Porphyria Treatment Market Size, Share, Industry, Forecast and outlook (2024-2031)

Porphyria Treatment Market is Segmented By Disease Type (Acute Porphyrias, and Cutaneous Porphyrias), By Diagnosis (Blood test, Urine test, Genetic test, Others), By Treatment (Medications, Phlebotomy, and Others), By End-user (Hospitals, Blood Centers, Others), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies, Others), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Global Industry Analysis 2024-2031

Market Overview

The global porphyria treatment market size is growing at a CAGR of 9.2% during the forecast period (2024-2031). 

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 porphyria treatments are available depending on the type of sickness.

As per DataM Intelligence, porphyria treatment market study analysis offers an in-depth outlook on the market containing quantitative and qualitative data. It gives an outlook and forecast of the global market based on market segmentation. It also provides global porphyria treatment market size, and growth, along with the latest trends, opportunities, and forecast till 2029 for the global market with esteem to major countries such as the United States, Canada, Brazil, Germany, Italy, Spain, United Kingdom, Russia, European countries, United Arab Emirates, Saudi Arabia, South Africa, Japan, China, India, South Korea, Australia, and rest of the countries over the globe.

Among all regions, the North American region is expected to hold the largest share of the global market over the forecast period. Porphyria treatment market in the United States and Canada produces the utmost share. Whereas the European porphyria treatment market is projected to continue its presence globally during the period of 2022- 2029.

Market Summary

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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 economic activities.

Segment Analysis:

The medications segment is expected to hold the largest market share in the porphyria treatment market

By Disease Type:

• Acute Porphyrias
  • Acute Intermittent Porphyria (AIP)
  • Hereditary Coproporphyria (HCP)
  • Variegate Porphyria (VP)
  • ALAD-Deficiency Porphyria (ADP)
  • Others
• Cutaneous Porphyrias
  • Porphyria Cutanea Tarda (PCT)
  • Erythropoietic Protoporphyria (EPP)
  • X-Linked Protoporphyria (XLP)
  • Congenital Erythropoietic Porphyria (CEP)
  • Hepatoerythropoietic Porphyria (HEP)
  • Others

By Diagnosis:

• Blood test
• Urine test
• Genetic test
• Others

By Treatment:

• Medications
  • Panhematin
  • GIVLAARI
  • SCENESSE
  • Hydroxychloroquine (Plaquenil)
  • Chloroquine (Aralen)
  • Others
• Phlebotomy
• Others

By End-user:

• Hospitals
• Blood Centers
• Others

By Distribution Channel:

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies
• Others

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 assists 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.