May 26, 2024

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More than 500 new drugs for blood diseases are in development.

More than 500 new drugs for blood diseases are in development.


More than 500 new drugs for blood diseases are in development.

Blood disorders are a broad group of disorders with different origins, symptoms and treatments.

For example, blood disorders that affect blood clotting and platelets can lead to hemophilia, blood clots, and anemia.

And diseases that affect white blood cells can lead to blood cancers, such as leukemia, lymphoma, and myeloma.

Management of blood and bleeding disorders places a huge burden on patients because such disorders often require lifelong treatment to control the condition and prevent serious complications.




Fortunately, PhRMA recently released a new report showing that the biopharmaceutical industry is taking advantage of new advances in science and technology to develop 549 drugs for blood disorders.

These drugs are already in clinical trials or under review by the U.S. Food and Drug Administration (FDA) and include:

  • 162 drugs for lymphoma , which accounts for nearly 5% of all new cancer diagnoses . Non-Hodgkin lymphoma accounts for 90% of all lymphomas .
  • 158 drugs for leukemia , the disease accounting for more than 3% of all new cancer cases . The most common types of leukemia in U.S. adults are chronic lymphocytic leukemia ( 38% ) and acute myeloid leukemia ( 31% ). In children and adolescents, 75% of leukemia cases are acute lymphoblastic leukemia.
  • 84 multiple myeloma drugs , with a projected 34,470 new cases and 12,640 deaths in 2022 .
  • 73 drugs for hematological malignancies , cancers caused by the uncontrolled division of abnormal cells that can lead to leukemia, lymphoma and multiple myeloma.
  • 55 medicines for myelodysplastic syndrome , a group of conditions that occur when blood-forming cells in the bone marrow become abnormal, resulting in a decrease in the number of blood cells.
  • 30 drugs for myeloproliferative diseases , including, for example, myelofibrosis, essential thrombocytopenia, and polycythemia vera.
  • 30 medicines for sickle cell disease , a disease of red blood cells that causes normally round, stretchy cells to form into a crescent or sickle shape. These misshapen blood cells can block blood vessels and hinder the normal flow of nutrients and oxygen throughout the body, leading to pain, organ damage and low blood counts. Sickle cell affects approximately 100,000 Americans.
  • 23 medications for various types of anemia , which affects more than 3 million Americans and is the most common bleeding disorder in the United States.
  • 19 medicines for platelet disorders, which include thrombocytopenia, a disorder characterized by abnormally low levels of platelets in the blood.
  • 18 drugs for hemophilia , a genetic disorder caused by a deficiency of factor VIII , which is type A , while type B is caused by a deficiency of factor IX (there are other, less common types).
  • 14 drugs for coagulation disorders , which mainly include deep vein thrombosis and pulmonary embolism. About 100,000 Americans die each year from blood clots.
  • 13 drugs for paroxysmal nocturnal hemoglobinuria , a rare blood disorder characterized by destruction of red blood cells, blood clots, and impaired bone marrow function.
  • 9 medicines for thalassemia , an inherited blood disorder in which the blood does not produce enough hemoglobin, causing red blood cells to not work properly for only a short time.
  • 16 medicines for other blood disorders , such as erythropoietic protoporphyria (a condition in which blood clots form in the small veins of the eye), bleeding, and retinal vein occlusion (blood clots in the veins of the eye).



More than 500 new drugs for blood diseases are in development.



Many of the 549 new drugs represent innovative approaches to blood disorders, most notably gene therapy.

Potential gene therapies may offer long-term benefits and even cure blood disorders, some in just one course of treatment.

For example, late-stage gene therapy has been able to significantly reduce bleeding rates and almost completely eliminate the need for factor replacement therapy in hemophilia A and hemophilia B patients within a few years of a one-time dose.


The potentially transformative impact of gene therapy could significantly improve patients’ quality of life, while substantially reducing the burdens and costs typically associated with current standards of care.

These savings are significant within a year after gene therapy, but may also accumulate over a patient’s lifetime.


Going forward, despite major breakthroughs in the treatment of blood disorders, incentives to encourage and promote biopharmaceutical innovation and advance existing knowledge are needed to ensure patient access to these new treatments.









PhRMA Medicines in Development for Blood Disorders in 2022 .

More than 500 new drugs for blood diseases are in development.

(source:internet, reference only)

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Important Note: The information provided is for informational purposes only and should not be considered as medical advice.