Homocystinuria (HCU) – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030


 Homocystinuria, caused by cystathionine β-synthase deficiency, is a rare inherited disorder involving methionine metabolism. Impaired synthesis of cystathionine leads to accumulation of homocysteine that affects several organ systems leading to abnormalities in the skeletal, cardiovascular, ophthalmic, and central nervous systems. The primary defect is a marked reduction in the activity of the critical enzyme for transsulfuration, cystathionine β-synthase (CBS). It is biochemically characterized by elevated methionine and total homocysteine and contractions in cystathionine, the immediate product of CBS, and cysteine. Other defects in the methionine metabolic cycle that result in homocystinuria are remethylation defects in which the conversion of homocysteine back to methionine is impaired. These defects produce reduced rather than increased methionine and increased homocysteine and may also be associated with an increase in methylmalonic acid. With one exception, methylenetetrahydrofolate reductase deficiency, they are collectively known as the cobalamin defects because defects produce them in vitamin B12 metabolism.

Homocystinuria is a rare disorder with a minimum prevalence of 1 to 1.5 cases per 100,000 population.

The competitive landscape of Homocystinuria (HCU) includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary.

KOLs insights of Homocystinuria (HCU) across 8 MM market 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.

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

Read more: Homocystinuria (HCU) – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          Pegtibatinase    Travere Therapeutics, Inc.         Phase 2

2          ACN00177        Aeglea Biotherapeutics  Phase 1/2

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