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Last Updated: January 20, 2025

CLINICAL TRIALS PROFILE FOR NITISINONE


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All Clinical Trials for Nitisinone

Trial IDTitleStatusSponsorPhaseStart DateSummary
NCT01734889 ↗ Taste and Palatability of Orfadin Suspension Completed Swedish Orphan Biovitrum Phase 1 2012-10-01 The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
NCT01682538 ↗ Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension Completed Swedish Orphan Biovitrum Phase 1 2012-08-01 The study is primarily being performed in order to demonstrate bioequivalence between the Orfadin (nitisinone) suspension and the marketed capsule formulation. The study will also contain a comparison of the bioavailability of the suspension given with food and on an empty stomach.
NCT01390077 ↗ Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria Completed University of California, San Diego Phase 2/Phase 3 2011-01-01 Nitisinone is a potent inhibitor of the enzyme that catalyzes the formation of homogentisic acid, and should be an even more logical treatment for alkaptonuria than for tyrosinemia, for which it has been approved by the FDA.The objective of this research is to explore reported age related differences in toxicity of nitisinone and its pharmacokinetic underpinnings and to develop an optimal therapeutic requirement for a targeted population of presymptomatic patients. The additional effect of mixtures of amino acids excluding tyrosine will be explored to take advantage of protein synthesis to avoid elevations of tyrosine that would otherwise limit the optimal dosage of nitisinone. The study is designed to treat patients and find the optimal dosage of nitisinone to obtain maximal reduction in levels of homogentisic acid and maintain safe levels of tyrosine. The long term objective in the target population of pre-symptomatic patients is the prevention of the characteristic effects on joint cartilage and tendons.
NCT00107783 ↗ Long-Term Study of Nitisinone to Treat Alkaptonuria Completed National Human Genome Research Institute (NHGRI) Phase 2 2005-01-01 This 3-year study will examine the safety and effectiveness of long-term use of nitisinone (Orfadin) for treating joint problems in patients with alkaptonuria, an inherited disease in which a compound called homogentisic acid accumulates. The excess homogentisic acid causes arthritis and limited joint movement. It can also cause heart valve damage and kidney stones. Patients between 30 and 80 years of age with alkaptonuria may be eligible for this study. Patients must have hip involvement, but at least one remaining hip joint. Candidates are recruited from among patients enrolled in protocol 00-HG-0141, "Clinical, Biochemical, and Molecular Investigations into Alkaptonuria." Participants may enter both protocols simultaneously. Participants are randomly assigned to one of two treatment groups: one group takes their regular medicines plus a 2-mg nitisinone capsule daily; the other group takes only their regular medicines. Patients taking nitisinone have blood tests to measure liver function 2 weeks and 6 weeks after starting treatment. Before starting therapy, all patients are admitted to the NIH Clinical Center for 4-5 days to undergo the following procedures: - Medical history and physical examination - 24-hour urine collection to test for sugar, protein, and other molecules - Blood tests for liver and thyroid function, blood counts, and blood chemistries - Blood and urine tests to measure tyrosine and other amino acids and homogentisic acid - Bone x-rays - Spiral CT (computed tomography) of the abdomen to detect kidney stones - Eye examination and evaluations by specialists in rehabilitation medicine and pain, plus other consults in skin, brain, lung, heart, and kidney, as needed All patients, whether or not they receive nitisinone, return to the Clinical Center for a 2-3 day follow-up admission every 4 months for a history and physical examination, blood tests, and two 24-hour urine collections. Every 12 months (12, 24 and 36 months after starting the study), patients also have repeat bone x-rays, spiral CT, kidney ultrasound, echocardiogram, and electrocardiogram. An Magnetic Resonance Imaging (MRI) of the brain is done at the end of the study. Sixteen months after the end of the study enrollment period, the treated and non-treated groups are evaluated. If nitisinone has delayed the progression of joint disease in the treated group, the study continues and all patients receive the drug for the remainder of the study. If not, the study continues for another 20 months, at which time the study ends and the evaluation process is repeated. Patients who develop symptoms such as corneal crystals, pain, or severe liver or nervous system toxicity may be taken off the study.
NCT00031161 ↗ Prevention of Dichloroacetate Toxicity Completed University of Florida N/A 2001-09-01 This is a study to determine the safety of dichloroacetate (DCA) with a low-tyrosine diet given with or without nitisinone (NTBC) in children with chronic lactic acidosis (CLA).
>Trial ID>Title>Status>Phase>Start Date>Summary
Showing 1 to 5 of 5 entries

Clinical Trial Conditions for Nitisinone

Condition Name

7411001234567Hereditary Tyrosinemia, Type IAlkaptonuriaAcidosis, LacticAlbinism[disabled in preview]
Condition Name for Nitisinone
Intervention Trials
Hereditary Tyrosinemia, Type I 7
Alkaptonuria 4
Acidosis, Lactic 1
Albinism 1
[disabled in preview] 0
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Condition MeSH

7441001234567TyrosinemiasOchronosisAlkaptonuriaAcidosis, Lactic[disabled in preview]
Condition MeSH for Nitisinone
Intervention Trials
Tyrosinemias 7
Ochronosis 4
Alkaptonuria 4
Acidosis, Lactic 1
[disabled in preview] 0
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Clinical Trial Locations for Nitisinone

Trials by Country

+
Trials by Country for Nitisinone
Location Trials
United Kingdom 4
United States 4
Germany 4
Netherlands 3
South Africa 3
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Trials by US State

+
Trials by US State for Nitisinone
Location Trials
Maryland 2
California 1
Florida 1
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Clinical Trial Progress for Nitisinone

Clinical Trial Phase

15.4%7.7%15.4%61.5%012345678Phase 3Phase 2/Phase 3Phase 2[disabled in preview]
Clinical Trial Phase for Nitisinone
Clinical Trial Phase Trials
Phase 3 2
Phase 2/Phase 3 1
Phase 2 2
[disabled in preview] 8
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Clinical Trial Status

87.5%12.5%002468101214CompletedUnknown status[disabled in preview]
Clinical Trial Status for Nitisinone
Clinical Trial Phase Trials
Completed 14
Unknown status 2
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Clinical Trial Sponsors for Nitisinone

Sponsor Name

trials01122334455667Swedish Orphan BiovitrumParexelCycle Pharmaceuticals Ltd.[disabled in preview]
Sponsor Name for Nitisinone
Sponsor Trials
Swedish Orphan Biovitrum 6
Parexel 4
Cycle Pharmaceuticals Ltd. 3
[disabled in preview] 4
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Sponsor Type

60.9%26.1%13.0%002468101214IndustryOtherNIH[disabled in preview]
Sponsor Type for Nitisinone
Sponsor Trials
Industry 14
Other 6
NIH 3
[disabled in preview] 0
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Nitisinone: Clinical Trials, Market Analysis, and Projections

Introduction

Nitisinone, also known as NTBC (2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione), is a drug that has garnered significant attention for its efficacy in treating rare genetic disorders, particularly hereditary tyrosinemia type 1 (HT-1) and alkaptonuria (AKU). This article delves into the recent clinical trials, market analysis, and future projections for nitisinone.

Clinical Trials and Efficacy

Hereditary Tyrosinemia Type 1 (HT-1)

Clinical trials for nitisinone in HT-1 have been highly successful. The multinational NTBC Study, conducted by 96 investigators across 87 hospitals in 25 countries, demonstrated the drug's efficacy in significantly improving survival rates and reducing liver and kidney toxicity associated with HT-1. The study showed that patients treated with nitisinone had a 1-year and 2-year survival rate of 88% and 95%, respectively, when initiated before 2 months of age, compared to much lower survival rates in historical control groups treated only with dietary restrictions[3].

Alkaptonuria (AKU)

For AKU, the SONIA 1 and SONIA 2 studies have provided robust evidence of nitisinone's effectiveness. These international, multicentre, open-label trials showed a significant decrease in homogentisic acid levels, which is the primary cause of AKU, and a reversal of the disease process known as ochronosis. The European Commission approved the extended indication for Orfadin (nitisinone) to treat adult patients with AKU based on these positive results[1][4].

Market Analysis

Global Market Size and Growth

The global nitisinone market is projected to experience substantial growth. As of 2024, the market size is estimated to be USD 101.5 million, with a compound annual growth rate (CAGR) of 10.60% from 2024 to 2031, reaching USD 205.47 million by 2031[2].

Regional Market Dynamics

  • North America: Currently dominates the nitisinone market due to its high prevalence of rare genetic disorders like HT-1, advanced healthcare infrastructure, and strong awareness of rare diseases. Comprehensive diagnostic programs and early detection initiatives, supported by robust healthcare policies and orphan drug incentives, contribute to this region's significant growth[2].
  • Asia Pacific: Growing at the fastest CAGR, driven by improving healthcare infrastructure, increased awareness of rare diseases, and expanding access to diagnostic and treatment services. Countries like China, India, and Japan are key drivers of this growth[2].
  • Latin America and Middle East & Africa: These regions hold smaller market shares but are expected to grow at CAGRs of 10.0% and 10.3%, respectively, from 2024 to 2031[2].

Key Drivers and Restraints

  • Key Drivers: Rising prevalence of rare genetic disorders, increased awareness and early diagnosis, supportive government incentives for orphan drugs, and technological advancements in diagnostic tools.
  • Restraints: High cost of nitisinone and regulatory challenges[2].

Market Projections

Forecasted Growth

The nitisinone market is expected to expand significantly over the next few years. By 2031, the market is projected to reach USD 205.47 million, driven by the increasing prevalence of HT-1 and AKU, as well as advancements in medical science and healthcare accessibility[2].

Emerging Trends

  • Personalized Medicine: There is a growing emphasis on personalized medicine, which is expected to drive the adoption of nitisinone.
  • Expanding Healthcare Infrastructure: Improving healthcare infrastructure in emerging markets will increase access to nitisinone.
  • Research and Development: Increased investment in research and development for rare diseases will further boost the market[2].

Applications and Market Share

Hereditary Tyrosinemia Type 1 (HT-1)

HT-1 remains a significant application for nitisinone, with the drug being crucial for managing this life-threatening condition. The Ornithine Transcarbamylase (OTC) deficiency, closely related to HT-1, also captures a large market share due to the high prevalence and critical need for effective treatment[2].

Alkaptonuria (AKU)

The approval for treating AKU has opened up new market opportunities for nitisinone. The SONIA 1 and SONIA 2 studies have demonstrated the drug's efficacy in this area, contributing to its growing market share[1][4].

Regulatory Approvals and Compliance

European Commission Approval

The European Commission approved the extended indication for Orfadin (nitisinone) to treat adult patients with AKU in October 2020, based on the positive results from clinical trials[1].

Health Canada Approval

Health Canada granted a Notice of Compliance to MendeliKABS Inc. for MDK-Nitisinone, a bioequivalent formulation of Orfadin, for the treatment of HT-1 patients. This approval was based on the demonstration of bioequivalence and the bridging of evidence from the Orfadin formulation[3].

Safety and Clinical Pharmacology

Mechanism of Action

Nitisinone acts as a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase, preventing the accumulation of toxic metabolites in patients with HT-1. This mechanism is crucial for reducing liver and kidney toxicity associated with the disease[3].

Safety Profile

While nitisinone has shown significant clinical benefits, it also comes with identified safety issues that can be managed through labelling and adequate monitoring. Appropriate warnings and precautions are in place to address these concerns[3].

Conclusion

Nitisinone has emerged as a critical drug in the treatment of rare genetic disorders such as HT-1 and AKU. The positive outcomes from clinical trials, coupled with regulatory approvals and a growing market, indicate a bright future for this medication.

Key Takeaways

  • Clinical Efficacy: Nitisinone has shown significant efficacy in treating HT-1 and AKU, improving survival rates and reducing disease-related complications.
  • Market Growth: The global nitisinone market is projected to grow at a CAGR of 10.60% from 2024 to 2031.
  • Regional Dynamics: North America dominates the market, while the Asia Pacific region is growing at the fastest CAGR.
  • Key Drivers: Rising prevalence of rare genetic disorders, increased awareness, and supportive government incentives.
  • Regulatory Approvals: Approved by the European Commission for AKU and by Health Canada for HT-1.

FAQs

What is nitisinone used for?

Nitisinone is used to treat rare genetic disorders such as hereditary tyrosinemia type 1 (HT-1) and alkaptonuria (AKU).

What are the key findings from the clinical trials of nitisinone?

Clinical trials have shown that nitisinone significantly improves survival rates and reduces liver and kidney toxicity in HT-1 patients, and decreases homogentisic acid levels and reverses ochronosis in AKU patients.

What is the projected market size of nitisinone by 2031?

The global nitisinone market is projected to reach USD 205.47 million by 2031, growing at a CAGR of 10.60% from 2024 to 2031.

Which regions are driving the growth of the nitisinone market?

North America currently dominates the market, while the Asia Pacific region is growing at the fastest CAGR due to improving healthcare infrastructure and increasing awareness of rare diseases.

What are the main safety concerns associated with nitisinone?

While nitisinone is effective, it comes with identified safety issues that can be managed through labelling and adequate monitoring. Appropriate warnings and precautions are in place to address these concerns.

Sources

  1. European Commission: "Success with the approval of Orfadin to treat patients with AKU"[1].
  2. Cognitive Market Research: "Global Nitisinone Market Report 2024"[2].
  3. Health Canada: "Summary Basis of Decision for MDK-Nitisinone"[3].
  4. MDPI: "Effects of Nitisinone on Oxidative and Inflammatory Markers in Alkaptonuria: Results from SONIA1 and SONIA2 Studies"[4].
  5. Valuates Reports: "Global and United States Nitisinone Market Research Report 2024"[5].

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