vorasidenib - Profile

What are the generic sources for vorasidenib and what is the scope of patent protection?

Vorasidenib is the generic ingredient in one branded drug marketed by Servier and is included in one NDA. There are five patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Vorasidenib has one hundred and two patent family members in thirty-nine countries.

Summary for vorasidenib

International Patents:	102
US Patents:	5
Tradenames:	1
Applicants:	1
NDAs:	1

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

Constraining patent/regulatory exclusivity:

TREATMENT OF ADULT AND PEDIATRIC PATIENTS 12 YEARS AND OLDER WITH GRADE 2 ASTROCYTOMA OR OLIGODENDROGLIOMA WITH A SUSCEPTIBLE ISOCITRATE DEHYDROGENASE-1 (IDH1) OR ISOCITRATE DEHYDROGENASE-2 (IDH2) MUTATION FOLLOWING SURGERY INCLUDING BIOPSY, SUB-TOTAL RESECTION, OR GROSS TOTAL RESECTION

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 vorasidenib

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No			Y	Y
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No			Y	Y
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No				Y
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No
Servier	VORANIGO	vorasidenib	TABLET;ORAL	218784-001	Aug 6, 2024		RX	Yes	No

International Patents for vorasidenib

Country	Patent Number	Title	Estimated Expiration
Israel	274123
New Zealand	754946	Therapeutically active compounds and their methods of use
Argentina	131555
Brazil	122017014843	métodos para preparar compostos úteis para tratamento de câncer
Japan	2021121628	治療活性化合物およびそれらの使用方法 (THERAPEUTICALLY ACTIVE COMPOUNDS AND THEIR METHODS OF USE)
Argentina	096902	DERIVADOS DE TRIAZINA Y PIRIMIDINA COMO INHIBIDORES DE LA IDH1 Y/O IDH2 MUTANTES Y SU USO EN EL TRATAMIENTO DEL CÁNCER
Japan	6538037

Supplementary Protection Certificates for vorasidenib

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3019483	301370	Netherlands		PRODUCT NAME: VORASIDENIB OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT OF HYDRAAT DAARVAN; REGISTRATION NO/DATE: EU/1/25/1912 20250919
3019483	C20265008	Finland
3019483	PA2026510	Lithuania		PRODUCT NAME: VORASIDENIBAS ARBA JO FARMACINIU POZIURIU PRIIMTINA DRUSKA ARBAHIDRATAS; REGISTRATION NO/DATE: EU/1/25/1912 20250917
3019483	CR 2026 00011	Denmark		PRODUCT NAME: VORASIDENIB ELLER ET FARMACEUTISK ACCEPTABELT SALT ELLER HYDRAT DERAF; REG. NO/DATE: EU/1/25/1912 20250919
3019483	C03019483/01	Switzerland		PRODUCT NAME: VORASIDENIB; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 69364 15.11.2024

Investment Scenario and Fundamentals Analysis for Vorasidenib

Last updated: February 21, 2026

Vorasidenib is a selective dual inhibitor targeting mutant isocitrate dehydrogenase 1 and 2 (IDH1/2). It is being developed primarily for patients with IDH1/2-mutant low-grade gliomas (LGGs). This assessment covers its clinical prospects, market potential, competitive landscape, and key risks.

Clinical Development and Regulatory Status

Vorasidenib is in late-stage clinical trials, chiefly Phase 3, with pivotal data expected in 2023-2024. It has received Orphan Drug Designation from the FDA for LGGs.

• Key trials: The IDHENTIFY trial (NCT03684811) is a randomized, placebo-controlled Phase 3 study evaluating vorasidenib's efficacy and safety.
• Regulatory prospects: Success depends on demonstrating significant progression-free survival (PFS) benefits. Accelerated approval potential exists given the orphan status and unmet need in LGGs.

Market Dynamics and Commercial Potential

Indications and Patient Population

• Low-grade gliomas (LGGs): A subset of gliomas that typically affect individuals aged 30-40 years.
• Market size estimates:
  • Approximately 10,000 new LGG cases annually in the US and EU.
  • Estimated 60% harbor IDH mutations, translating to roughly 6,000 patients/year.
  • Only a fraction are eligible for investigational therapies due to prior treatments or health status.

Competitive Landscape

• Current standard of care: Surgical resection, radiotherapy, chemotherapy (e.g., temozolomide).
• Competing drugs: Other IDH inhibitors like ivosidenib (IDH1) and enasidenib (IDH2), approved for AML but with emerging data in gliomas.
• Vorasidenib differentiators: Dual inhibition may translate into better efficacy in IDH1/2-mutant tumors.

Pricing and Reimbursement

• Pricing benchmarks:
  • IDH inhibitors for AML are priced at ~$150,000–200,000 annually.
  • Oncology drugs for rare tumors command premiums, especially with accelerated approval.
• Reimbursability: Coverage likely hinges on demonstrated clinical benefit and orphan drug designation.

Financial and Commercial Risks

• Clinical risk: Failure to meet primary endpoints in Phase 3 may halt development.
• Regulatory risk: Regulatory agencies may demand additional data, delaying approval.
• Market risk: Competition from other IDH inhibitors and emerging therapies can limit market penetration.
• Pricing and reimbursement risk: Reimbursement decisions may restrict access if clinical benefit evidence is inadequate.

Intellectual Property and Licensing

• Patents: Proprietary compounds and formulation patents expected to secure exclusivity until at least 2030.
• Licensing agreements: Possible collaborations with larger pharma firms to fund commercialization; beneficial in markets with weak infrastructure or reimbursement barriers.

Financial Outlook and Investment Considerations

• Valuation prospects: High risk, high reward—potentially valuing the candidate based on market size, clinical data, and approval timing.
• Partnership opportunities: Pharmas with development and commercial experience in neuro-oncology may seek partnerships.
• Funding needs: Ongoing clinical trial costs estimated at $50–100 million to bring to approval.

Key Risks and Uncertainties

Risk Type	Impact	Mitigation Strategy
Clinical failure	Loss of investment	Continue rigorous trial design; diversify pipeline
Regulatory delays	Postponed market entry	Engage early with regulators; adaptive trial designs
Competitive pressure	Reduced market share	Position as first-in-class; demonstrate superior efficacy
Pricing and reimbursement	Limited revenue	Generate robust evidence of clinical benefit

Conclusion

Investing in vorasidenib entails significant scientific and regulatory risks given the preliminary clinical stage. Its market opportunity depends on successful trial outcomes, regulatory approval, and favorable reimbursement landscapes in oncology niches with high unmet needs. Strategic partnerships and intellectual property protection bolster its potential long-term value.

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Key Takeaways

• Vorasidenib is a dual IDH1/2 inhibitor in late-stage development for IDH-mutant LGGs.
• The therapy targets a small but growing market with high unmet medical need.
• Success hinges on Phase 3 trial results, regulatory approval, and reimbursement conditions.
• Competitive pressures from existing IDH inhibitors are significant but may be mitigated by dual inhibition prospects.
• The investment risk remains high until clinical efficacy is established, but the potential market size warrants ongoing monitoring.

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FAQs

1. What makes vorasidenib different from other IDH inhibitors?
It targets both IDH1 and IDH2 mutations, potentially offering broader efficacy in IDH-mutant tumors compared to selective inhibitors.

2. What is the significance of Orphan Drug Designation?
It provides incentives like market exclusivity, fee waivers, and tax credits, which can improve commercialization prospects for rare diseases.

3. How does the clinical trial progress affect the investment outlook?
Positive Phase 3 data increases the likelihood of approval and market success; delays or failures diminish valuation prospects.

4. What are the major hurdles in commercializing vorasidenib?
Regulatory approval, demonstrating a clear clinical benefit, pricing negotiations, and competing therapies.

5. What is the potential size of the target market?
Approximately 6,000 patients annually in the US and EU, with a subset eligible for targeted therapy after diagnosis.

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References

1. National Cancer Institute. (2023). Low-grade glioma. https://seer.cancer.gov
2. FDA. (2023). Orphan Drug Designations. https://www.fda.gov
3. ClinicalTrials.gov. (2023). NCT03684811.
4. IQVIA. (2023). Oncology drug pricing data.
5. Deloitte. (2022). Oncology market analysis.