Global Paroxysmal Nocturnal Hemoglobinuria Treatment Market - 2022-2029

Global Paroxysmal Nocturnal Hemoglobinuria Treatment Market - 2022-2029

Market Overview

The global paroxysmal nocturnal hemoglobinuria treatment market size was valued at 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).

PNH (paroxysmal nocturnal hemoglobinuria) is a rare and life-threatening blood disease. The disease symptoms are red blood cell destruction, blood clots, and impaired bone marrow function. Aplastic anemia and PNH are closely linked.

Market Dynamics

The factors influencing the global paroxysmal nocturnal hemoglobinuria treatment market are the increasing research and development and the growing prevalence of paroxysmal nocturnal hemoglobinuria.

The increasing research and development are expected to drive the market growth in the forecast period

PNH (paroxysmal nocturnal hemoglobinuria) is an acquired (not inherited) disorder that causes the premature death of blood cells and impairs their production. The disorder affects red blood cells (erythrocytes), which transport oxygen, white blood cells (leukocytes), which fight infections, and platelets (thrombocytes), which help the body clot. PNH can strike anyone at any age, but it is mostly diagnosed in young adults.

PNH patients are more likely to develop blood-forming cells (leukemia) cancers. People who have or have been treated for aplastic anemia, a type of blood disease, may develop PNH in some cases. The signs and symptoms of PNH go away in a small percentage of people. PNH treatment used to be mostly supportive. Patients received a blood transfusion and iron supplementation for recurrent hemolysis and anemia. To prevent thrombosis, they were given anti-thrombosis prophylaxis. An allogeneic bone marrow transplant was offered for bone marrow complications. Eculizumab is a life-saving therapy that reduces the need for blood transfusions by more than half and reduces the risk of thrombotic events and serious vascular complications by nearly seventy percent. Eculizumab works by preventing factor C5 from working. Membrane attack complexes are formed by all complement pathways (MAC). C3b, C5b, and other complement factors combine to form MAC. Several new complement inhibitors are currently being tested in humans to treat PNH. Some of these new therapeutic agents block the 5th component of complement, similar to eculizumab/ravulizumab, while others inhibit complement at other sites.

Side effects associated with paroxysmal nocturnal hemoglobinuria medication are expected to hamper the market growth

Some of the side effects of eculizumab are the onset, duration, and severity of eculizumab side effects are frequently predictable. Eculizumab side effects are almost always reversible and disappear once treatment is finished. The side effects of eculizumab may be manageable. There are numerous ways to reduce or avoid the negative effects of eculizumab.

For patients taking eculizumab, the following side effects are common (occurring in more than 30% of cases) blood pressure problems, diarrhea, and headache.

COVID-19 Impact Analysis

The coronavirus disease (COVID-19) pandemic has complicated cancer treatment. Important issues include balancing the risk of treatment delay versus COVID-19 harm, minimizing the negative effects of social distancing during care delivery, and allocating limited health care resources appropriately and fairly. COVID-19 infection results in an overactive complement system and an inflammatory response that worsens lung injury and has poor clinical outcomes. Because of the interaction between the complement system and COVID-19 infection, immunosuppression may be a viable option for reducing the effects of complement-mediated inflammatory destruction in COVID-19 infection. Furthermore, patients with PNH may be vulnerable to COVID-19 complications due to their compromised immune status. The therapeutic use of complement inhibitors may be an effective strategy to control systemic inflammation in COVID-19 infection based on this immunological rationale. Specific clinical and economically cost-effective therapeutic options should be explored and prioritized considering the worldwide COVID19 pandemic. In a patient with PNH, treatment with ravulizumab during COVID19 pneumonia was safe, cost-effective, and had a favorable clinical course. Hence, covid-19 is expected to have impacted the market growth.

Segment Analysis

Stem Cell Transplant segment is expected to dominate the market growth

PNH patients can only be treated with allogeneic stem cell transplantation. Clinical results from various SCT programs show that this procedure is only appropriate for a small number of patients with similar rare disorders. Only severe cases of PNH with life-threatening complications such as transformation to leukemia or aplastic anemia should be treated with stem cell transplants. By eliminating the defective clone with conditioning intensity and/or the graft-versus-haematopoiesis effect and replacement with a donor haematopoietic system, logeneic HSCT remains a potentially curative option for PNH.

However, because of the high treatment-related mortality, HSCT for classical PNH is not recommended in countries where eculizumab is available, even though it is a therapeutic option for patients with PNH/BMF. Moreover, because eculizumab is unavailable in China, steroid and allogeneic HSCT are usually considered first-line therapy for classical PNH and PNH/BMF. In a randomized trial of patients with PNH, eculizumab treatment has never been directly compared to HSCT.

Geographical Analysis

North America region is expected to hold the largest market share in the global paroxysmal nocturnal hemoglobinuria treatment market

The regional market is expected to grow due to the potential approval of promising pipeline drugs and the growing prevalence of paroxysmal nocturnal hemoglobinuria.

While epidemiologic data is limited, it is estimated that 5,000-6,000 people in the United States are affected by PNH. Moreover, on 21st December 2018, Ravulizumab, a long-acting complement inhibitor that prevents hemolysis, was approved by the US Food and Drug Administration (FDA) to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Hence, the increasing drug approvals in this region are expected to drive market growth.

Competitive Landscape

With mergers, acquisitions, and product launches, the global paroxysmal nocturnal hemoglobinuria treatment market is moderately competitive. Some of the key players in the market are Alexion Pharmaceuticals, Inc., Akari Therapeutics, Plc, Novartis AG, Apellis Pharmaceuticals, Inc., Regeneron Pharmaceuticals

Alexion Pharmaceuticals, Inc.

Overview: Alexion Pharmaceuticals, a subsidiary of AstraZeneca, is a Boston-based pharmaceutical company that specializes in orphan drugs for the treatment of rare diseases. The company was founded in 1992.

Product Portfolio: Soliris is a complement inhibitor that is used to reduce hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Key Development: On 7th June 2021, the US Food and Drug Administration (FDA) approved Alexion Pharmaceuticals, Inc.'s ULTOMIRIS (ravulizumab-cwvz) in children and adolescents with paroxysmal nocturnal hemoglobinuria (PNH) (PNH). The first and only FDA-approved medicine for children and adolescents with PNH is ULTOMIRIS, a long-acting C5 inhibitor that provides immediate, complete, and sustained complement inhibition.

Why Purchase the Report?

Visualize the composition of the global paroxysmal nocturnal hemoglobinuria treatment market segmentation by treatment and end-users highlighting the key commercial assets and players.

identify commercial opportunities in global paroxysmal nocturnal hemoglobinuria treatment market by analyzing trends and co-development deals.

Excel data sheet with thousands of data points of global paroxysmal nocturnal hemoglobinuria 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 paroxysmal nocturnal hemoglobinuria treatment market report would provide an access to an approx. 45+market data table, 40+figures and 180 pages.

Target Audience

Service Providers/ Buyers

Industry Investors/Investment Bankers

Education & Research Institutes

Research Professionals

Emerging Companies

Manufacturers