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

• By Treatment Type, RNA Interference-based Therapies are projected to dominate the acute intermittent porphyria market with a 57.3% share in 2025. Their dominance is driven by their novel mechanism of action, which targets and reduces the production of toxic heme precursors, and by promising clinical outcomes demonstrated by therapies like givosiran. These treatments offer targeted, disease-modifying benefits that significantly reduce the frequency and severity of acute attacks.
• By Distribution Channel, Hospital Pharmacies are expected to account for the largest share of 43.7% in 2025. This is due to the frequent need for acute care interventions, including intravenous treatments and pain management, which are typically administered in hospital settings during severe porphyric crises. The complexity of care and requirement for specialized administration further reinforce the dominance of hospital-based dispensing.
• Regionally, North America is expected to lead the acute intermittent porphyria market in 2025, supported by well-established rare disease networks, strong reimbursement frameworks, increased awareness among healthcare professionals, and early adoption of advanced therapies. The presence of major pharmaceutical players and ongoing investment in orphan drug development also contribute to the region's leadership in market share.

Acute Intermittent Porphyria Market Report Coverage

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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

• Adoption of RNA Interference-Based Therapies as First-Line Treatment

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.

• Increasing Focus on Genetic and Biomarker-Based Diagnostics for Early Detection

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

• Expansion into Underserved and Low-Resource Markets

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.

• Integration of Digital Health Tools for Acute Symptom Management and Remote Consultation

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

• The acute intermittent porphyria market is entering a transformative phase, driven by rising clinical awareness, enhanced diagnostic capabilities, and the emergence of RNA interference-based therapies as standard treatment. With the greater inclusion of AIP in rare disease registries and medical education curricula, earlier diagnosis, and proactive disease management are becoming more achievable, especially in high-income countries.
• The approval and adoption of givosiran, the first RNAi therapy for AIP, has redefined clinical expectations for disease control. As data from real-world studies continue to validate its efficacy in reducing annualized attack rates, hospitalization needs, and hemin dependency, the therapy is positioned to become a first-line choice for patients with recurrent episodes. Continued investment in similar disease-modifying therapeutics, including gene-based approaches, is likely to expand the treatment arsenal in the coming decade.
• Technological convergence is reshaping patient care in acute intermittent porphyria. From remote genetic counseling and AI-based triage tools to wearable-enabled symptom monitoring, digital health platforms are emerging as key enablers of early intervention and personalized therapy. These tools are especially vital for geographically dispersed or mobility-limited populations, enabling timely care access and real-time clinical insights.
• The market is also witnessing a shift toward equity in rare disease treatment, with orphan drug frameworks and global awareness campaigns fostering increased access to care in underserved regions. However, high treatment costs and limited diagnostic infrastructure remain barriers in low- and middle-income countries. Strategic partnerships with governments, NGOs, and diagnostics firms will be crucial to unlocking market potential in these geographies.
• Looking forward, the acute intermittent porphyria treatment landscape is poised to evolve into a precision medicine-driven ecosystem. With advancements in genetic profiling, biomarker-based monitoring, and novel delivery systems, the future of AIP care will likely emphasize individualized treatment strategies, proactive prevention of attacks, and improved quality of life outcomes for patients globally.

Recent Key Developments

• In September 2023, Moderna, Inc. announced significant business and clinical pipeline progress during its annual R&D Day. The company reported positive Phase 3 results for its mRNA-based influenza vaccine, marking a third consecutive success in respiratory disease programs following earlier advancements in COVID-19 and RSV. CEO Stéphane Bancel emphasized that Moderna is preparing for near-term product launches across its oncology, latent, rare, and infectious disease franchises. Additionally, the company anticipates providing pivotal data in Q4 2025 on its next-generation COVID and flu combination vaccine (mRNA-1083) and updated efficacy results from its Phase 2 INT study, signaling robust pipeline momentum and a strong focus on execution.

Competitive Landscape

• Alnylam Pharmaceuticals
• Recordati Rare Diseases
• Mitsubishi Tanabe Pharma
• Dicerna Pharmaceuticals
• Moderna

Acute Intermittent Porphyria Market Segmentation

• By Treatment
• RNA Interference-based Therapy
• Givosiran
• Symptomatic Treatment
• By Distribution Channel
• Hospital Pharmacies
• Online Pharmacies
• Retail Pharmacies

Regional Insights

• North America
  • U.S.
  • Canada
• Latin America
  • Mexico
  • Brazil
  • Argentina
  • Rest of Latin America
• Europe
  • Italy
  • Germany
  • U.K.
  • Spain
  • France
  • Russia
  • Rest of Europe
• Asia Pacific
  • South Korea
  • India
  • Japan
  • China
  • Australia
  • ASEAN
  • Rest of Asia Pacific
• Middle East
  • GCC Countries
  • Israel
  • Rest of Middle East
• Africa
  • South Africa
  • North Africa
  • Central Africa