TOVORAFENIB - Generic Drug Details

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

Tovorafenib is the generic ingredient in one branded drug marketed by Day One Biopharms and is included in two NDAs. There are two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Tovorafenib has forty-nine patent family members in twenty-seven countries.

One supplier is listed for this compound.

Summary for TOVORAFENIB

International Patents:	49
US Patents:	2
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	7
Patent Applications:	669
What excipients (inactive ingredients) are in TOVORAFENIB?	TOVORAFENIB excipients list
DailyMed Link:	TOVORAFENIB at DailyMed

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

Constraining patent/regulatory exclusivity:

TREATMENT OF PATIENTS 6 MONTHS OF AGE AND OLDER WITH RELAPSED OR REFRACTORY PEDIATRIC LOW-GRADE GLIOMA (LGG) HARBORING A BRAF FUSION OR REARRANGEMENT, OR BRAF V600 MUTATION

Dosage:

FOR SUSPENSION;ORAL

Constraining patent/regulatory exclusivity:

TREATMENT OF PATIENTS 6 MONTHS OF AGE AND OLDER WITH RELAPSED OR REFRACTORY PEDIATRIC LOW-GRADE GLIOMA (LGG) HARBORING A BRAF FUSION OR REARRANGEMENT, OR BRAF V600 MUTATION

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.

Recent Clinical Trials for TOVORAFENIB

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Nationwide Children's Hospital	PHASE2
National Cancer Institute (NCI)	PHASE1
Daniel Morgenstern	EARLY_PHASE1

See all TOVORAFENIB clinical trials

Pharmacology for TOVORAFENIB

Drug Class	Kinase Inhibitor
Mechanism of Action	Cytochrome P450 3A Inducers Type II RAF Kinase Inhibitors

US Patents and Regulatory Information for TOVORAFENIB

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Day One Biopharms	OJEMDA	tovorafenib	FOR SUSPENSION;ORAL	218033-001	Apr 23, 2024		RX	Yes	Yes
Day One Biopharms	OJEMDA	tovorafenib	FOR SUSPENSION;ORAL	218033-001	Apr 23, 2024		RX	Yes	Yes
Day One Biopharms	OJEMDA	tovorafenib	TABLET;ORAL	217700-001	Apr 23, 2024		RX	Yes	Yes			Y	Y
Day One Biopharms	OJEMDA	tovorafenib	FOR SUSPENSION;ORAL	218033-001	Apr 23, 2024		RX	Yes	Yes			Y
Day One Biopharms	OJEMDA	tovorafenib	TABLET;ORAL	217700-001	Apr 23, 2024		RX	Yes	Yes
Day One Biopharms	OJEMDA	tovorafenib	TABLET;ORAL	217700-001	Apr 23, 2024		RX	Yes	Yes			Y

International Patents for TOVORAFENIB

Country	Patent Number	Title	Estimated Expiration
Japan	6571105
Peru	20170773
Taiwan	I444379
Israel	202835
Canada	2693182
Eurasian Patent Organization	031190

Market Dynamics and Financial Trajectory for Tovorafenib

Last updated: July 28, 2025

Introduction

Tovorafenib emerges as a promising targeted therapy, primarily designed for oncology indications involving aberrations in the fibroblast growth factor receptor (FGFR) pathway. As a potent selective FGFR inhibitor, its development aligns with the burgeoning precision medicine sector, which aims to provide tailored treatments for specific genetic profiles. This analysis explores the current landscape, market forces, and financial prospects shaping Tovorafenib’s trajectory within the pharmaceutical sphere.

Therapeutic Background and Clinical Development Status

Tovorafenib, developed by Blueprint Medicines, is an orally administered, reversible, selective FGFR inhibitor. It is continuing clinical trials for various indications, notably those involving FGFR-driven tumors such as gliomas and certain pediatric cancers. Notably, Blueprint’s vafidemstat and futibatinib establish a strategic alliance with ongoing research and development efforts for FGFR-related malignancies. As of 2023, Tovorafenib remains in Phase II clinical trials, with preliminary data indicating durable responses and manageable safety profiles (Blueprint Medicines, 2022[1]).

Its targeted mechanism focuses on inhibiting aberrant FGFR signaling, which is implicated in multiple tumorigenic processes, including cell proliferation, angiogenesis, and resistance to therapy. The promising early data position Tovorafenib as a candidate to address unmet needs in specific oncological subpopulations resistant to existing therapies.

Market Landscape and Competitive Positioning

Global Oncology Market and FGFR Inhibitors

The global oncology drug market, valued at approximately $200 billion in 2022, exhibits robust growth driven by rising cancer incidence rates, technological advancements, and increasing approvals of targeted therapies (MarketWatch, 2023). FGFR inhibitors constitute a significant segment within this space, with several candidates commercially available and others in development.

Key competitors include:

• Futibatinib (TAK-788): Approved for intrahepatic cholangiocarcinoma, exhibits a broader kinase inhibition profile.
• Pemigatinib (Infigratinib): Approved for cholangiocarcinoma with FGFR2 fusions.
• Erdafitinib (Janssen): Approved for bladder cancer with FGFR alterations.

While these drugs target different tumor types or stages, they collectively influence market expectations for FGFR inhibitors. Tovorafenib's differentiators hinge upon its selectivity, safety profile, and potential efficacy in pediatric populations and rare tumor subtypes, areas where current competitors are less prominent or lacking.

Market Entry and Adoption Prospects

Upon successful demonstration of clinical efficacy and safety, Tovorafenib is poised to capture a niche, especially for FGFR-driven pediatric tumors and rare adult cancers. Its oral administration aligns with current patient preferences for outpatient, minimally invasive therapies. Payers are increasingly favoring targeted therapies with documented benefits, which could facilitate reimbursement pathways if clinical benefits are confirmed.

Furthermore, regulatory pathways such as the FDA's Breakthrough Therapy designation could accelerate approval processes, expediting market entry. Strategic partnerships with healthcare providers, patient advocacy groups, and payers will be critical to establish market presence.

Market Dynamics Influencing Tovorafenib's Financial Trajectory

Regulatory and Accessibility Factors

Regulatory endorsement hinges on positive Phase II data, emphasizing safety and preliminary efficacy. The potential for expedited review processes, supported by the drug’s targeted mechanism and unmet medical need, can bolster investor confidence. Additionally, orphan drug designation for specific pediatric indications could provide benefits like market exclusivity and tax incentives, strengthening financial prospects.

Market access will rely on demonstrating value through clinical outcomes and cost-effectiveness. As health systems globally increasingly favor precision medicine, Tovorafenib’s targeted approach aligns with reimbursement trends favoring high-value therapies.

Market Penetration and Commercial Strategies

Effective commercialization depends on establishing clinical guidelines, clinician awareness, and integrating the drug into treatment algorithms. Neurological, pediatric, and rare tumor markets represent initial focus areas. Collaborations with leading oncology centers and clinical researchers will facilitate adoption, while health economic studies will influence payer decisions.

Intellectual Property and Patent Landscape

Patents extending into the late 2030s will provide market exclusivity, deterring generic competition during this window. Patent strength depends on formulation, method of use, and manufacturing processes, which Blueprint Medicines actively defend.

Pricing and Reimbursement Outlook

Premium pricing is anticipated given the targeted therapy context, with potential costs in the range of other FGFR inhibitors (approximately $10,000 to $15,000 per month). Reimbursement will depend on demonstrating superior or comparable efficacy profiles, manageable safety, and quality-of-life improvements.

Commercial Challenges

Potential hurdles include competition from established FGFR inhibitors, development setbacks, and unforeseen safety issues. Additionally, the rarity of indications may limit commercialization scale but allows for premium pricing and favorable reimbursement terms.

Financial Outlook and Investment Considerations

Based on early clinical data and unmet need, Tovorafenib’s market potential is promising. If regulatory approval is secured expeditiously, sales could reach hundreds of millions annually within 5-7 years post-launch, particularly targeting niche pediatric and adult oncologies.

However, risks include clinical setbacks, approval delays, and competitive dynamics. Investment in companion diagnostics development may enhance market targeting, further improving financial prospects.

Key Market Drivers

• Growing prevalence of FGFR-driven cancers and rare tumors.
• Advancements in personalized oncology.
• Regulatory incentives for orphan and pediatric indications.
• Commercial flexibility with oral administration.
• Potential for combination therapies increasing efficacy.

Conclusion

Tovorafenib is positioned to capitalize on the expanding landscape of targeted cancer therapies, driven by precision medicine, unmet clinical needs, and favorable regulatory trends. Strategic execution—through clinical success, regulatory approval, and effective commercialization—will determine its financial trajectory. Early-stage trials showing promising efficacy, particularly in pediatric tumors, offer a compelling narrative for stakeholders seeking innovative oncology solutions.

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Key Takeaways

• Tovorafenib’s development aligns with the growing demand for FGFR-targeted therapies in oncology, particularly in rare and pediatric tumors.
• Market entry depends heavily on clinical trial success, regulatory acceleration prospects, and strategic commercialization.
• Competitive advantages include selectivity, oral administration, and potential orphan drug status, enhancing pricing power.
• The global FGFR inhibitor market remains competitive but offers opportunities for niche positioning based on unique indications.
• Long-term success hinges on demonstrating superior clinical outcomes, securing reimbursement pathways, and navigating competitive challenges.

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FAQs

1. What specific indications is Tovorafenib targeting?
Tovorafenib primarily targets FGFR-driven tumors, including gliomas, pediatric cancers, and certain adult solid tumors. It is in clinical trials for specific indications such as pediatric low-grade gliomas and other rare malignancies (Blueprint Medicines, 2022[1]).

2. How does Tovorafenib differentiate from existing FGFR inhibitors?
Its high selectivity, favorable safety profile, and potential application in pediatric populations distinguish Tovorafenib. Its oral administration also enhances convenience compared to some injectable competitors.

3. What are the main regulatory hurdles for Tovorafenib’s commercial launch?
Key hurdles include demonstrating robust efficacy and safety in clinical trials, securing regulatory designations like Breakthrough Therapy or orphan status, and satisfying quality and manufacturing standards to gain approval.

4. How significant is the market opportunity for Tovorafenib in the coming years?
The opportunity is moderate to substantial within niche markets—particularly pediatric and rare FGFR-driven cancers—potentially generating hundreds of millions in annual revenue if approval and commercialization proceed successfully.

5. What factors could influence the financial success of Tovorafenib?
Factors include clinical trial outcomes, regulatory decisions, competitive landscape, pricing strategies, reimbursement policies, and the ability to establish strong clinical collaborations and market access pathways.

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References

[1] Blueprint Medicines. (2022). Tovorafenib Clinical Trial Data and Development Updates.