Acute Hepatic Porphyria Treatment Market Trends

Growth Drivers

• Advancements in diagnostic capabilities and disease awareness: Initially more accurate diagnosis was critical and difficult for expanding the treatable patient pool. Various initiatives such as NIH funded on research related to genetic testing and biomarker identification to reduce the traditional diagnostic delay. As per the NLM report on December 2024, the combined biochemical and genetic testing increases the diagnosis rate by up to 18 %, from 56% to 74%, highlighting the advances in the diagnosis. Further, the number of diagnosed patients eligible for treatment grows, directly driving demand for therapeutic interventions.
• 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.
• Investment in research on novel therapeutic modalities: The continuous R&D in enhancing the treatment in AHP is improving gradually. The investment is directed towards next-gen therapies that provide more convenient administration for oral formulations, improved safety profiles, and novel mechanisms of action that overcome the main cause of the disease. The NIH report in January 2025 has stated that USD 48 billion is allocated to medical research focusing on enhancing life and minimizing illness and disability. Continuous innovation makes the evolution in the treatment environment and drives the market further.

Therapeutic siRNAs in clinical trials

Source: NLM January 2023

Challenges

• 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 very high 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.
• Orphan drug pricing pressures and caps: Government-imposed robust price control is the key restraint for both industry players and customers. The clinical added value (ASMR), which directly influences the negotiated price in France, is assessed by the Transparency Committee (HAS). An ASMR level V rating with minor improvement translates into severe pricing restraints. Japan's NHI system also gives a robust cost-effectiveness evaluation, which tends to result in prices that are less than in the U.S., reducing profit margins for manufacturers entering the market.