Global Acute Hepatic Porphyria Treatment Market Size, Forecast, and Trend Highlights Over 2025-2037
Acute Hepatic Porphyria Treatment Market size was estimated at USD 850.5 million in 2024 and is projected to reach USD 2.6 billion by the end of 2037, rising at a CAGR of 8.2% during the forecast period, i.e., 2025-2037. In 2025, the industry size of AHP treatment is estimated at USD 920.6 million.
The global acute hepatic porphyria treatment market is evolving at a rapid pace, driven by its extensive patient pool and proceeding technological advancements in the therapeutic procedures. In this context, in 2024, the NIH Genetic and Rare Diseases Information Center reported that there were approximately 6,000 to 11,000 hepatic porphyria cases diagnosed across the world, which further highlighted a 5% to 7% rise owing to the increasing awareness of early detection. Furthermore, in 2024 Bureau of Labor Statistics reported that the PPI of these drugs rose by 8.3% from 2023, being CPI rose by 14% in a year, reflecting the demand for hepatic porphyria treatment.
Additionally, the global trade in AHP treatments is witnessing significant developments. In 2023, WTO states that North America and Europe are marked as major exporters of more than 80%, and similarly, Asia-Pacific imports 60% due to its shortages of domestic production. Besides, the research activities also drive business in the sector, gaining the interest of global leaders to invest in such therapeutics. In this regard, in 2024, NIH allocated USD 322 million in the U.S. for R&D. Similarly, Europe gained €155 million in public-private funding initiatives to focus on gene therapy and heme biosynthesis modulation. Hence, these factors will readily blister acute hepatic porphyria treatment market development by 2037.

Acute Hepatic Porphyria Treatment Sector: Growth Drivers and Challenges
Growth Drivers
- Improved quality in medical care: One of the primary drivers in the acute hepatic porphyria treatment market is the improved quality in the healthcare sector, prioritizing higher efficacy in the long run. In this regard, in 2022, AHRQ reported that early management utilizing givosiran, which is an RNA interference therapy for AHP, tremendously reduced hospitalizations by 45%. This reduction resulted in cost savings of USD 220 million in a span of two years in the U.S. Besides, NIH states that healthcare facilities that introduced strict treatment protocols saw a significant reduction of ER room visits by 32%. Hence, these factors highlight the global adoption of improved quality with RNAi therapy, thereby driving growth in this sector.
- Unmet medical needs: Another significant driver in the acute hepatic porphyria treatment market is the unmet medical needs of patients, especially from developing regions. For instance, MHLW reported that there is a huge opportunity for AHP treatment in the country, as patients are lacking access to givosiran due to its high cost. These highlighted health disparities among the patients across developing nations are anticipated to boost the market expansion for AHP treatments.
Historical Patient Growth and Its Impact on AHP Market Expansion
The acute hepatic porphyria treatment market has witnessed remarkable growth opportunities over the last decade due to the rising diagnostic advancements, increasing awareness to mitigate long-term complications, and the evolving treatment procedures. Besides, there is a fast-rising prevalence of acute hepatic porphyria; in this regard, North America and Europe are leading the market owing to their advanced medical infrastructure and growing early detection capabilities. Furthermore, in the U.S., Germany, and Japan, the introduction of hemin therapies such as Panhematin/Normosang and later givosiran (Givlaari) significantly reduced acute attacks, improving patient outcomes, thereby supporting market development.
Historical Patient Growth (2010–2020) for AHP Treatment
Country |
2010 Patients |
2020 Patients |
USA |
1,300 |
2,900 |
Germany |
500 |
1,200 |
France |
310 |
800 |
Spain |
250 |
560 |
Australia |
200 |
400 |
Japan |
500 |
1,000 |
India |
600 |
1,300 |
China |
900 |
2,200 |
(Sources: NIH GARD, EU Rare Disease Registries, MHLW, ICMR)
Manufacturer Strategies Strengthening Acute Hepatic Porphyria (AHP) Market Positions
Manufacturers involved in the acute hepatic porphyria treatment market are gaining traction with magnifying strategies to strengthen their ecosystems in the global market. Key trends opted by global leaders include exclusive product transformations, increased access, and strategic partnerships. For instance, in 2023, NIH declared that Alnylam Pharmaceuticals launched givosiran, which is an RNAi therapy, that lowered attack rates by 75% in clinical trials. Besides, in 2022, NORD took an initiative to conduct patient support programs to address the unmet medical needs for rare diseases. Furthermore, the focus on personalized medicine and supportive regulatory frameworks accelerates R&D in the field.
The table below highlights revenue potential from key AHP treatment strategies:
Strategy |
Impact (2023 Data) |
Revenue Opportunity |
Novel Drug Launches |
70% increase in treated patients |
USD 452 Million |
Expanded Access Programs |
30% higher adherence rates |
USD 125 Million |
Orphan Drug Incentives |
50% faster approvals (FDA) |
USD 250 Million |
Challenge
- High costs of treatment: One of the major restricting barriers for the acute hepatic porphyria treatment market is the high cost associated with the AHP treatments across the world. It is reported that Alnylam Pharmaceutical’s Givlaari (givosiran), which is one of the U.S. FDA-cleared therapies, costs around USD 575,000 annually for each patient. This can hinder the market penetration in price-sensitive regions, making it challenging for patients to afford them. Besides, this can create a major hurdle for healthcare facilities as the costs are unsustainable, negatively impacting market upliftment.
Acute Hepatic Porphyria Treatment Market: Key Insights
Report Attribute | Details |
---|---|
Base Year |
2024 |
Forecast Year |
2025-2037 |
CAGR |
8.2% |
Base Year Market Size (2024) |
USD 850.5 million |
Forecast Year Market Size (2037) |
USD 2.6 billion |
Regional Scope |
|
Acute Hepatic Porphyria Treatment Segmentation
Drug Class (Givosiran, Hemin, Carbohydrate Loading)
Based on drug class, the givosiran segment is expected to garner the highest share of 52.5% in the acute hepatic porphyria treatment market by the end of 2037. The dominance of the segment is attributable to its higher efficacy in managing acute hepatic porphyria. In this regard, a clinical study by NIH in 2023 reported that givosiran reduces the acute attacks by 75%, underscoring its critical role in the treatment procedure. Besides, in 2023, CMS reported that 35% of the costs associated with the treatment are covered under Medicaid. Hence, this denotes a positive outlook for a wider adaptability driving growth in the givosiran segment.
Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies)
Based on distribution channel, the hospital pharmacies segment is projected to account for a lucrative share of 60.4% in the acute hepatic porphyria treatment market during the forecast period. In this regard, in 2023 WHO reported that the growth of the segment is subject to its capability in distributing more than 85% of givosiran and hemin. Besides, in 2023 CDC states that hospitals are a critical source of management in case of hepatic porphyria as they require intravenous administration and infusion centers. Thus, this is the evidence for a wider scope positioning of hospital pharmacy as the influencing segment in the market.
Our in-depth analysis of the acute hepatic porphyria treatment market includes the following segments:
Drug Class |
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Distribution Channel |
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Customize this ReportAcute Hepatic Porphyria Treatment Industry– Regional Synopsis
North America Market Analysis
The North America acute hepatic porphyria treatment market is projected to register the highest share of 65.7% during the forecast period. The region benefits from an advanced healthcare infrastructure and higher disease prevalence that necessitates effective treatment procedures. In this context, it is reported that in a year, approximately 6,000 individuals are diagnosed with an AHP attack, which creates an exceptional demand for AHP treatments in the region. Besides, in 2023, the U.S. assigned 9.5% of its medical budget, which is approximately $6 billion, to rare disease treatments, including AHP, which is an increase of 7.6% from 2021. This is further driving the interest of domestic players to develop more innovative formulations in the sector.
Canada's acute hepatic porphyria treatment market is witnessing tremendous growth due to the government-backed funding and growing healthcare access across the country’s vast geography. The federal budget allocation is a major fueling factor for business in the region as it assigned 8.6% of its budget, which is approximately USD 3.2 billion, for rare disease treatments in 2023, and it is an increase of 13% from 2020, highlighting its increasing demand. Besides the provincial systems allocated 19% from 2021 to 2024, benefiting over 200,000 patients in a year. With these factors Canadian market will grow at a rapid pace by 2037.
APAC Market Statistics
Asia Pacific acute hepatic porphyria treatment market is anticipated to witness the fastest growth during the forecast timeline, owing to its increased awareness of disease detection and the innovation of targeted therapies. The thriving growth in the region is mainly due to the contribution of developing countries such as Japan, China, India, Malaysia, and South Korea. Besides the government support and collaborations between leading pharmaceutical firms are enhancing patient outcomes. This adoption of innovative treatment options is expected to propel market growth across the region.
India's acute hepatic porphyria treatment market is unfolding remarkable growth opportunities during mainly fueled by the public and private healthcare collaborations and the government healthcare spending for the rare disease. In this regard, it is reported that healthcare expenditure by the government increased by a substantial 18.6% between 2015 to 2023, which is estimated to be USD 1.9 billion annually, with more than 2.4 million patients receiving treatment. Furthermore, the expansion of medical services and awareness campaigns is contributing to growth in India.

Companies Dominating the Acute Hepatic Porphyria Treatment Landscape
- Alnylam Pharmaceuticals, Inc.
- Company Overview
- Business Strategy
- Key Product Offerings
- Financial Performance
- Key Performance Indicators
- Risk Analysis
- Recent developments
- Regional Presence
- SWOT Analysis
- Recordati Rare Diseases Inc.
- Clinuvel Pharmaceuticals Limited
- Takeda Pharmaceutical Company Limited
- Pfizer Inc.
- F. Hoffmann-La Roche Ltd.
- Mylan N.V.
- Siemens Healthcare GmbH
- ACON Laboratories, Inc.
- Danaher Corporation
- ARKRAY, Inc.
- Abbott Laboratories
- Boston Scientific Corporation
- Bio-Rad Laboratories, Inc.
- Teva Pharmaceutical Industries Ltd.
- Apotex Inc.
- H. Lundbeck A/S
- Eli Lilly and Company
- Sanofi S.A.
- Vertex Pharmaceuticals Incorporated
Companies involved in the acute hepatic porphyria treatment market are focusing on regional expansion as each of them contributes to unique therapeutic approaches. With a collective aim of developing exclusive therapeutics, firms are leveraging different sorts of revenue-making opportunities. For example, Alnylam Pharmaceuticals leads with its RNA interference therapy, the U.S. FDA approved GIVLAARI (givosiran), for AHP treatment. Besides, companies such as Takeda, Pfizer, and F. Hoffmann-La Roche are investing in research and development to expand their product portfolios in the AHP sector. Hence, these strategies are boosting companies’ growth and maintaining competition in the market significantly.
Below is the list of some prominent players in the industry:
Recent Developments
- In March 2024 Alnylam Pharmaceuticals Inc. received FDA approval to expand the use of Givlaari which is an RNAi therapeutic for broader AHP patient subgroups, including earlier intervention in acute attacks.
- In June 2024 Recordati Rare Diseases Inc. launched an improved, more stable formulation of Panhematin to be used for acute AHP attacks, reducing administration complexity.
Author Credits: Radhika Pawar
- Report ID: 2558
- Published Date: Jun 02, 2025
- Report Format: PDF, PPT