Global Patisiran Market Outlook
Revenue, 2024
$1.5B
Forecast, 2034
$3.9B
CAGR, 2025 - 2034
9.6%
The Patisiran industry revenue is expected to be around $1.7 billion in 2025 and expected to showcase growth with 9.6% CAGR between 2025 and 2034. The pharmaceutical industry maintains Patisiran as a relevant drug because of its strong growth pattern which results from multiple essential factors. The worldwide increase in hereditary transthyretin-mediated amyloidosis cases together with insufficient treatment choices has driven Patisiran market growth. The RNA interference mechanism of Patisiran which targets hATTR genes has established itself as a revolutionary genetic disorder treatment approach thus driving its market importance.
Patisiran, also known as Onpattro, functions as an innovative therapeutic solution which provides distinctive benefits for hATTR treatment. The drug uses RNAi technology to decrease liver production of disease-causing proteins. The mechanism of Patisiran has established it as a leading personalized medicine solution. The rising use of RNAi therapeutics together with the expanding focus on individualized patient care has been key to increasing Patisiran demand.
Market Key Insights
- The Patisiran market is projected to grow from $1.5 billion in 2024 to $3.9 billion in 2034. This represents a CAGR of 9.6%, reflecting rising demand across Treatment of Hereditary Transthyretin-Mediated Amyloidosis (hATTR) and Management of Polyneuropathy.
- Alnylam Pharmaceuticals Inc., Ionis Pharmaceuticals Inc., Arrowhead Pharmaceuticals Inc. are among the leading players in this market, shaping its competitive landscape.
- U.S. and Germany are the top markets within the Patisiran market and are expected to observe the growth CAGR of 7.0% to 10.1% between 2024 and 2030.
- Emerging markets including Brazil, India and South Africa are expected to observe highest growth with CAGR ranging between 9.2% to 12.0%.
- Transition like Advancements in Gene Silencing is expected to add $109 million to the Patisiran market growth by 2030.
- The Patisiran market is set to add $2.3 billion between 2024 and 2034, with manufacturer targeting Specialty Neurology Clinics & Home Healthcare End User projected to gain a larger market share.
- With The rising prevalence of hereditary transthyretin-mediated amyloidosis, and Advancements in rna interference technology, Patisiran market to expand 150% between 2024 and 2034.
Opportunities in the Patisiran
The delivery of Patisiran can also be improved through the use of new drug delivery systems. Nanocarriers can improve the delivery of Patisiran to the target site, thus increasing its therapeutic effect and reducing its side effects. This could lead to a wider acceptance and higher demand for Patisiran.
Growth Opportunities in North America and Europe
The North American market has experienced substantial growth of Patisiran because of rising hereditary ATTR amyloidosis cases in the United States. The regions strong healthcare system together with major pharmaceutical companies operating in the region have driven up Patisiran market demand. Multiple pharmaceutical companies compete fiercely for market share in this competitive environment. The main drivers in this region stem from increasing Patisiran benefit awareness and RNAi therapeutic progress and positive reimbursement policies.
The European market shows substantial demand for Patisiran because the United Kingdom, Germany and France serve as its main customer bases. The high number of hereditary transthyretin-mediated amyloidosis cases in these countries drives the need for Patisiran as an effective treatment. Multiple pharmaceutical companies actively compete in this market through their research and development efforts to improve their product effectiveness. The main market drivers in this region are the increasing geriatric population, the prevalence of rare diseases, and the strong presence of healthcare facilities. The European Medicines Agencys approval of Patisiran has created favorable market conditions for this product.
Market Dynamics and Supply Chain
01
Driver: The Rising Prevalence of Hereditary Transthyretin-Mediated Amyloidosis, and Increased Investment in Rare Disease Research
The RNA interference therapeutic Patisiran functions as a specific treatment for hereditary transthyretin-mediated amyloidosis which also represents a rare fatal genetic disease. The increasing number of hATTR cases worldwide also creates a growing need for effective treatments which also drives the expansion of the Patisiran market. The growing focus on rare diseases and the increasing investment in research and development activities are also other significant s for the Patisiran market. The heightened interest from pharmaceutical companies and the support from regulatory bodies for orphan drug development are also fostering the growth of the Patisiran market.
RNA interference technology advancements have also resulted in the creation of new therapeutic approaches such as Patisiran. The advanced technology enables the targeted suppression of disease-causing genes which provides fresh treatment possibilities for patients with hATTR genetic disorders. The Patisiran market also advances because of ongoing technological progress and increasing acceptance of RNA interference therapeutics.
02
Restraint: High Cost of Treatment
The RNA interference therapeutic patisiran represents a major advancement for treating hereditary transthyretin-mediated amyloidosis. The expensive nature of this treatment might restrict its ability to reach more patients. The expensive production process of this drug results from its advanced manufacturing methods which require specialized equipment and innovative technologies. The expensive nature of this treatment could slow down market growth because many patients and healthcare systems lack the financial means to purchase it.
03
Opportunity: Expanding into Untapped Markets and Strategic Collaborations for Research and Development
The RNA interference therapeutic Patisiran demonstrates substantial promise for treating hereditary transthyretin-mediated amyloidosis. The market availability of Patisiran remains restricted to developed nations. The market expansion of Patisiran into developing countries presents a major opportunity because these nations have high hATTR prevalence rates yet restricted access to advanced treatments like Patisiran. The expansion of Patisiran into new markets has the potential to generate substantial growth for the product.
RNAi therapeutics continue to transform at a fast pace because scientists discover new breakthroughs and develop innovative solutions on a regular basis. Through strategic partnerships with research institutions and biotech companies Patisiran can access recent developments which will lead to better formulations and new applications. The brands reputation would improve through these collaborations which would drive market growth and increase market share.
04
Challenge: Limited Awareness and Understanding
Despite its potential benefits, there is a lack of widespread awareness and understanding of Patisiran and its mechanism of action. This limited knowledge could hinder the adoption of Patisiran in the market. Moreover, the complexity of the disease it treats may also contribute to this , as hereditary transthyretin-mediated amyloidosis is a rare condition that is often misdiagnosed or underdiagnosed.
Supply Chain Landscape
1
Raw Material Procurement
Sigma-AldrichThermo Fisher Scientific
2
Drug Synthesis
Alnylam PharmaceuticalsSanofi Genzyme
3
Drug Formulation & Packaging
Catalent Pharma SolutionsWest Pharmaceutical Services
4
Distribution & End User
McKesson CorporationCardinal HealthAmerisourceBergen
1
Raw Material Procurement
Sigma-AldrichThermo Fisher Scientific
2
Drug Synthesis
Alnylam PharmaceuticalsSanofi Genzyme
3
Drug Formulation & Packaging
Catalent Pharma SolutionsWest Pharmaceutical Services
4
Distribution & End User
McKesson CorporationCardinal HealthAmerisourceBergen
Use Cases of Patisiran in Treatment of Hereditary Transthyretin-Mediated Amyloidosis (hATTR) & Management of Polyneuropathy
Treatment of Hereditary Transthyretin-Mediated Amyloidosis (hATTR) : Patisiran, an RNA interference therapeutic, is primarily used in the treatment of hATTR. This rare, rapidly progressive, and life-threatening disease is caused by mutations in the transthyretin gene. Patisiran works by targeting and silencing specific messenger RNA, preventing the production of TTR protein before it is made. This helps to reduce the accumulation of TTR deposits in peripheral nerves, heart, and gastrointestinal tract, improving quality of life and life expectancy for patients. Alnylam Pharmaceuticals, the developer of Patisiran, holds a strong market position in this application owing to their pioneering work in RNAi therapeutics.
Management of Polyneuropathy : Patisiran is also widely used in the management of polyneuropathy in adult patients with hATTR. By reducing the production of TTR protein, Patisiran helps to slow the progression of nerve damage, thereby managing the symptoms of polyneuropathy. This application of Patisiran offers a unique advantage as it directly targets the root cause of the disease. Alnylam Pharmaceuticals, with its innovative RNAi technology, is a key player in this market segment.
Recent Developments
Onpattro, a revolutionary RNA interference therapeutic, has been making significant strides in the global pharmaceutical market. Recent advancements in the Onpattro landscape have been largely driven by its effectiveness in treating hereditary transthyretin-mediated amyloidosis , a rare, life-threatening disease. The drugs unique mechanism of action, which involves silencing specific genetic material, has positioned it as a trailblazer in the RNAi therapeutics sector.
December 2024 : Alnylam Pharmaceuticals, the manufacturer of Patisiran, announced the expansion of its production facilities to meet the increasing global demand for Patisiran
October 2024 : Patisiran received regulatory approval in Japan, opening a new market for Alnylam Pharmaceuticals
August 2024 : A significant breakthrough in clinical trials demonstrated Patisirans potential in treating other rare genetic diseases beyond its initial application for hereditary transthyretin-mediated amyloidosis.
Impact of Industry Transitions on the Patisiran Market
As a core segment of the Pharmaceutical industry,
the Patisiran market develops in line with broader industry shifts.
Over recent years, transitions such as Advancements in Gene Silencing and Expansion in Personalized Medicine have redefined priorities
across the Pharmaceutical sector,
influencing how the Patisiran market evolves in terms of demand, applications and competitive dynamics.
These transitions highlight the structural changes shaping long-term growth opportunities.
01
Advancements in Gene Silencing
The pharmaceutical industry underwent substantial changes after Patisiran became available as a new gene silencing drug. The RNA interference-based therapy has transformed the medical treatment of hereditary transthyretin-mediated amyloidosis. The drug functions by stopping the creation of harmful proteins which provides hope for treating this uncommon genetic condition. The transition has created substantial effects which result in better patient results and decreased hospital stays and better life quality for patients. This industry transition is expected to add $109 million in the industry revenue between 2024 and 2030.
02
Expansion in Personalized Medicine
The development and approval of Patisiran signifies a major shift towards personalized medicine. This transition is characterized by therapies tailored to individual patients genetic makeup, enhancing treatment efficacy and minimizing adverse effects. Patisirans success has encouraged pharmaceutical companies to invest more in genetic research and personalized therapies, fostering innovation and growth in the industry.