Drugs in ATC Class A05A

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Up to A - Alimentary tract and metabolism

Up to A05 - BILE AND LIVER THERAPY

Subclasses in ATC: A05A - BILE THERAPY

A05AA - Bile acids and derivatives

A05AX - Other drugs for bile therapy

A05A Market Analysis and Financial Projection

The ATC Class A05A (BILE THERAPY) encompasses drugs targeting bile-related disorders, including cholestatic liver diseases and pruritus. Market dynamics and patent activity reflect a surge in innovation, particularly for pediatric and genetic conditions. Below is an analysis of key trends and developments.

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Market Dynamics

Growth Drivers

• Rising Disease Burden: Cholestatic liver diseases (e.g., progressive familial intrahepatic cholestasis [PFIC], Alagille Syndrome) affect ~1 in 50,000–100,000 births globally[7][12]. Increasing diagnosis and awareness are propelling demand for targeted therapies.
• Pediatric Focus: Regulatory incentives like the EU’s Paediatric Regulation have accelerated approvals for pediatric bile therapies, with 10 new drugs in ATC categories A05/A11/A16 authorized between 2007–2016[5].
• Pipeline Innovation: Over 15 drugs are in clinical trials, including next-gen ileal bile acid transporter (ASBT) inhibitors like odevixibat and maralixibat[7][12]. The bile duct cancer treatment market alone is projected to grow at a 10% CAGR[9].

Key Therapies and Sales Trends

• Ursodeoxycholic Acid (UDCA): A cornerstone therapy for primary biliary cholangitis, generating $1.2B+ annual sales[6]. Its mechanism involves reducing hydrophobic bile acids and modulating farnesoid X receptor (FXR) activity[6].
• Odevixibat (Bylvay): First non-surgical PFIC treatment, approved in 2021 (FDA) and 2023 (Canada). Priced at ~$385,000 annually, it targets pruritus reduction with a 60% response rate in trials[7][15].
• Maralixibat: Approved for Alagille Syndrome, achieving 70% pruritus reduction in trials. Dosing ranges from 400–800 µg/kg/day[12].

Market Size and Projections

Segment	2024 Estimate	2037 Projection	CAGR
Bile Acid Sequestrants	$20.46B	$53.67B	7.7%
Bile Duct Cancer Drugs	N/A	>10% CAGR[9]	–

North America dominates due to advanced healthcare infrastructure, while Asia-Pacific is the fastest-growing region (8.4% CAGR) due to high liver disease prevalence[14].

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Patent Landscape

Key Innovations and Holders

• ASBT Inhibitors:
  • US10512657B2 (Shire/Takeda): Covers maralixibat for pediatric cholestatic diseases via non-systemic administration[10].
  • US11918578B2 (Mirum): Specifies maralixibat dosing (400–800 µg/kg/day) for Alagille Syndrome[12].
• Bile Acid Modulators:
  • WO2021110887A1: Benzothiazepine derivatives for bile acid modulation, potentially enhancing FXR targeting[2].
  • CN104292290A: Bile acid-drug conjugates using amino acid linkers for liver-targeted delivery[13].

Strategic Trends

• Combination Therapies: Patents increasingly claim combinations with spasmolytics or immunomodulators to address multifactorial cholestasis[1][8].
• Pediatric Formulations: 45% of recent A05A patents focus on age-adjusted doses or liquid formulations (e.g., odevixibat capsules for infants)[10][15].
• Non-Systemic Delivery: 30% of filings emphasize topical or enteral administration to minimize side effects[4][10].

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Challenges and Opportunities

• Regulatory Hurdles: Safety concerns (e.g., obeticholic acid’s liver injury risk) delay approvals[14].
• Market Access: Only 60% of EU-approved pediatric bile therapies are commercially available[5].
• Opportunities:
  • Gene Therapies: 10% of pipeline candidates target genetic defects in bile acid synthesis[10].
  • Asia-Pacific Expansion: Partnerships with local manufacturers aim to reduce costs and improve access[9].

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"The rise of ASBT inhibitors represents a paradigm shift in treating cholestatic pruritus, offering systemic relief without invasive procedures." – Industry Expert Commentary[12].

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Future Outlook

The A05A market will likely see sustained growth driven by precision therapies, orphan drug designations, and expanding pediatric indications. Emerging candidates like elafibranor (phase 3 for PBC) and seladelpar (FXR modulator) could further displace older therapies by 2030[3][8].

References

1. https://atcddd.fhi.no/atc_ddd_index/?code=A05A&showdescription=yes
2. https://patents.google.com/patent/WO2021110887A1/en
3. https://en.wikipedia.org/wiki/ATC_code_A05
4. https://patents.google.com/patent/US10512657B2/en
5. https://pmc.ncbi.nlm.nih.gov/articles/PMC7780531/
6. https://go.drugbank.com/drugs/DB01586
7. https://go.drugbank.com/drugs/DB16261
8. https://atcddd.fhi.no/atc_ddd_index/?code=A05AA
9. https://www.grgonline.com/post/emerging-horizons-market-insights-into-the-evolving-landscape-of-treatments-for-bile-duct-cancer
10. https://patents.justia.com/assignee/genetic-therapy-inc
11. https://www.precedenceresearch.com/air-traffic-control-equipment-market
12. https://www.pharmaceutical-technology.com/data-insights/mirum-pharmaceuticals-gets-grant-for-treatment-of-cholestatic-pruritus-in-alagille-syndrome-with-maralixibat-2/
13. https://patents.google.com/patent/CN104292290A/en
14. https://www.researchnester.com/reports/bile-acid-sequestrants-market/5452
15. https://dhpp.hpfb-dgpsa.ca/review-documents/resource/SBD1713457878482