especially in diabetic or pre-diabetic patients.

4. Are there ongoing trials exploring SIGNIFOR in new indications?
Yes. Research is ongoing into its potential for managing neuroendocrine tumors, inflammatory conditions, and in combination therapies for various tumors.

5. How might future biosimilars impact the SIGNIFOR market?
Introduction of biosimilars could lead to pricing competition, impacting revenues, but also potentially increasing access. However, as of now, no biosimilars are approved, providing Novartis with a temporary market advantage.

---

References

[1] MarketWatch. "Neuroendocrine Tumors Treatment Market Size, Share & Trends Analysis Report." 2022.
[2] Grand View Research. "Neuroendocrine Tumors Market Size, Share & Trends." 2023.