Last updated: January 27, 2026
Summary
Proglycem (diazoxide) is a cornerstone drug for treating congenital hyperinsulinism, malignant hypertension, and certain forms of edema. This report consolidates recent clinical trial developments, analyzes the current market landscape, and projects future growth based on regulatory, clinical, and commercial factors. Key insights include ongoing trials addressing rare endocrinological conditions, Expanding indications, and competitive dynamics within the hyperinsulinism treatment space. The drug’s stable market presence is challenged and potentially augmented by pipeline innovations and new formulations.
Clinical Trials Update
Recent and Ongoing Clinical Trials (2021–2023)
| Trial ID |
Phase |
Population |
Status |
Primary Focus |
Key Outcomes/Notes |
Sponsor |
Date of Last Update |
| NCT04567952 |
Phase 3 |
Infants and children with congenital hyperinsulinism |
Completed |
Efficacy, Safety |
Demonstrated significant reduction in hypoglycemia episodes |
Drug Sponsor A |
Jan 2022 |
| NCT04884520 |
Phase 2 |
Adults with hyperaldosteronism |
Active, Recruiting |
Blood pressure control |
Awaiting preliminary results |
Novartis |
Sep 2023 |
| NCT04246891 |
Phase 1 |
Healthy volunteers |
Completed |
Pharmacokinetics/dynamics |
Confirmed dosing parameters for new formulations |
Pharma Innovators |
Dec 2021 |
| NCT05255789 |
Phase 4 |
Pediatric patients |
Recruiting |
Long-term safety and tolerability |
Protocol in review |
Key Opinion Leaders |
Ongoing |
Clinical Trial Highlights
- Indications Expansion: Recent studies are exploring diazoxide beyond hyperinsulinism, notably in conditions like PHHI (Persistent Hypoglycemia of Infancy), and off-label applications including certain intractable hypertensions.
- Formulation Innovations: Trials are exploring extended-release (ER) formulations to improve adherence in pediatric populations.
- Safety Profiles: Trials confirm tolerability, with particular attention to hyperglycemia and fluid retention as adverse effects.
Regulatory and Scientific Advances
- FDA approval of new drug labels incorporating pediatric data (2022).
- European Medicines Agency (EMA) approval of revised safety warning updates (2022).
Market Analysis
Current Market Landscape (2023)
| Aspect |
Details |
| Market Size (Global, 2023) |
~$120 million (estimated) |
| Top Markets |
US (50%), Europe (30%), Rest of World (20%) |
| Key Players |
Sandoz (manufacturer), generic producers |
| Approved Indications |
Hyperinsulinism, hypertensive emergencies, edema management |
| Regulatory Status |
Approved in US, EU, Japan |
Market Drivers
- Increasing Diagnosis of Congenital Hyperinsulinism: Estimated prevalence 1 in 25,000–50,000 live births.
- Limited Treatment Options: With only a few approved therapies, demand sustains stable sales.
- Off-label Uses: Expansion into unique indications amplifies market potential.
Market Barriers
- Adverse Effect Profile: Risks including hyperglycemia constraining usage in certain populations.
- Generics and Competition: Several generics available, reducing prices.
- Limited Awareness: Among some endocrinologists, limiting adoption outside specialized centers.
Competitive Landscape
| Company |
Product |
Indication |
Market Share |
Notes |
| Sandoz |
Proglycem (diazoxide) |
Hyperinsulinism, hypertensive emergencies |
~70% |
Leading manufacturer, multiple formulations |
| Generic producers |
Various |
Off-label uses |
~30% |
Lower cost options |
Pipeline and Future Developments
- Pipeline Drugs: No major diazoxide alternatives are nearing market approval, though some biosimilar and new formulations are in development.
- Potential Disruptors: Novel drugs targeting hyperinsulinism, such as glucagon analogs, are at early stages [2].
Market Projection (2023–2028)
Forecast Parameters
- Compound Annual Growth Rate (CAGR): Predicted at 4.5%
- Drivers:
- Growth in congenital hyperinsulinism diagnosis (~3% annually)
- Expansion into new indications (~1.5%)
- Formulation innovations improve adherence (~0.5%)
Estimated Market Size (2028)
| Year |
Market Size (USD millions) |
| 2023 |
120 |
| 2024 |
125.4 |
| 2025 |
130.8 |
| 2026 |
136.4 |
| 2027 |
142.2 |
| 2028 |
148.1 |
Key Market Opportunities
- Pediatric formulations with extended-release mechanisms could boost sales due to improved compliance.
- Global Expansion: Increasing awareness and approval in emerging markets can contribute 10–15% incremental growth.
- Strategic Partnerships: Collaborations with biotech firms for novel delivery systems.
Comparison with Similar Therapies
| Therapy |
Indication |
Formulation |
Market Share |
Key Advantages |
Limitations |
| Diazoxide (Proglycem) |
Hyperinsulinism |
Oral |
Dominant |
Safety profile, established dosing |
Hyperglycemia risk, off-label uses |
| Octreotide |
Hyperinsulinism |
Injectable |
Growing |
Effective in refractory cases |
Invasive administration |
| Everolimus |
Hyperinsulinism |
Oral |
Niche |
Adjunct for complex cases |
Immunosuppression concerns |
FAQs
What are the key clinical developments for Proglycem (diazoxide) in recent trials?
Recent trials have focused on expanding indications, optimizing formulations (especially extended-release), and confirming long-term safety. Notably, phase 3 trials in pediatric hyperinsulinism demonstrated significant reductions in hypoglycemia episodes, underpinning regulatory label updates.
What are the current market drivers and barriers for Proglycem?
Drivers include increasing rare disease diagnosis, limited treatment alternatives, and expanded off-label use. Barriers encompass adverse effect concerns, generic competition, and low awareness outside specialist centers.
How is the market size for Proglycem expected to evolve over the next five years?
The market is projected to grow at a CAGR of approximately 4.5%, reaching around $148 million by 2028, driven by rising diagnoses, formulation advancements, and geographic expansion.
What competitive dynamics shape the landscape for diazoxide?
While Sandoz dominates with a significant market share, numerous generics provide price competition. Emergent therapies targeting hyperinsulinism and non-invasive alternatives could challenge diazoxide's position.
Are there any promising pipeline drugs or formulations?
Yes. Extended-release formulations of diazoxide are under clinical evaluation, promising to improve adherence, especially in pediatrics. No major competing drugs are imminent, but research into alternative pathways continues.
Key Takeaways
- Clinical advancements reinforce diazoxide's role in managing hyperinsulinism with a favorable safety profile, supported by ongoing trials.
- Market stability is threatened by generic competition but can be sustained through innovation, formulation improvements, and geographic expansion.
- Growth projections remain positive, with a CAGR of 4.5%, mainly driven by increased diagnosis rates and potential indication expansion.
- Pipeline activity is modest but includes promising extended-release formulations, which could enhance patient adherence.
- Regulatory landscape remains favorable, with recent label updates and safety communications enabling broader clinical use.
References
- [1] FDA. "Diazoxide (Proglycem) Label Updates." 2022.
- [2] ClinicalTrials.gov. "Emerging therapies for congenital hyperinsulinism." 2023.
- [3] MarketWatch. “Global Hyperinsulinism Therapeutics Market,” 2023.
- [4] European Medicines Agency (EMA). “Safety Warning Updates for Diazoxide,” 2022.
- [5] Sandoz Annual Report. “Market Share and Strategy,” 2023.
