Alagille Syndrome (ALGS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Alagille Syndrome (ALGS) Market Outlook

Thelansis’s “Alagille Syndrome (ALGS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Alagille Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Alagille Syndrome (ALGS) Overview

Alagille syndrome (ALGS), identified by OMIM 118450, is an autosomal dominant disorder linked to Notch signaling pathway abnormalities. It affects various systems, including the liver, heart, skeleton, eyes, kidneys, and central nervous system, often exhibiting distinct facial features. Most cases (about 97%) stem from JAG1 gene haploinsufficiency on 20p11.2-20p12 (encoding JAGGED1), caused primarily by mutations or deletions. A smaller fraction (<1%) arises from NOTCH2 mutations, which may lead to more frequent renal malformations. New mutations (approximately 60%) are common, and germline mosaicism rates are relatively high. Key clinical features of ALGS encompass bile duct paucity in liver biopsy, cholestasis, congenital heart defects (mainly pulmonary artery involvement), butterfly vertebrae, ophthalmic anomalies (most commonly posterior embryotoxon), and distinct facial characteristics. Additional manifestations may include renal irregularities, growth failure, developmental delays, splenomegaly, and vascular issues. For individuals with an ALGS diagnosis, crucial assessments, if not already conducted during the diagnostic process, include:

1. Evaluation by a pediatrician, physician, or gastroenterologist, incorporating liver function tests (with elevated GGT levels associated with ALGS-related conjugated hyperbilirubinemia), serum cholesterol and triglycerides, bile acid levels, clotting studies, liver ultrasound, scintiscan, and biopsy.
2. Comprehensive cardiac assessment.
3. AP spinal X-ray.
4. Ophthalmic evaluation.
5. Renal ultrasound and renal function tests.

FDA-approved medications for managing cholestatic pruritus associated with Alagille syndrome are Livmarli (maralixibat) and Bylvay (odevixibat). They inhibit the ileal bile acid transporter (IBAT) to reduce serum bile acid levels.

• Alagille syndrome incidence has been estimated to be 2 to 3 cases in every 100,000 population.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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