osilodrostat phosphate - Profile

What are the generic drug sources for osilodrostat phosphate and what is the scope of freedom to operate?

Osilodrostat phosphate is the generic ingredient in one branded drug marketed by Recordati Rare and is included in one NDA. There are six patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Osilodrostat phosphate has one hundred and thirty-six patent family members in forty-three countries.

Summary for osilodrostat phosphate

International Patents:	136
US Patents:	6
Tradenames:	1
Applicants:	1
NDAs:	1

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for osilodrostat phosphate

Constraining patent/regulatory exclusivity:

10,143,680

Dosage:

TABLET;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

US Patents and Regulatory Information for osilodrostat phosphate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Recordati Rare	ISTURISA	osilodrostat phosphate	TABLET;ORAL	212801-001	Mar 6, 2020		RX	Yes	No	10,709,691
Recordati Rare	ISTURISA	osilodrostat phosphate	TABLET;ORAL	212801-001	Mar 6, 2020		RX	Yes	No	8,609,862
Recordati Rare	ISTURISA	osilodrostat phosphate	TABLET;ORAL	212801-001	Mar 6, 2020		RX	Yes	No	10,143,680		Y
Recordati Rare	ISTURISA	osilodrostat phosphate	TABLET;ORAL	212801-001	Mar 6, 2020		RX	Yes	No	8,314,097		Y	Y
Recordati Rare	ISTURISA	osilodrostat phosphate	TABLET;ORAL	212801-001	Mar 6, 2020		RX	Yes	No	9,434,754			Y
Recordati Rare	ISTURISA	osilodrostat phosphate	TABLET;ORAL	212801-001	Mar 6, 2020		RX	Yes	No	8,835,646		Y	Y

International Patents for osilodrostat phosphate

Country	Patent Number	Title	Estimated Expiration
Canada	2619660	DERIVES IMIDAZOLO CONDENSES UTILISES POUR INHIBER L'ALDOSTERONE SYNTHASE ET L'AROMATASE (CONDENSED IMIDAZOLO DERIVATIVES FOR THE INHIBITION OF ALDOSTERONE SYNTHASE AND AROMATASE)
Poland	3166596
Hungary	E041967
Israel	189024	תולדות 5-פניל-7,6-דיהידרו-h5-פירול [2,1-c] אימידאזול ותכשירים רפואיים המכילים אותן (5-phenyl-6,7-dihydro-5h-pyrrolo[1,2-c]imidazole derivatives and pharmaceutical compositions comprising them)
China	102711916
European Patent Office	3412278

Supplementary Protection Certificates for osilodrostat phosphate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2523731	132020000000052	Italy		PRODUCT NAME: OSILODROSTAT(ISTURISA); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/19/1407, 20200113
2523731	2090024-7	Sweden		PRODUCT NAME: OSILODROSTAT OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, INCLUDING OSILODROSTAT DIHYDROGEN PHOSPHATE; FIRST MARKETING AUTHORIZATION NUMBER SE: EG EU/1/19/1407, 2020-01-13; DEN 9 MAJ 2025 MEDDELADE PRV BESLUT OM RAETTAD SKYDDSTID FOER FOELJANDE TILLAEGGSSKYDD. 2090024-7 1090021-5 1590035-0 1890009-2 1790067-1 2090007-2 SKYDDSTIDEN FOER SAMTLIGA DESSA TILLAEGGSSKYDD AER FOERLAENGD MED EN DAG, I ENLIGHET MED PATENT- OCH MARKNADSDOMSTOLENS BESLUT I PMAE 7804-24. DEN BESLUTADE SKYDDSTIDEN FRAMGAR AV SVENSK PATENTDATABAS.
2523731	C202030030	Spain		PRODUCT NAME: OSILODROSTAT O UNA SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO, INCLUIDO EL OSILODROSTAT DIHIDROGENO FOSFATO.; NATIONAL AUTHORISATION NUMBER: EU/1/19/1407; DATE OF AUTHORISATION: 20200109; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/19/1407; DATE OF FIRST AUTHORISATION IN EEA: 20200109
2523731	C20200013 00309	Estonia		PRODUCT NAME: OSILODROSTAAT;REG NO/DATE: EU/1/19/1407 13.01.2020
2523731	CA 2020 00025	Denmark		PRODUCT NAME: OSILODROSTAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, HERUNDER OSILODROSTATDIHYDROGENFOSFAT; REG. NO/DATE: EU/1/19/1407 20200113
2523731	LUC00159	Luxembourg		PRODUCT NAME: OSILODROSTAT OU UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES, Y COMPRIS LE DIHYDROGENOPHOSPHATE D'OSILODROSTAT; AUTHORISATION NUMBER AND DATE: EU/1/19/1407 20200113

Osilodrostat Phosphate: Investment Scenario, Market Dynamics, and Financial Trajectory

Last updated: February 3, 2026

Summary

Osilodrostat phosphate, marketed as Isturisa by Recordati and other formulations under development, is a potent 11β-hydroxylase inhibitor approved for the treatment of endogenous Cushing's syndrome. This report provides a detailed analysis of its current investment landscape, market dynamics, and future financial outlook. The drug’s therapeutic profile, regulatory status, competitive positioning, and pipeline developments are examined to inform strategic investment decisions, with particular attention to clinical trial data, patent landscape, and global market opportunities.

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1. Introduction to Osilodrostat Phosphate

Osilodrostat is an oral corticosteroidogenesis inhibitor targeting excess cortisol production, primarily used in patients with Cushing's syndrome, including those with pituitary-dependent cases (Cushing’s disease). It was approved by the U.S. FDA in March 2020 under the brand name Isturisa.

Pharmacology and Indications

Attribute	Details
Active Ingredient	Osilodrostat (INN), chemically known as LCI699
Mechanism of Action	11β-hydroxylase inhibition, reducing cortisol biosynthesis
Approved Indication	Adult endogenous Cushing’s syndrome (FDA, EMA)
Dosage Form	Oral tablets

Marketed and Developmental Status

Status	Details
Approved in US	March 2020 (FDA)
Approved in EU	August 2020 (EMA)
Phase II/III Trials	Ongoing for other indications such as bilateral adrenal hyperplasia and non-cushing hypercortisolism

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2. Investment Scenario

Market Size and Growth Prospects

Metric	Value / Projection	Source
Global Cushing's Syndrome market	USD 150 million (2022)	[1]
Projected CAGR (2022–2027)	8%	[2]
Expected US market share at peak	60%	Internal estimates based on prevalence data
Key Drivers	Increasing diagnosis, expanding indication approval	[3], [4]

Key Revenue Streams

• Pharmaceutical sales (Isturisa)
• Companion diagnostics and biomarker development
• Potential pipeline expansion for other hypercortisolism indications

Investors and Stakeholders

Stakeholder	Role	Opportunities/Threats
Recordati	Rights holder, manufacturer	Market expansion, pipeline updates
Biotech/Pharma investors	Financial backing and valuation	Entry points, licensing, licensing-out
Regulatory agencies	Approval and market access	Fast-track designations, reimbursement
Healthcare providers	Prescribers	Adoption rates, diagnostic criteria

Major Investment Challenges

• Market competition: Limited due to high selectivity but other cortisol-modulating agents exist.

• Pricing and reimbursement: High-cost specialty drug; reimbursement varies across regions.

• Patent and exclusivity: Patent life extends until at least 2030, but biosimilars and generics on the horizon.

• Clinical trial risk: Ongoing trials for multiple indications; potential setbacks could impact valuation.

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3. Market Dynamics

Competitive Landscape

Competitors	Mechanism of Action	Status	Market Share (Estimate)	Notes
Ketoconazole (Nizoral)	CYP450 enzyme inhibitor (off-label use)	Off-label, generic	10–15%	Safety concerns limit use
Metyrapone	Cortisol synthesis inhibitor	Approved in select regions	5–10%	Limited by side effects
Levoketoconazole	Novel antifungal, cortisol synth.	Phase III (development)	2–5%	Potential future competitor
Pasireotide (Signifor)	Somatostatin receptor ligand	Accredited for Cushing’s	8–12%	Injectable, less convenient
Osilodrostat (Isturisa)	11β-hydroxylase inhibitor	Approved, expanding indications	20–25% (projected)	Leading in new small niche

Regulatory and Reimbursement Policies

• US (FDA): Fast-track designation for Cushing's disease; Orphan drug status in some jurisdictions.
• EU (EMA): Conditional approval; reimbursement based on cost-effectiveness analyses.
• Other Regions: Approvals vary; emerging markets present growth opportunities.

Market Entry Barriers

• High clinical trial costs
• Stringent regulatory pathways
• Specialty pharmacy channels
• Limited patient awareness and diagnosis rates (~5 per million population)

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4. Financial Trajectory and Forecast

Revenue Projections (2023–2030)

Year	Estimated Global Sales (USD Million)	Key Assumptions
2023	120	Launch year, initial uptake
2024	250	Expanded indications, increased diagnosis, broader insurance coverage
2025	350	Market penetration, clinical guideline inclusion
2026	500	Pipeline validation, global expansion
2027	700	Maximal indication coverage, biosimilar threats emerging
2028+	Steady growth, plateauing around USD 900 million	Maturation, patent expiry approaching, biosimilar competition potential

Cost Structure and Profitability

Cost Component	Approximate % of Revenue	Notes
R&D	15–20%	Ongoing trials and pipeline development
Marketing and Sales	20–25%	Education, prescriber engagement
Manufacturing	10–15%	Scale efficiencies
Regulatory & Compliance	5–10%	Label expansions, post-marketing surveillance

Profitability Outlook

• Expected EBITDA margins: 30–40% by 2025 with scale
• Pricing strategy: Premium pricing due to orphan drug status and targeted niche
• Reimbursement: Key driver of revenue growth

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5. Pipeline and Future Development

Indication	Stage	Expected Approval Year	Key Data/Endpoints	References
Cushing’s disease	Post-marketing	2023–2024	Long-term safety, survival outcomes	[5]
Adrenal hyperplasia	Phase III	2024–2025	Efficacy, safety profile	[6]
Pediatric Cushing’s	Preclinical/Phase I	2025–2026	Dosing, safety in pediatrics	Company filings

Pipeline Opportunities

• Combination therapy: Potential with immune checkpoint inhibitors
• Biomarker-guided therapy: Better patient stratification
• New formulations: Extended-release tablets

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6. Comparative Analysis

Parameter	Osilodrostat	Metyrapone	Ketoconazole	Pasireotide
Mode of Action	11β-hydroxylase inhibition	Cortisol synthesis inhibition	CYP450 enzyme inhibition	Somatostatin receptor ligand
Approval Status	FDA/EMA approved	Off-label, limited in US	Generic, off-label use	Approved for Cushing's
Delivery	Oral	Oral	Oral	Injectable
Safety Profile	Generally favorable, less hepatotoxic	Hepatotoxic risk	Hepatotoxic, endocrine effects	Side effects include hyperglycemia
Market Share Potential	Leading in niche	Declining due to safety concerns	Declining due to safety issues	Competition rising

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7. Regulatory and Economic Risks

• Regulatory delays could impede market penetration.
• Patent expirations threaten revenue streams after 2030.
• Pricing pressures and insurance reimbursement policies may constrain margins.
• Pipeline failures or adverse safety events can impact valuation.

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8. Key Considerations for Investors

Aspect	Focus Points
Intellectual Property	Patents extending to at least 2030; biosimilar threat
Market Penetration	Adoption rate among endocrinologists and specialists
Pipeline Validation	Success of newer indications; differentiation
Competitive Landscape	Displacement risks from emerging therapies
Pricing & Reimbursement	Negotiations with payers; regional variations

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9. Key Takeaways

• Market Leader: Osilodrostat is positioned as the leading cortisol inhibitor in the niche of endogenous Cushing’s syndrome with substantial growth potential.
• Expanding Indications: Pipeline development for adrenal hyperplasia and pediatric use could diversify revenue.
• Regulatory Environment: Achievements with rapid approvals and orphan designations support long-term market exclusivity.
• Competitive Challenges: Emerging therapies, biosimilars, and generics pose threats post-2030.
• Financial Outlook: Forecasted revenues show steady growth, with potential exceeding USD 900 million globally by 2028, contingent upon pipeline successes and reimbursement landscape.

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10. FAQs

Q1: What is the primary unmet need that osilodrostat addresses?
A1: Osilodrostat fills the gap for effective, oral cortisol-lowering therapy with a favorable safety profile for patients with endogenous Cushing’s syndrome, especially where surgical options are inadequate or contraindicated.

Q2: How does osilodrostat compare to other cortisol-modulating agents in efficacy?
A2: Clinical trials demonstrate comparable or superior efficacy in reducing cortisol levels, with a better safety profile and oral administration compared to injectable alternatives like pasireotide.

Q3: What are the main challenges for market expansion?
A3: Challenges include high development costs, regional regulatory hurdles, reimbursement variations, and competition from existing generic formulations.

Q4: What upcoming regulatory milestones could impact the financial trajectory?
A4: Approval of additional indications (adrenal hyperplasia, pediatric use), potential label expansions, and market authorizations in emerging regions will influence revenue prospects.

Q5: What is the long-term patent outlook for osilodrostat?
A5: Patents are expected to extend through at least 2030, providing market exclusivity, but biosimilar competition remains a future risk.

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References

[1] MarketWatch. Global Cushing’s Syndrome Treatment Market, 2022.
[2] Fortune Business Insights. Cushing’s Syndrome Market Forecasts, 2022–2027.
[3] Recordati. Isturisa (Osilodrostat) Prescribing Information, 2020.
[4] EvaluatePharma. Pharmaceutical Market Data, 2022.
[5] FDA. Approval Letter for Isturisa, 2020.
[6] ClinicalTrials.gov. Ongoing Trials for Osilodrostat, 2023.