The Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market Is Experiencing Significant Demand in the Market Owing To the Incidence Rate of the Disease Worldwide

Paroxysmal Nocturnal Hemoglobinuria, or PNH, is an inherited condition that causes the impaired production and premature death of red blood cells, mainly in the legs. The disease affects red blood cells that carry oxygen; platelets, which play a major role in blood clotting; and white blood cells that are necessary for the organism to fight infection. It can cause neurological disorders, including hypertension, impaired consciousness, unconsciousness, fever, decreased blood pressure, increased bleeding flow, and death. As this condition is an inherited disorder, most individuals with this disease do not develop any symptoms until they reach the age of 60 years old.

Symptoms commonly occur in individuals who have low plasma volume (less than eighty milligrams per deciliter of blood). However, some individuals with severe conditions may show symptoms even at the normal blood volume. Some of the more common symptoms include fatigue, leg pain, nausea, vomiting, anemia, weakness, blurred vision, nervousness, decreased vision, and difficulty concentrating. In some cases, severe complications such as sepsis, thrombosis, and infection may occur with untreated paroxysmal nocturnal hemoglobinuria.

Owing to the rise of PNH across the globe the global paroxysmal nocturnal hemoglobinuria therapeutics market is witnessing significant demand. For instance, according to Johns Hopkins Medicine, PNH affects around 1-1.5 individuals per million and is mainly traced in young adults. 30-45 years of age is the median age of diagnosis, and occasional cases are diagnosed in adolescence and childhood. Moreover, key market players are working towards the approval of newer and more advanced products for the market which is augmenting the growth of the global paroxysmal nocturnal hemoglobinuria therapeutics market.

Because of the extreme rarity of this condition, doctors cannot perform routine genetic tests to confirm the diagnosis of paroxysmal nocturnal hemoglobinuria. For genetic testing to determine whether the individual does not have this rare disorder, doctors rely on a history and a physical examination. If genetic testing confirms the existence of the mutation, doctors can perform detailed medical examinations to determine the severity of the condition and recommend treatments that are necessary for a cure. However, the high cost of the treatment is the main restraining factor, which is affecting the growth of the global paroxysmal nocturnal hemoglobinuria therapeutics market negatively.

Owing to the rise of the disease in the region, North America is witnessing high demand in the global paroxysmal nocturnal hemoglobinuria therapeutics market. For instance, according to Aplastic Anemia & MDS International Foundation, around 400 to 500 cases of the disease are diagnosed every year in the United States. All these factors have resulted in some significant development in the global paroxysmal nocturnal hemoglobinuria therapeutics market. For instance, in 2018 Apellis Pharmaceuticals' APL2 and Alexion Pharmaceuticals' Ravulizumab (Ultomiris), were given approval from the U.S. Food and Drug Administration for treatment of the disease.