Breakthrough Clinical Results
Shanton Pharma announced that the FDA granted Fast Track designation to its investigational new drug, SAP-001, for treating hyperuricemia in adult gout patients refractory to conventional therapy. SAP-001 is a novel, once-daily oral urate-lowering therapy with a unique mechanism of action. The Fast Track designation is based on positive Phase 2b clinical trial results demonstrating SAP-001's potential to significantly improve upon existing urate-lowering therapies. This designation will expedite the regulatory review process, aiming to bring this much-needed treatment to patients sooner.
Key Highlights
- FDA granted Fast Track designation to SAP-001 for refractory gout.
- SAP-001 demonstrated potential for meaningful improvement over existing therapies in Phase 2b trials.
- Fast Track designation will expedite regulatory review and potential approval.
- SAP-001 is a novel, once-daily oral urate-lowering therapy with a First-in-Class mechanism of action.
Incidence and Prevalence
Global Prevalence and Incidence of Gout
According to recent epidemiological data, gout remains one of the oldest known diseases and is currently the most common form of inflammatory arthritis worldwide. The global landscape of gout shows significant variations across different regions and populations.
A 2023 literature review revealed that gout is more prevalent in developed countries than in developing countries, although its prevalence is increasing globally. This trend indicates a shifting epidemiological pattern that affects populations worldwide.
Gender distribution analysis shows that gout is much more prevalent among males than among females, representing a consistent demographic pattern across studies. This gender disparity remains a significant characteristic of gout epidemiology.
Regional trends indicate a precipitating number of gout cases from the developed countries as reported in a 2021 review. However, the same review highlighted that developing countries are faced with an even higher threat of increasing gout prevalence, suggesting a concerning global trajectory.
In specific regional data, a 2024 Korean study found the weighted prevalence of gout was 2.1% (weighted n=808,778) in the Korean adult population. This provides a recent benchmark for East Asian prevalence rates.
Age-related patterns show that gout is a common problem in middle-aged males but has an increasing prevalence in older patients, particularly women. This age and gender shift represents an important epidemiological trend.
The overall disease burden of gout remains substantial and may be increasing according to data from 2006, a trend that appears to have continued in subsequent years. Recent research has also identified that gout is associated with several new comorbidities in recent years, complicating its clinical presentation and management.
A 2024 study noted that pediatric gout is a condition that differs from traditional adult gout and has attracted significant attention, suggesting changing patterns in age of onset.
The epidemiology of gout is influenced by multiple risk factors including hyperuricemia, chronic renal disease, genetic factors, alcohol consumption, dietary factors, diuretic use, hypertension, obesity, and metabolic syndrome. Among these, hyperuricemia has the largest role in gout development, although the other factors contribute significantly to its increasing prevalence.
Genetic studies, including a 2016 meta-analysis of ABCG2 Q141K (rs2231142), found that gender and ethnicity affect the association between gout susceptibility and genetic variants, suggesting that genetic factors vary by population.
Economic Burden
Economic Burden of Treating Gout in USA and Europe
United States Economic Burden
The economic burden of gout in the United States is substantial and appears to be increasing. The annual direct burden of illness for new cases of acute gout in the US is estimated at $27,378,494.
For US employees, the total annual cost for those with gout versus without gout was $6,870 versus $3,705, with significantly higher costs for medical claims, prescription claims, sick leave, short-term disability, and workers' compensation benefits. Costs were significantly higher across various service locations including office visits, outpatient hospital or clinic, inpatient hospital, and laboratory services for gout patients.
For older US adults with gout, the mean unadjusted per-patient gout-related healthcare cost was $876 (standard deviation $3,373) in 2005 dollars, representing 5.9% of the total all-cause healthcare cost of $14,734. After statistical adjustment for comorbidities, the difference in total 12-month all-cause healthcare costs between US gout patients and gout-free members was $3,038.
The incremental direct cost of gout has been suggested in the range of US$3,165 to US$5,515 (2004 and 2005 values, respectively), climbing to US$10,222 to US$21,467 (2008 values) per annum where patients are experiencing regular acute flares and have tophi present.
US gout patients with tophi had significantly higher costs - after adjustment for comorbidities, the difference in total 12-month all-cause healthcare costs between gout patients with and without tophi was $5,501. The difference in total adjusted 12-month gout-related costs between US patients with and without tophi was $1,710.
Patients with very high serum UA levels (≥9 mg/dL) had significantly higher regression-adjusted total 12-month all-cause healthcare costs and gout-related costs compared with those with low serum UA levels ($3,103 and $276 higher, respectively).
Notably, only 0.9% of employees with gout generated 20% of the total gout-specific medical and prescription costs, indicating a highly skewed distribution of economic burden.
Global and European Perspective
The Global Burden of Disease Study 2017 suggests a globally rising trajectory of gout burden between 1990 and 2017. The highest increase in gout burden was detected in high-SDI countries, especially in high-income North America.
In Spain (Basque Country), socio-economic deprivation was associated with higher costs across the majority of rheumatic and musculoskeletal conditions; however, patterns were more blurred, and an inverse relationship was observed for gout.
Published data to support healthcare decision making in non-US countries with regards to treatments for gout are currently limited, which is a key limitation of the current evidence base. More research is required to extend our understanding of the impact of gout on indirect costs, particularly in European healthcare systems.
The prevalence of gout appears to be on the rise worldwide, creating a heavy economic burden across healthcare systems.
HTA Approvals
Health Technology Assessment Approvals for Gout Treatments in the UK, France, and Germany
Based on a comprehensive review of available information, there is no specific data regarding which drugs have been approved by the Health Technology Assessment (HTA) agencies of the UK, France, and Germany for gout treatment in the last three years.
While several treatments for gout are mentioned in clinical literature, including:
- Colchicine: An anti-inflammatory drug used to treat gout, calcium pyrophosphate deposition disease, and Familial Mediterranean Fever
- Urate-lowering therapy (ULT): Discussed primarily in relation to renal protection
- Interleukin-1 inhibition: Noted as having previous success in gout treatment
- Symptomatic slow acting drugs for OA (including glucosamine and chondroitin)
- Anti-oxidant drugs (such as curcumin, ginger extracts, and copper)
There is no explicit information about which of these treatments, if any, have received formal approval from the HTA agencies in the United Kingdom, France, or Germany within the specified three-year timeframe.
It is worth noting that there has been some research interest in repurposing colchicine for potential cardiovascular disease benefits, but this is separate from its established use in gout management.
The current clinical literature appears to focus more on the efficacy and mechanisms of action of various gout treatments rather than their regulatory approval status in specific European countries. Without specific HTA approval information from these three countries over the past three years, a definitive list of approved gout medications cannot be provided.
For patients and healthcare providers seeking information about officially approved treatments for gout in these countries, consulting the official publications and databases of the respective national HTA bodies would be advisable: - NICE (National Institute for Health and Care Excellence) in the UK - HAS (Haute Autorité de Santé) in France - G-BA/IQWiG (Gemeinsamer Bundesausschuss/Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen) in Germany
Drug used in other indications
SAP-001 Clinical Trials Beyond Gout
After a thorough review of available information, there is no data available regarding additional indications for which SAP-001 is being trialed beyond gout. The clinical trial portfolio for this investigational compound appears to be either not publicly disclosed or not yet established for conditions other than gout.
Similarly, there is no information available about the intervention models being utilized in any potential trials of SAP-001 for non-gout indications. Without documented clinical trials for other conditions, the specific study designs, treatment protocols, and dosing regimens cannot be determined.
The pharmaceutical development pathway for SAP-001 may still be in early stages, with gout potentially serving as the primary indication before expansion to other conditions. Many investigational compounds initially focus on a single indication to establish safety profiles and efficacy data before broadening their clinical applications.
For patients and clinicians interested in SAP-001's potential applications beyond gout, it would be advisable to monitor clinical trial registries such as ClinicalTrials.gov for future updates, or to contact the developing pharmaceutical company directly for the most current information regarding their development pipeline and planned studies.
As with any investigational drug, the therapeutic potential across multiple indications typically becomes clearer as the compound progresses through various phases of clinical development and as more research data becomes available to the scientific and medical communities.