The acute intermittent porphyria market, projected to be valued at USD 1.52 Billion in 2025, is anticipated to grow steadily, reaching USD 2.33 Billion by 2032 at a CAGR of 6.3% during the forecast period. This growth is driven by increasing diagnosis rates and greater awareness of AIP as a rare but serious genetic disorder characterized by neurovisceral attacks. Advancements in genetic testing and improved clinical recognition are enabling earlier detection and timely treatment initiation. The development and adoption of targeted therapies—such as RNA interference-based treatments and enzyme replacement options—are enhancing symptom control and reducing the frequency of acute attacks. Additionally, rising investments in orphan drug development, favorable regulatory support, and growing patient advocacy efforts are further accelerating the market growth by expanding access to innovative therapeutics and improving long-term disease management.
Market Takeaways
Acute Intermittent Porphyria Market Report Coverage
The full report is now available for purchase: https://www.coherentmi.com/industry-reports/acute-intermittent-porphyria-market
|
Report Coverage |
Details |
|
Market Revenue in 2025 |
USD 1.52 Billion |
|
Estimated Value by 2032 |
USD 2.33 Billion |
|
Growth Rate |
Poised to exhibit a CAGR of 6.3% |
|
Historical Data |
2020-2024 |
|
Forecast Period |
2025–2032 |
|
Forecast Units |
Value (USD Billion) |
|
Report Coverage |
Revenue Forecast, Competitive Landscape, Growth Factors, and Trends |
|
Segments Covered |
By Treatment and Distribution Channel |
|
Geographies Covered |
North America (U.S. and Canada), Latin America (Brazil, Argentina, Mexico, and Rest of Latin America), Europe (Italy, Spain, U.K., Germany, France, Russia, and Rest of Europe), Asia Pacific (China, India, Japan, Australia, South Korea, ASEAN, and Rest of Asia Pacific), Middle East (GCC Countries, Israel, and Rest of Middle East), and Africa (South Africa, North Africa, Central Africa) |
|
Growth Drivers |
|
|
Trends |
|
|
Opportunities |
|
|
Restraints & Challenges |
|
Market Dynamics
The acute intermittent porphyria market is experiencing steady growth, driven by improved disease recognition, expanded access to genetic testing, and increased physician awareness of rare metabolic disorders. Acute intermittent porphyria, a life-threatening condition marked by neurovisceral attacks due to toxic heme precursor accumulation, has historically been underdiagnosed, but advances in clinical guidelines and diagnostic protocols are enabling earlier and more accurate detection. This has led to a growing pool of patients eligible for targeted interventions.
The demand for RNA interference-based therapies is rising, owing to their ability to reduce levels of aminolevulinic acid (ALA) and porphobilinogen (PBG), the neurotoxic intermediates central to AIP pathophysiology. Givosiran (Givlaari), approved by the U.S. FDA and EMA, represents a major breakthrough in AIP treatment, offering sustained attack reduction and quality-of-life improvements for patients with recurrent episodes.
In December 2023, a real‑world study published in The Lancet Regional Health – Europe confirmed that givosiran (an RNA interference therapy) significantly reduced annualized attack rates and enhanced patient-reported outcomes in individuals with acute intermittent porphyria, solidifying its role as a groundbreaking, disease-modifying treatment.
Furthermore, orphan drug incentives, fast-track regulatory designations, and heightened investment in rare disease drug development are fostering a robust pipeline of next-generation porphyria treatments. As awareness spreads and healthcare infrastructure evolves in both developed and emerging markets, the AIP treatment landscape is expected to become more proactive, personalized, and innovation-driven in the years ahead.
Market Trends
There is a growing trend toward the use of RNA interference (RNAi) therapies as the preferred treatment for patients with recurrent acute intermittent porphyria attacks. These therapies, such as givosiran, work by silencing the ALAS1 gene, thereby reducing the production of toxic heme precursors like ALA and PBG. Clinical trials and real-world studies have demonstrated sustained reductions in attack frequency, hospitalizations, and the need for analgesics.
According to an article published by the National Library of Medicine in October 2024, a Phase 1/2 open-label extension study (NCT02949830) involving up to 48 months of givosiran treatment demonstrated a 97% reduction in annualized porphyria attack rates and a 96% reduction in hemin usage. Median urinary ALA and PBG levels dropped by approximately 95% and 98%, respectively, with significant improvements in patient-reported quality-of-life scores.
The diagnostic landscape for acute intermittent porphyria is shifting toward the broader implementation of genetic testing and biochemical marker profiling for early, presymptomatic identification. With many patients remaining undiagnosed until after a severe episode, there is rising emphasis on family screening, next-generation sequencing (NGS), and identification of pathogenic HMBS gene mutations.
In December 2022, a case report in the World Journal of Clinical Cases identified a new HMBS mutation (p.Arg173Trp) in a 22-year-old pregnant Chinese woman, emphasizing the vital role of genetic testing in timely and accurate AIP diagnosis, especially in atypical and reproductive-age patients.
Market Opportunities
Acute intermittent porphyria remains significantly underdiagnosed in many low- and middle-income countries (LMICs), particularly in regions such as Southeast Asia, Latin America, and Sub-Saharan Africa. These areas often lack access to genetic testing, specialist care, and awareness regarding the clinical presentation of rare metabolic disorders like AIP. With many patients experiencing prolonged diagnostic delays—often misdiagnosed with psychiatric or gastrointestinal conditions—the demand for accurate screening tools and specialized care is rising.
In LMICs, AIP frequently presents with non-specific symptoms such as abdominal pain, seizures, and confusion, which are often misdiagnosed as psychiatric or infectious conditions. For example, a case reported in a March 2022 article on the National Library of Medicine described a child in India who presented with quadriparesis and seizures. The diagnosis of AIP was significantly delayed due to the unavailability of local testing resources.
Given the episodic and potentially life-threatening nature of acute intermittent porphyria, there is a significant opportunity to integrate digital health solutions—including telemedicine platforms, AI-enabled triage systems, and remote genetic counseling tools—to support timely diagnosis and personalized care. These technologies can facilitate early intervention during acute attacks, reduce diagnostic delays, and extend specialist access to patients in rural or resource-limited regions.
Analyst View
Recent Key Developments
Competitive Landscape
Acute Intermittent Porphyria Market Segmentation
Regional Insights
Related Reports :