Congenital Adrenal Hyperplasia (CAH) - Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026

Congenital Adrenal Hyperplasia (CAH) Emerging Therapy and TPP Insights

Thelansis's "Congenital Adrenal Hyperplasia (CAH) Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

Congenital Adrenal Hyperplasia (CAH) Overview

Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive adrenal steroidogenesis disorders, with 21-hydroxylase deficiency caused by CYP21A2 mutations accounting for over 90% of cases, disrupting cortisol and aldosterone biosynthesis while redirecting steroid precursors toward androgen excess. Disease severity spans classic salt-wasting CAH presenting neonatally with adrenal crisis, hyponatremia, hyperkalemia, and virilization, through simple virilizing and non-classic forms manifesting with premature adrenarche, hirsutism, and subfertility. Newborn screening via 17-hydroxyprogesterone measurement enables early diagnosis, confirmed by genotyping and adrenal steroid profiling. Glucocorticoid replacement suppresses ACTH-driven androgen excess while restoring deficient cortisol; fludrocortisone addresses salt-wasting. Balancing glucocorticoid dosing remains clinically challenging, as under-replacement perpetuates androgen excess while over-replacement risks iatrogenic Cushing syndrome and growth suppression. The medical paradigm shifted with the FDA approval of Crenessity (crinecerfont), an oral CRF1 receptor antagonist indicated for classic CAH patients aged four and older. Crinecerfont directly reduces excessive ACTH and downstream adrenal androgens through a non-glucocorticoid mechanism, successfully enabling major reductions in daily steroid maintenance doses. Conversely, development of alternative CRF1 antagonists like tildacerfont has been entirely discontinued due to poor clinical efficacy. Preventing adrenal crisis through sick-day rules remains critical to optimizing lifelong patient outcomes.

Key Highlights

• In France, prevalent CAH cases are projected to increase from approximately 74,390 in 2025 to 76,704 by 2035.
• CAH remains a rare endocrine disorder characterized by impaired cortisol synthesis and hormonal imbalance.
• Lifelong disease management is required to prevent adrenal crises and optimize growth and development outcomes.
• Increasing newborn screening programs are supporting earlier diagnosis and intervention.
• Novel therapeutic approaches aim to improve hormonal control while reducing steroid-related complications.

Market Overview

• The Italy CAH market is projected to grow from approximately $60 MN to $170 MN by 2035.
• Market growth is supported by:
• Increasing utilization of improved hormone replacement therapies
• Growing awareness and earlier disease diagnosis
• Market expansion is driven by long-term treatment requirements and emerging targeted therapies.
• Future growth will depend on next-generation endocrine therapies and disease-modifying approaches.

Insights driven by surveys with physician / key opinion leaders:

• Survey findings are corroborated and enriched by insights from interviews with leading KOLs
• Survey is customized based on client requirements

Deliverables format:

• PowerPoint presentation
• MS Excel

Key business questions answered:

• Detailed emerging competitive landscape
• Pipeline analysis
• Target patients for emerging therapies
• Key companies
• Key mechanism of actions
• Launch date estimates, etc.
• Clinical trial landscape analysis
• Target patient segments
• Trial endpoints
• Trial design
• Recruitment criteria, etc.
• Unmet Needs and Opportunities
• Performance of key current therapies
• Top areas of unmet needs
• Opportunity sizing for key unmet needs
• Target Product Profiles
• Attributes and levels
• Physician likelihood of prescribing
• Expected patient shares
• KOL insights on key emerging therapies
• Level of awareness
• Expected use / line of therapy
• Extent to fulfil key unmet needs
• KOL quotes

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• Crinetics Pharmaceuticals Inc.
• Neurocrine Biosciences
• Spruce Biosciences
• H. Lundbeck A/S
• Neurocrine UK Limited
• Adrenas Therapeutics Inc.