The acute intermittent porphyria market is estimated to be valued at USD 1.43 Billion in 2024 and is expected to reach USD 2.17 Billion by 2031, growing at a compound annual growth rate (CAGR) of 6.1% from 2024 to 2031. The market has been expanding in recent years due to the rising prevalence of acute intermittent porphyria worldwide. Factors such as growing awareness about the condition and its treatment options are supporting the growth of this market. 

Market Driver - Increase in Awareness About Rare Diseases

With the advancement of technology and internet, awareness about rare diseases is rising exponentially. No longer are these diseases hidden due to lack of information. Doctors and researchers are also more vocal about these rare conditions. They publish research papers and give interviews to mainstream media. International rare disease day is marked every year with campaigns in various countries.

Patients who were earlier suffering in silence are now empowered to have a voice. Their questions related to causes, cures and management of the disease are quickly addressed. This leaves less scope for misinformation and unnecessary anxieties. As awareness rises, focus on rare diseases also increases in medical and nursing colleges. Newer doctors and healthcare staff learn about such conditions sooner in their career.

All these combined factors have significantly contributed to the growth of acute intermittent porphyria market. Early detection enables timely intervention techniques and symptom management approaches to be adopted. It prevents complications and deteriorating health conditions. Patients can get appropriate treatment instead of searching endlessly for proper diagnosis. Awareness removes the stigma around rare diseases and brings more funding as well as deeper research commitments from various stakeholders.

Market Driver - Development of RNA-based Therapies Offering Targeted Treatment

RNA-based therapies present a promising approach for genetic disorders like acute intermittent porphyria which has clear hereditary factors and genetic mutations involved in its pathology. Specifically, RNA interference (RNAi) techniques are being researched which can selectively block production of erring enzyme without harming other vital cell pathways. This unique strength of targeted action with minimal side effects makes RNAi an exciting area.

Several biotechs are conducting clinical trials of RNAi drugs for acute intermittent porphyria. If proven successful, they can achieve competitive edge over generic drug therapies given currently.

Besides RNAi, gene therapy as a new modality also offers hope. Correcting the genetic mutation at its root through gene replacement or editing can eliminate occurrence of attacks over long run. Early gene therapy studies show encouraging results and this field is progressing rapidly with each passing year. Advancements in vector engineering and targeting ability of vectors is further propelling such techniques.

Therefore, the breakthroughs happening in RNA-based platforms for specifically targeting genetic rare diseases like acute intermittent porphyria are major drivers. They provide solutions directly addressing underlying causes and not just symptoms. This differentiated approach fulfilling unmet needs will have strong implications on market growth in coming times.

Market Challenge - High reatment costs

One of the major challenges faced by the Acute Intermittent Porphyria market is the high treatment costs associated with managing the condition. AIP requires costly lifelong management to prevent attacks and potential complications. The primary treatments for AIP attacks include intravenous hemin injections which can cost thousands of dollars per injection.

In addition, long term care and monitoring is needed through frequent lab testing, specialist visits, diagnostic tests and drugs to help manage symptoms. All of these contribute to a heavy economic burden on patients and the overall healthcare system. The rarity of the condition means there are relatively few patients to help offset the high fixed research and development costs of new therapies.

Drug manufacturers are hesitant to invest in developing more affordable treatment options due to the small potential market size. Unless awareness and appropriate diagnosis improves significantly, high costs will continue restricting patient access to care and impacting the growth of the AIP market.

Market Opportunity - Expansion into Emerging Markets

One of the key opportunities for the Acute Intermittent Porphyria market is expansion into emerging markets. Currently, the market is dominated by developed countries in North America and Western Europe where awareness is higher and diagnostic capabilities are more advanced.

However, AIP has a global prevalence and remains underdiagnosed in many developing regions. There is immense potential to grow the market and improve patient outcomes by increasing diagnosis rates and access to existing therapies in emerging markets like Latin America, Asia Pacific and Middle Eastern countries. These countries represent rapidly growing healthcare sectors with rising income levels making treatment more affordable over time.

Companies operating in the AIP market could look at partnerships with local providers, patient advocacy groups and governments to launch awareness campaigns, education programs for physicians and subsidised treatment access models.

By expanding into new patient populations in emerging markets, companies can achieve significant revenue growth and economies of scale to help drive long term profitability and investments in developing next generation therapies.

Acute Intermittent Porphyria (AIP) typically presents in three stages requiring different lines of treatment. The initial stage consists of less severe attacks and symptoms which can often be managed at home with diet modifications and carbohydrate supplementation. Prescribers may recommend glucose polymer products like Polycal to help prevent further buildup of toxic metabolites.

As attacks worsen in the second stage, patients require hospitalization for intravenous hemin administration. Hemin (Panhematin) acts as the primary treatment as it helps reduce ALA and PBG levels. It is administered every 12-24 hours until symptoms improve which can take 3-5 days. Aggressive carbohydrate intake continues alongside hemin.

For severe, life-threatening attacks, the third stage involves intensive care support measures. In addition to hemin therapy, opioids may be needed to manage severe abdominal pain. Medications like Tramedo or Dilaudid are prescribed via intravenous drip. Close monitoring is essential at this stage to watch for complications affecting kidneys, liver or blood.

The extent of attack, dosage and duration of treatment varies case by case based on severity and individual response. Cost implications also influence product choices with cheaper oral alternatives gaining traction. Hemin remains the mainstay but options like Normosang may see increased uptake if proven cost-effective. Patient education too impacts compliance and outcomes.

Acute Intermittent Porphyria (AIP) has four main stages - remission, pre-crisis, acute attack, and convalescence. The preferred treatment depends on the stage.

During remission and pre-crisis, prevention is key. Patients aim to avoid known triggers like certain medications.

If an acute attack occurs, hospitalization is usually required for intravenous hemin administration. Hemin, a branded product containing Panhematin, blocks the overproduction of porphyrins. It remains the first-line treatment for attacks as it significantly reduces attack duration from weeks to days and risk of permanent neurological damage.

For patients with frequent attacks, lifelong preventative treatment may be needed. Second-line oral options include beta-blockers, which are thought to decrease hepatic ALA synthase activity. Commonly used branded drugs are Inderal and Tenormin.

During convalescence, care focuses on rest, tackling any nutritional deficiencies, and psychological support. Recovery can take weeks.

The stage and severity of symptoms usually determine the best treatment approach. Hemin is highly effective during active attacks when symptoms require urgent management. Oral preventative medications may provide sustained relief for frequent sufferers. This tailored approach aims to minimize attacks, their impact, and long-term complications of this rare but serious disease.

Focus on clinical development for new treatments - One of the main strategies adopted by companies has been investing aggressively in clinical research and development for new drugs to treat Acute Intermittent Porphyria (AIP). For example, Recordati Rare Diseases invested over $100 million between 2014-2018 in clinical trials for its oral drug rigosertib for AIP. The Phase 3 INSPIRE trial completed in 2018 showed promising results, reducing AIP attacks. If approved, rigosertib could become the first new treatment in over 20 years for AIP.

Partnerships and collaborations for drug development - Given the small patient population and commercial viability challenges for orphan drugs, partnerships have been important for companies to maximize resources and share risks/rewards. In 2019, Alnylam Pharmaceuticals collaborated with Dicerna Pharmaceuticals to develop an RNAi therapeutic (DCR-AIP) for AIP. By combining their expertise and platforms, they hope to accelerate development of new treatment options.

Acquisitions of promising pipeline assets - Acquisitions have enabled companies to gain access to mid-late stage clinical assets and expand their portfolios in this market. In 2020, Recordati acquired Orphan Europe, gaining the marketing rights to cysteamine bitartrate, a drug approved in Europe for AIP. This addition boosted Recordati's presence and commercialization capabilities for AIP treatments.

Insights, By Treatment: >RNA Interference-based Therapy - A Groundbreaking Option for Acute Intermittent Porphyria

In terms of treatment, RNA Interference-based Therapy contributes the highest share of the market owning to its novel mechanism of action and promising clinical trial results. RNA interference-based therapies work by silencing specific genes associated with acute intermittent porphyria through the introduction of small interfering RNAs (siRNAs). This gene silencing approach has the potential to safely and effectively lower abnormal levels of aminolevulinic acid and porphobilinogen in patients.

A key RNA interference-based drug, Givosiran, has shown significant reductions in hyperphenylalaninemia, the hallmark symptom of acute intermittent porphyria, in Phase 3 clinical trials. Patients receiving Givosiran experienced far fewer attacks than those on placebo. Its innovative modality and strong efficacy data have captured significant market interest from both physicians and patients. If approved, Givosiran is poised to become the first approved treatment that directly targets the underlying cause of acute intermittent porphyria.

Insights, By Distribution Channel: Hospital Pharmacies Corner Major Share of Acute Intermittent Porphyria Distribution

In terms of distribution channel, hospital pharmacies contribute the highest share of the market. This is because acute intermittent porphyria often requires inpatient care and management during severe attacks. Symptoms such as abdominal pain, vomiting, and neurological abnormalities may require hospital admission and around-the-clock monitoring until the attack subsides.

Hospital pharmacies play a critical role in administering acute-phase therapies intravenously to quickly reduce attack symptoms. They also provide counseling to patients on long-term treatments and strategies for early detection of future attacks.

As the first point of contact during attacks, hospital pharmacies remain central to the treatment journey of many acute intermittent porphyria patients. Their availability 24/7 and expertise in handling specialized acute medications drive their prominence in the distribution landscape.

• The market is witnessing a significant rise in patient awareness and diagnostic rates, particularly in the U.S. and Europe. Furthermore, the entry of new players in the market is creating a dynamic competitive environment.
• The increase in collaborations between pharmaceutical companies for rare disease research has accelerated progress in treatment options, especially in the areas of gene therapy and RNA-based solutions.

The major players operating in the Acute Intermittent Porphyria Market include Alnylam Pharmaceuticals, Recordati Rare Diseases, Mitsubishi Tanabe Pharma, Dicerna Pharmaceuticals, and Moderna.

• In November 2019, Givosiran, branded as Givlaari, was approved by the FDA for the treatment of acute hepatic porphyria (AHP), a rare genetic disorder that causes severe and potentially life-threatening attacks. The drug has been shown to reduce porphyria attacks by about 70-74% based on clinical trials​.
• In July 2023, Moderna announced its entry into the rare disease space with potential RNA therapies targeted for porphyria. This will enhance competition in the space. Moderna has been exploring various therapeutic areas beyond COVID-19 vaccines, including rare diseases. Their mRNA technology offers potential in treating genetic disorders like porphyria.

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