ABCC6 Deficiency – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

ABCC6 Deficiency Market Outlook

Thelansis’s “ABCC6 Deficiency Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" 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 ABCC6 Deficiency treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

ABCC6 Deficiency Overview

The ATP-binding cassette (ABC) transporters represent an extensive family of membrane efflux transporters comprising 48 distinct members. These transporters exhibit diverse substrate specificities, including lipids, peptides, polysaccharides, organic molecules, and ions. Phylogenetic analysis reveals that ABCC6 shares the highest sequence homology with ABCC1 and ABCC2, which are recognized as multidrug resistance transporters and are known to transport a variety of substrates relevant to clinically significant pharmaceutical agents. The endogenous substrate for ABCC6 remains unidentified. Mutations in ABCC6 are the genetic basis for the rare disorder pseudoxanthoma elasticum (PXE). PXE is an autosomal recessive condition characterized by ectopic mineralization of the skin, retina, and arteries, leading to skin papules, blindness, and arterial sclerosis. Histologically, PXE is defined by the calcification of elastic fibers. Infants with ABCC6 deficiency may present with Generalized Arterial Calcification (GACI) Type 2, which resembles GACI Type 1, the infantile form of ENPP1 deficiency. In older individuals, ABCC6 deficiency manifests as PXE, characterized by pathological mineralization in blood vessels and soft tissues, clinically impacting the skin, eyes, and vascular system.

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: 2023-2033

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.

Visit our social media pages: