Report Overview

The UK Paroxysmal Nocturnal Haemoglobinuria Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare and debilitating disease of the bone marrow that affects the blood and major organs. It is a chronic, life-threatening illness and can have devastating effects upon a patient physically, mentally, and emotionally.

PNH is an acquired illness caused by an injury to a bone marrow stem cell and is not hereditary. The bone marrow produces all blood cells for the body, being white cells, red cells, and platelets. PNH occurs when a gene required for producing normal blood cells becomes mutated resulting in a PNH clone of bone marrow cells that produce defective blood cells. The white blood cells and platelets do not function as effectively as normal cells, leaving patients prone to infections and blood clots. While all three types of blood cells are affected by PNH, most of the symptoms experienced by patients are due to the destruction of red blood cells.

The overall incidence rate was estimated at 0.35 cases per 100,000 people per year. This equates to 220 cases newly diagnosed in the UK each year. The overall prevalence rate was 3.81 per 100,000, this equates to an estimated 2,400 prevalent cases in the UK. PNH is believed to affect males and females in equal numbers. The disorder has been described in many racial groups.



Market Growth Drivers

Factors driving the PNH treatment market include a rise in the number of blood & bone marrow related disorders, increase in incidence and prevalence of patients suffering from PNH disease, rise in aging population, growing disposable income coupled with the unmet need of treatment of PNH, and technological advancements in stem cells transplantation.

In addition to this, the emergence of biologics and adoption of novel therapeutics are further pushing the market growth and anticipated to account for the higher market share over the forecast period.

Market Restraints

However, an increase in the cost of medical equipment, specifically surgical equipment required for stem cell transformation, lack of reimbursement policies, and occurrence of side effects in related currently available treatments may hamper the PNH treatment market over the forecast period.