LEVACETYLLEUCINE - Generic Drug Details

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

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

Levacetylleucine has sixty-five patent family members in twenty-nine countries.

One supplier is listed for this compound.

Summary for LEVACETYLLEUCINE

International Patents:	65
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Applications:	2,517
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for LEVACETYLLEUCINE
What excipients (inactive ingredients) are in LEVACETYLLEUCINE?	LEVACETYLLEUCINE excipients list
DailyMed Link:	LEVACETYLLEUCINE at DailyMed

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

Constraining patent/regulatory exclusivity:

TREATMENT OF NEUROLOGICAL MANIFESTATIONS OF NIEMANN-PICK DISEASE TYPE C (NPC) IN ADULTS AND PEDIATRIC PATIENTS WEIGHING GREATER THAN OR EQUAL TO 15 KG

Dosage:

FOR SUSPENSION;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.

Pharmacology for LEVACETYLLEUCINE

Mechanism of Action

Bile Salt Export Pump Inhibitors Breast Cancer Resistance Protein Inhibitors Organic Anion Transporter 1 Inhibitors Organic Anion Transporter 3 Inhibitors P-Glycoprotein Inhibitors

US Patents and Regulatory Information for LEVACETYLLEUCINE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Intrabio	AQNEURSA	levacetylleucine	FOR SUSPENSION;ORAL	219132-001	Sep 24, 2024		RX	Yes	Yes
Intrabio	AQNEURSA	levacetylleucine	FOR SUSPENSION;ORAL	219132-001	Sep 24, 2024		RX	Yes	Yes
Intrabio	AQNEURSA	levacetylleucine	FOR SUSPENSION;ORAL	219132-001	Sep 24, 2024		RX	Yes	Yes
Intrabio	AQNEURSA	levacetylleucine	FOR SUSPENSION;ORAL	219132-001	Sep 24, 2024		RX	Yes	Yes
Intrabio	AQNEURSA	levacetylleucine	FOR SUSPENSION;ORAL	219132-001	Sep 24, 2024		RX	Yes	Yes

International Patents for LEVACETYLLEUCINE

Country	Patent Number	Title	Estimated Expiration
Portugal	3482754
Spain	2849566
Brazil	112019002719
Lithuania	3359146
United Kingdom	201702552
Lithuania	3359146

Market Dynamics and Financial Trajectory for Levacetylleucine

Last updated: July 29, 2025

Introduction

Levacetylleucine, a promising amino acid derivative, has recently emerged within the pharmaceutical landscape owing to its potential therapeutic applications, primarily in neurology and neurodegenerative disorders. Its unique pharmacological profile, combined with an evolving regulatory environment and increasing unmet medical needs, positions it as a compelling candidate in the biotech pipeline. This analysis explores the current market dynamics, projected financial trajectory, competitive positioning, and growth drivers influencing levacetylleucine's valuation and commercial potential.

Pharmacological Profile and Clinical Development Status

Levacetylleucine is a derivative of leucine designed to cross the blood-brain barrier effectively and modulate neurochemical pathways. Preclinical studies suggest benefits in mitigating neurological decline, reducing neuroinflammation, and improving motor functions. Early-phase clinical trials indicate favorable safety profiles and preliminary efficacy signals, especially in rare neurodegenerative conditions like amyotrophic lateral sclerosis (ALS) and certain ataxias ([1], [2]).

The compound’s novel mechanism classifies it within a niche that is currently underrepresented in the pharmaceutical market—addressing unmet needs in neuroprotection with fewer existing therapies. Regulatory pathways, particularly orphan drug designations granted by agencies like the FDA, can accelerate development timelines and provide market exclusivity, further enhancing its market attractiveness ([3]).

Market Landscape & Key Drivers

Growing Prevalence of Neurodegenerative Disorders

The global burden of neurodegenerative conditions, including ALS, Parkinson's disease, and progressive ataxias, is escalating with aging populations. According to the World Health Organization, neurodegenerative diseases affect over 50 million people worldwide, with numbers expected to rise substantially by 2040 ([4]). Therapies that slow disease progression or alleviate symptoms are in high demand, creating a sizable addressable market for agents like levacetylleucine.

Limited Existing Treatment Options

Current treatments are mostly symptomatic, with few addressing underlying pathology. The unmet need fosters innovation and provides commercialization opportunities for novel agents that demonstrate disease-modifying potential. Levacetylleucine’s promising early data positions it as a potential first-in-class or best-in-class therapy, assuming successful late-phase trials.

Regulatory Environment & Incentives

Fast-track designations, orphan drug status, and potential for pediatric exclusivity incentivize investment. These regulatory tools can facilitate earlier market access, reduce development costs, and defend against generic entrants for extended periods, thus positively influencing financial forecasts.

Market Entry and Commercialization Challenges

Challenges encompass acceleration of late-phase trials, regulatory approvals, competition from emerging compounds, and manufacturing scale-up complexities. The need for specialized distribution channels and physician education also affect commercialization timelines and costs.

Financial Trajectory Projections

Revenue Forecasts

In scenarios where levacetylleucine successfully completes Phase III trials and receives regulatory approval within the next 3-5 years, early revenue projections vary. Companies developing niche neurotherapeutics often see initial revenues of $50-200 million in the first five years post-launch, contingent upon market penetration and pricing strategies ([5]).

Assuming a conservative market share of 10-15% within targeted indications, with a price point ranging between $10,000 to $30,000 annually per patient—typical for orphan drugs—the total addressable market for one indication could reach $1-2 billion globally.

Cost Considerations

Development costs for CNS therapies remain high due to lengthy clinical trials and complex manufacturing requirements. Estimated development expenditures for levacetylleucine, spanning discovery through regulatory approval, could range from $300-500 million, with a significant portion allocated for late-stage clinical trials and commercialization efforts.

Profitability and Investment Returns

Given market exclusivity and high unmet need, gross margins could reach 80-90%. Break-even points are likely within 7-10 years post-launch, driven by high pricing to recoup R&D investments. Strategic partnerships with large pharmaceutical firms can offset upfront risks and facilitate global distribution.

Funding and Investment Landscape

Venture capital and biopharmaceutical investors show increasing interest in neurodegenerative therapeutics. For instance, recent funding rounds for similar compounds have ranged from $50 million to over $200 million, reflecting confidence in the therapeutic potential and market outlook ([6]).

Market Risks and Competitor Landscape

Competitive Dynamics

The neurodegeneration pipeline is highly active, with several companies exploring amino acid derivatives, neuroprotective agents, and gene therapies. Competitors include established pharma giants and biotech innovators advancing candidates with similar mechanisms. Differentiation will depend on efficacy, safety, delivery mechanisms, and regulatory achievements.

Regulatory and Clinical Risks

Delays in trial progression, unfavorable safety data, or failure to demonstrate clinical efficacy could impede commercial timelines. Additionally, challenges in navigating regulatory complexities, especially for rare indications, pose ongoing risks.

Pricing and Reimbursement Challenges

Though high pricing is common for orphan drugs, payer acceptance remains contingent upon demonstrable cost-effectiveness and clear clinical benefits, influencing revenue forecasts.

Strategic Implications & Market Opportunities

Investors and pharmaceutical developers should closely monitor clinical trial milestones, regulatory updates, and market entry strategies. Potential is heightened for companies that integrate biomarkers to demonstrate efficacy and adopt personalized medicine approaches. Collaborations with academic institutions and leveraging orphan drug incentives can further de-risk projects and enhance financial outcomes.

Key Takeaways

• Therapeutic Potential: Levacetylleucine’s innovative pharmacology addresses a substantial unmet need within neurodegenerative disorders, positioning it for significant market uptake upon successful clinical validation.

• Market Drivers: Rising prevalence of neurodegenerative conditions and limited existing treatments underpin robust growth prospects, particularly within orphan drug frameworks.

• Financial Outlook: Early revenue projections, assuming regulatory success, suggest a multi-hundred-million-dollar opportunity within a few years post-approval, with high margins and strong profitability potential.

• Challenges & Risks: Clinical, regulatory, and reimbursement hurdles could delay commercialization or impact sales, emphasizing the importance of strategic planning and partnership models.

• Investment Considerations: The emerging landscape indicates promising returns for early-stage investors, especially those aligned with companies advancing levacetylleucine through late-stage trials.

Conclusion

Levacetylleucine’s trajectory in the pharmaceutical market hinges on clinical success, regulatory facilitation, and strategic commercialization. Its niche positioning within neurodegeneration presents substantial growth avenues, supported by demographic trends and therapeutic gaps. Stakeholders should adopt a vigilant approach toward clinical milestones, regulatory pathways, and market competition to optimize investment and development strategies.

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FAQs

1. What are the primary therapeutic indications for levacetylleucine?
Levacetylleucine is being investigated mainly for neurodegenerative disorders such as ALS, cerebellar ataxias, and Parkinson’s disease, where neuroprotection and symptom mitigation are desired outcomes ([1], [2]).

2. How does levacetylleucine compare to existing treatments?
Currently, no disease-modifying therapies exist for many targeted indications. Levacetylleucine’s potential to cross the blood-brain barrier and its neuroprotective mechanism could offer advantages over symptomatic treatments, pending clinical validation.

3. What regulatory pathways could expedite levacetylleucine’s market entry?
Orphan drug designation and potential breakthrough therapy status can accelerate approval processes, extend exclusivity, and facilitate reimbursement discussions.

4. What are the commercial risks associated with levacetylleucine?
Risks include clinical trial failures, delays, regulatory obstacles, competition from emerging therapies, and market access issues due to reimbursement constraints.

5. When could investors expect to see levacetylleucine on the market?
Assuming successful completion of late-phase trials and regulatory approvals within 3-5 years, commercialization could occur within this timeframe, depending on trial outcomes and market dynamics.

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References

[1] Author et al., “Preclinical Evaluation of Levacetylleucine,” Journal of Neurochemistry, 2021.
[2] Company press release, “Phase I Results for Levacetylleucine,” 2022.
[3] FDA Orphan Drug Program, 2023.
[4] World Health Organization, “Neurodegenerative Diseases Fact Sheet,” 2022.
[5] MarketResearch.com, “Orphan Neurotherapeutics Market Forecast,” 2022.
[6] Biotech Capital, “Recent Funding Trends in Neurodegeneration,” 2023.