Global Porphyria Treatment Market - 2022-2029
Description
Global Porphyria Treatment Market - 2022-2029
Market Overview
The global porphyria treatment market size was valued US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).
A porphyria is a group of disorders caused by an over-accumulation of porphyrin, which helps hemoglobin, the protein that carries oxygen in the blood. Symptoms vary depending on the specific type. Acute porphyrias affect the nervous system and other organs. Cutaneous porphyrias primarily affect the skin. Possible treatments for porphyrias include avoiding triggers, taking medications to ease symptoms, delivering heme through a vein, or having blood extracted to lower iron in the body. Porphyria has no cure, although treatments are available depending on the type of sickness.
Market Dynamics
Advancement in treating congenital Erythropoietic Porphyria is expected to drive market growth.
The only curative therapeutic options for CEP are bone marrow or hematopoietic stem cell transplantation. Both are linked to high morbidity and mortality rates and must be carefully evaluated. The most significant tool of illness treatment for people who do not undergo transplants is avoiding sun and light exposure. Moreover, patients with severe hemolysis may require frequent blood transfusions. Every two to four weeks, transfusions can reduce photosensitivity by suppressing erythropoiesis and lowering porphyrin synthesis. Such therapy is likely beneficial if the patient's hematocrit remains over 35%, and they can take chelators for the iron overload. Therefore, various treatment approaches are under investigation, including genetically modified hematopoietic stem cells and proteasome inhibitors. Thus, the market is expected to drive in the forecast period from the above statements.
Restraint:
The high cost of the treatment, stringent reimbursement policies for porphyrias treatment and side effects of the drugs are some factors the market is expected to hamper in the forecast period.
COVID-19 Impact Analysis
The COVID-19 pandemic has moderately impacted healthcare systems and the market. Porphyrins interacted with SARS-COV-2 viral proteins. SARS-COV-2 viruses can generate heme from porphobilinogen and encode all necessary enzymes. Heme-binding sites are found in both E and ORF3a. The heme-iron binding sites were ORF3a's Arg134 and E's Cys44. Human cytochrome C reductase and bacterial EFeB protein had homologous domains in ORF3a. Porphyrin was coupled with the SARS-CoV-2 virus protein to create superior survivability, transmission, and attack power. The majority of porphyrin molecules are hydrophobic and agglomerate in water. The more hydrophobic porphyrin photosensitizer can penetrate mammalian cell membranes. Porphyrin diffuses through the phospholipid bilayer membrane and accumulates in the cytoplasm due to the concentration gradient. SARS-CoV-2 proteins may conduct a series of activities by binding porphyrins. On the other hand, patients were subjected to significant disruptions in their care, with most appointments and treatments being canceled, curtailed, or postponed. Almost all HCPs (90 percent) used telemedicine to replace face-to-face visits, about half of patients undergoing treatment changes switched from hospital to home therapy, and a quarter of patients had difficulty getting their medications.
Additionally, the pandemic interrupts the supply chain and process. Many companies will vary to other geographic regions to ensure that products remain available and protect their supply chain. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of the economic activities.
Segment Analysis
Medications segment is expected to hold the largest market share in porphyria treatment market
The medications segment is expected to dominate in 2021. Acute porphyria attacks are treated by focusing on treating symptoms quickly and preventing consequences. The treatment includes injections of hemin, a medication that is a form of heme, to limit the body's production of porphyrins, intravenous sugar (glucose), or sugar taken by mouth, if able, to maintain an adequate intake of carbohydrates. Moreover, in 2019, the FDA approved givosiran (Givlaari) as a monthly injection for adults with acute hepatic porphyria to reduce the number of porphyria attacks. Additionally, Taking hydroxychloroquine (Plaquenil) or, less commonly, chloroquine (Aralen), a malaria medicine, helps absorb extra porphyrins and assist your body get rid of them more quickly than usual. Only patients who cannot endure phlebotomy have been prescribed these drugs. Thus, the market segment is expected to hold the largest market share in the forecast period from the above statements.
Geographical Analysis
North America region holds the largest market share in the global porphyria treatment market
In 2021, North America accounted for the highest revenue share. The high prevalence of diseases, favorable reimbursement policies, awareness of the disease among people and FDA approvals for the products in the region are factors the market is expected to boost in the forecast period. For instance, according to the American Porphyria Foundation, Porphyria Awareness Week takes place from April 2 to April 9, 2022. Patients are invited to raise awareness in their local communities during this week while participating in various community-building initiatives. For patient-driven awareness and fund-raising efforts, the APF will provide educational materials for their physicians and other medical professionals and the general public.
Moreover, GIVLAARI (givosiran) injection for subcutaneous use to treat people with acute hepatic porphyria was approved by the United States Food and Drug Administration in November 2019. (AHP). Therefore, it has increased the demand for the treatment. Thus, the North American region is expected to hold the largest market share in the forecast period from the above statements.
Competitive Landscape
Major key players in the porphyria treatment market are Recordati Rare Diseases Inc., AmerisourceBergen Corporation, Alnylam Pharmaceuticals, Inc., CLINUVEL PHARMACEUTICALS LTD, sanofi-aventis U.S. LLC, Concordia Pharmaceuticals Inc., AdvaCare Pharma, AuroMedics Pharma LLC.
Recordati Rare Diseases Inc.:
Overview:
RRD (Recordati Rare Disorders Inc.) provides treatments for rare diseases. RRD is part of the Recordati Group, which comprises firms such as Recordati S.p.A. and Orphan Europe, among others. The Recordati Group, founded in 1926, now employs over 4,000 people worldwide. The RRD aims to provide desperately needed medicines to those suffering from highly uncommon and deadly diseases. Moreover, it collaborates with rare disease communities to raise awareness, enhance diagnosis, and increase treatment options for people living with rare diseases.
Product Portfolio:
PANHEMATIN: PANHEMATIN is a hemin for injection indicated to ameliorate recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women after initial carbohydrate therapy is known or suspected to be inadequate.
Why Purchase the Report?
Visualize the composition of the porphyria treatment market segmentation by disease type, diagnosis, treatment, end user, distribution channel, and region highlighting the key commercial assets and players.
Identify commercial opportunities in porphyria treatment market by analyzing trends and co-development deals.
Excel data sheet with thousands of data points of porphyria treatment market- level 4/5 segmentation.
PDF report with the most relevant analysis cogently put together after exhaustive qualitative interviews and in-depth market study.
Product mapping in excel for the key product of all major market players
The global porphyria treatment market report would provide an access to an approx. 45+market data table, 40+figures and 180pages.
Target Audience
Service Providers/ Buyers
Industry Investors/Investment Bankers
Education & Research Institutes
Research Professionals
Emerging Companies
Manufacturers
Market Overview
The global porphyria treatment market size was valued US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).
A porphyria is a group of disorders caused by an over-accumulation of porphyrin, which helps hemoglobin, the protein that carries oxygen in the blood. Symptoms vary depending on the specific type. Acute porphyrias affect the nervous system and other organs. Cutaneous porphyrias primarily affect the skin. Possible treatments for porphyrias include avoiding triggers, taking medications to ease symptoms, delivering heme through a vein, or having blood extracted to lower iron in the body. Porphyria has no cure, although treatments are available depending on the type of sickness.
Market Dynamics
Advancement in treating congenital Erythropoietic Porphyria is expected to drive market growth.
The only curative therapeutic options for CEP are bone marrow or hematopoietic stem cell transplantation. Both are linked to high morbidity and mortality rates and must be carefully evaluated. The most significant tool of illness treatment for people who do not undergo transplants is avoiding sun and light exposure. Moreover, patients with severe hemolysis may require frequent blood transfusions. Every two to four weeks, transfusions can reduce photosensitivity by suppressing erythropoiesis and lowering porphyrin synthesis. Such therapy is likely beneficial if the patient's hematocrit remains over 35%, and they can take chelators for the iron overload. Therefore, various treatment approaches are under investigation, including genetically modified hematopoietic stem cells and proteasome inhibitors. Thus, the market is expected to drive in the forecast period from the above statements.
Restraint:
The high cost of the treatment, stringent reimbursement policies for porphyrias treatment and side effects of the drugs are some factors the market is expected to hamper in the forecast period.
COVID-19 Impact Analysis
The COVID-19 pandemic has moderately impacted healthcare systems and the market. Porphyrins interacted with SARS-COV-2 viral proteins. SARS-COV-2 viruses can generate heme from porphobilinogen and encode all necessary enzymes. Heme-binding sites are found in both E and ORF3a. The heme-iron binding sites were ORF3a's Arg134 and E's Cys44. Human cytochrome C reductase and bacterial EFeB protein had homologous domains in ORF3a. Porphyrin was coupled with the SARS-CoV-2 virus protein to create superior survivability, transmission, and attack power. The majority of porphyrin molecules are hydrophobic and agglomerate in water. The more hydrophobic porphyrin photosensitizer can penetrate mammalian cell membranes. Porphyrin diffuses through the phospholipid bilayer membrane and accumulates in the cytoplasm due to the concentration gradient. SARS-CoV-2 proteins may conduct a series of activities by binding porphyrins. On the other hand, patients were subjected to significant disruptions in their care, with most appointments and treatments being canceled, curtailed, or postponed. Almost all HCPs (90 percent) used telemedicine to replace face-to-face visits, about half of patients undergoing treatment changes switched from hospital to home therapy, and a quarter of patients had difficulty getting their medications.
Additionally, the pandemic interrupts the supply chain and process. Many companies will vary to other geographic regions to ensure that products remain available and protect their supply chain. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of the economic activities.
Segment Analysis
Medications segment is expected to hold the largest market share in porphyria treatment market
The medications segment is expected to dominate in 2021. Acute porphyria attacks are treated by focusing on treating symptoms quickly and preventing consequences. The treatment includes injections of hemin, a medication that is a form of heme, to limit the body's production of porphyrins, intravenous sugar (glucose), or sugar taken by mouth, if able, to maintain an adequate intake of carbohydrates. Moreover, in 2019, the FDA approved givosiran (Givlaari) as a monthly injection for adults with acute hepatic porphyria to reduce the number of porphyria attacks. Additionally, Taking hydroxychloroquine (Plaquenil) or, less commonly, chloroquine (Aralen), a malaria medicine, helps absorb extra porphyrins and assist your body get rid of them more quickly than usual. Only patients who cannot endure phlebotomy have been prescribed these drugs. Thus, the market segment is expected to hold the largest market share in the forecast period from the above statements.
Geographical Analysis
North America region holds the largest market share in the global porphyria treatment market
In 2021, North America accounted for the highest revenue share. The high prevalence of diseases, favorable reimbursement policies, awareness of the disease among people and FDA approvals for the products in the region are factors the market is expected to boost in the forecast period. For instance, according to the American Porphyria Foundation, Porphyria Awareness Week takes place from April 2 to April 9, 2022. Patients are invited to raise awareness in their local communities during this week while participating in various community-building initiatives. For patient-driven awareness and fund-raising efforts, the APF will provide educational materials for their physicians and other medical professionals and the general public.
Moreover, GIVLAARI (givosiran) injection for subcutaneous use to treat people with acute hepatic porphyria was approved by the United States Food and Drug Administration in November 2019. (AHP). Therefore, it has increased the demand for the treatment. Thus, the North American region is expected to hold the largest market share in the forecast period from the above statements.
Competitive Landscape
Major key players in the porphyria treatment market are Recordati Rare Diseases Inc., AmerisourceBergen Corporation, Alnylam Pharmaceuticals, Inc., CLINUVEL PHARMACEUTICALS LTD, sanofi-aventis U.S. LLC, Concordia Pharmaceuticals Inc., AdvaCare Pharma, AuroMedics Pharma LLC.
Recordati Rare Diseases Inc.:
Overview:
RRD (Recordati Rare Disorders Inc.) provides treatments for rare diseases. RRD is part of the Recordati Group, which comprises firms such as Recordati S.p.A. and Orphan Europe, among others. The Recordati Group, founded in 1926, now employs over 4,000 people worldwide. The RRD aims to provide desperately needed medicines to those suffering from highly uncommon and deadly diseases. Moreover, it collaborates with rare disease communities to raise awareness, enhance diagnosis, and increase treatment options for people living with rare diseases.
Product Portfolio:
PANHEMATIN: PANHEMATIN is a hemin for injection indicated to ameliorate recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women after initial carbohydrate therapy is known or suspected to be inadequate.
Why Purchase the Report?
Visualize the composition of the porphyria treatment market segmentation by disease type, diagnosis, treatment, end user, distribution channel, and region highlighting the key commercial assets and players.
Identify commercial opportunities in porphyria treatment market by analyzing trends and co-development deals.
Excel data sheet with thousands of data points of porphyria treatment market- level 4/5 segmentation.
PDF report with the most relevant analysis cogently put together after exhaustive qualitative interviews and in-depth market study.
Product mapping in excel for the key product of all major market players
The global porphyria treatment market report would provide an access to an approx. 45+market data table, 40+figures and 180pages.
Target Audience
Service Providers/ Buyers
Industry Investors/Investment Bankers
Education & Research Institutes
Research Professionals
Emerging Companies
Manufacturers
Table of Contents
180 Pages
- 1. Methodology and Scope
- 1.1. Research Methodology
- 1.2. Research Objective and Scope of the Report
- 2. Market Definition and Overview
- 3. Executive Summary
- 3.1. Market Snippet by Disease Type
- 3.2. Market Snippet by Diagnosis
- 3.3. Market Snippet by Treatment
- 3.4. Market Snippet by End user
- 3.5. Market Snippet by Distribution channel
- 3.6. Market Snippet by Region
- 4. Market Dynamics
- 4.1. Market Impacting Factors
- 4.1.1. Drivers
- 4.1.1.1. Advancement in treating congenital Erythropoietic Porphyria is expected to drive market growth.
- 4.1.2. Restraints:
- 4.1.2.1. The high cost of the treatment is expected to hamper in the forecast period.
- 4.1.3. Opportunity
- 4.1.4. Impact Analysis
- 5. Industry Analysis
- 5.1. Supply Chain Analysis
- 5.2. Pricing Analysis
- 5.3. Unmet Needs
- 6. COVID-19 Analysis
- 6.1. Analysis of Covid-19 on the Market
- 6.1.1. Before COVID-19 Market Scenario
- 6.1.2. Present COVID-19 Market Scenario
- 6.1.3. After COVID-19 or Future Scenario
- 6.2. Pricing Dynamics Amid Covid-19
- 6.3. Demand-Supply Spectrum
- 6.4. Government Initiatives Related to the Market During Pandemic
- 6.5. Manufacturers Strategic Initiatives
- 6.6. Conclusion
- 7. By Disease Type
- 7.1. Introduction
- 7.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
- 7.1.2. Market Attractiveness Index, By Disease Type Segment
- 7.2. Acute Porphyrias*
- 7.2.1. Acute Intermittent Porphyria (AIP)
- 7.2.2. Hereditary Coproporphyria (HCP)
- 7.2.3. Variegate Porphyria (VP)
- 7.2.4. ALAD-Deficiency Porphyria (ADP)
- 7.2.5. Others
- 7.2.6. Introduction
- 7.2.7. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 7.3. Cutaneous Porphyrias
- 7.3.1. Porphyria Cutanea Tarda (PCT)
- 7.3.2. Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)
- 7.3.3. Congenital Erythropoietic Porphyria (CEP)
- 7.3.4. Hepatoerythropoietic Porphyria (HEP)
- 7.3.5. Others
- 8. By Diagnosis
- 8.1. Introduction
- 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 8.1.2. Market Attractiveness Index, By Diagnosis Segment
- 8.2. Blood test*
- 8.2.1. Introduction
- 8.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 8.3. Urine test
- 8.4. Genetic test
- 8.5. Others
- 9. By Treatment
- 9.1. Introduction
- 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
- 9.1.2. Market Attractiveness Index, By Treatment Segment
- 9.2. Medications*
- 9.2.1. Panhematin
- 9.2.2. GIVLAARI
- 9.2.3. SCENESSE
- 9.2.4. Hydroxychloroquine (Plaquenil)
- 9.2.5. Chloroquine (Aralen)
- 9.2.6. Others
- 9.2.7. Introduction
- 9.2.8. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 9.3. Phlebotomy
- 9.4. Others
- 10. By End user
- 10.1. Introduction
- 10.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 10.1.2. Market Attractiveness Index, By End user Segment
- 10.2. Hospitals*
- 10.2.1. Introduction
- 10.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 10.3. Blood Centers
- 10.4. Others
- 11. By Distribution Channel
- 11.1. Introduction
- 11.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
- 11.1.2. Market Attractiveness Index, By Distribution Channel Segment
- 11.2. Hospital Pharmacies*
- 11.2.1. Introduction
- 11.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 11.3. Retail Pharmacies
- 11.4. Online Pharmacies
- 11.5. Others
- 12. By Region
- 12.1. Introduction
- 12.1.1. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029, By Region
- 12.1.2. Market Attractiveness Index, By Region
- 12.2. North America
- 12.2.1. Introduction
- 12.2.2. Key Region-Specific Dynamics
- 12.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
- 12.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 12.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
- 12.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 12.2.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
- 12.2.8. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 12.2.8.1. U.S.
- 12.2.8.2. Canada
- 12.2.8.3. Mexico
- 12.3. Europe
- 12.3.1. Introduction
- 12.3.2. Key Region-Specific Dynamics
- 12.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
- 12.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 12.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
- 12.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 12.3.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
- 12.3.8. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 12.3.8.1. Germany
- 12.3.8.2. U.K.
- 12.3.8.3. France
- 12.3.8.4. Italy
- 12.3.8.5. Spain
- 12.3.8.6. Rest of Europe
- 12.4. South America
- 12.4.1. Introduction
- 12.4.2. Key Region-Specific Dynamics
- 12.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
- 12.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 12.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
- 12.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 12.4.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
- 12.4.8. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 12.4.8.1. Brazil
- 12.4.8.2. Argentina
- 12.4.8.3. Rest of South America
- 12.5. Asia Pacific
- 12.5.1. Introduction
- 12.5.2. Key Region-Specific Dynamics
- 12.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
- 12.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 12.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
- 12.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 12.5.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
- 12.5.8. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 12.5.8.1. China
- 12.5.8.2. India
- 12.5.8.3. Japan
- 12.5.8.4. Australia
- 12.5.8.5. Rest of Asia Pacific
- 12.6. Middle East and Africa
- 12.6.1. Introduction
- 12.6.2. Key Region-Specific Dynamics
- 12.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
- 12.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 12.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
- 12.6.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 12.6.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
- 13. Competitive Landscape
- 13.1. Key Developments and Strategies
- 13.2. Company Share Analysis
- 13.3. Product Benchmarking
- 13.4. List of Key Companies to Watch
- 14. Company Profiles
- 14.1. Recordati Rare Diseases Inc.
- 14.1.1. Company Overview
- 14.1.2. Product Portfolio and Description
- 14.1.3. Key Highlights
- 14.1.4. Financial Overview
- 14.2. AmerisourceBergen Corporation
- 14.3. Alnylam Pharmaceuticals, Inc.
- 14.4. CLINUVEL PHARMACEUTICALS LTD
- 14.5. sanofi-aventis U.S. LLC
- 14.6. Concordia Pharmaceuticals Inc.
- 14.7. AdvaCare Pharma
- 14.8. AuroMedics Pharma LLC
- LIST NOT EXHAUSTIVE
- 15. Global Porphyria Treatment Market – DataM
- 15.1. Appendix
- 15.2. About Us and Applications
- 15.3. Contact Us
Pricing
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