Does leronlimab have orphan drug status? Also, do
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Posted On: 07/19/2025 5:25:33 PM
Does leronlimab have orphan drug status? Also, do we have any update on the GVHD?
Yes, leronlimab has received Orphan Drug Designation from the FDA for the prevention of graft-versus-host disease (GVHD).
It's important to note that while leronlimab has also been investigated for various other conditions, including HIV, metastatic triple-negative breast cancer (mTNBC), and COVID-19, its orphan drug status specifically applies to GVHD. Orphan Drug Designation is granted to encourage the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S. and provides certain benefits to the developer, such as tax credits and market exclusivity if approved.
Graft-versus-host disease (GVHD) is a serious complication that can occur after an allogeneic stem cell transplant, which is a transplant using stem cells from a donor rather than the patient's own. In GVHD, the donor's immune cells (the "graft"
recognize the recipient's body (the "host" as foreign and attack its healthy tissues and organs. ????
Causes of GVHD
GVHD arises because the donor's immune cells, particularly T cells, identify the recipient's cells as different due to variations in their human leukocyte antigens (HLAs). HLAs are proteins on the surface of cells that act as a unique identifier for each person. Even with a close HLA match, some differences can exist, triggering an immune response.
Factors that increase the risk of GVHD include:
Degree of HLA mismatch: The less the donor and recipient's HLAs match, the higher the risk.
Donor relatedness: Unrelated donors carry a higher risk than related donors, even with close matches.
Age of donor and recipient: Older age in both increases the risk.
Sex differences between donor and recipient.
Donor previously being pregnant.
Source of stem cells: Using stem cells from the bloodstream rather than bone marrow may increase risk in some cases.
Types of GVHD
GVHD can manifest in two main forms:
Acute GVHD
Acute GVHD typically develops within the first 100 days after a transplant, though it can sometimes occur later. It commonly affects the:
Skin: Rashes (often resembling a sunburn), itching, redness, blistering, and peeling.
Gastrointestinal (GI) tract: Nausea, vomiting, abdominal pain, diarrhea (which can be severe and bloody), and loss of appetite.
Liver: Jaundice (yellowing of the skin and eyes), dark urine, and elevated liver enzymes.
Chronic GVHD
Chronic GVHD usually starts more than 100 days after a transplant, but it can also develop years later or progress from acute GVHD. It can affect almost any part of the body, leading to more widespread and long-term issues. Symptoms can be mild to very severe and may include:
Skin: Dryness, tightening, hardening, darkening, itching, hair loss, and brittle nails.
Mouth: Dryness, sores, pain, sensitivity to certain foods, and difficulty eating.
Eyes: Dryness, irritation, sensitivity to light, and blurred vision.
Lungs: Persistent cough, shortness of breath, and wheezing.
Muscles and joints: Stiffness, pain, and limited movement.
GI tract: Persistent diarrhea, weight loss, and difficulty absorbing nutrients.
Genitals: Dryness, irritation, and painful intercourse.
Treatment of GVHD
The primary goal of GVHD treatment is to suppress the donor's immune response to prevent or reduce the attack on the recipient's tissues.
Corticosteroids: These are the most common first-line treatment for both acute and chronic GVHD. They work by weakening the immune system and reducing inflammation. Examples include prednisone and methylprednisolone.
Other Immunosuppressants: If steroids aren't effective or cause too many side effects, other drugs like cyclosporine, tacrolimus, mycophenolate mofetil (MMF), or sirolimus may be used.
Extracorporeal Photopheresis (ECP): This treatment involves removing a patient's blood, treating the white blood cells with a light-sensitive drug and UV light, and then returning them to the body. It's often used for chronic GVHD, especially skin involvement.
Targeted Therapies: Newer medications specifically approved for steroid-refractory chronic GVHD include ruxolitinib, ibrutinib, belumosudil, and axatilimab.
Treatment for GVHD can be prolonged, sometimes lasting years. Early diagnosis and consistent treatment are crucial to manage symptoms and prevent permanent organ damage.
Yes, leronlimab has received Orphan Drug Designation from the FDA for the prevention of graft-versus-host disease (GVHD).
It's important to note that while leronlimab has also been investigated for various other conditions, including HIV, metastatic triple-negative breast cancer (mTNBC), and COVID-19, its orphan drug status specifically applies to GVHD. Orphan Drug Designation is granted to encourage the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S. and provides certain benefits to the developer, such as tax credits and market exclusivity if approved.
Graft-versus-host disease (GVHD) is a serious complication that can occur after an allogeneic stem cell transplant, which is a transplant using stem cells from a donor rather than the patient's own. In GVHD, the donor's immune cells (the "graft"
recognize the recipient's body (the "host" as foreign and attack its healthy tissues and organs. ???? Causes of GVHD
GVHD arises because the donor's immune cells, particularly T cells, identify the recipient's cells as different due to variations in their human leukocyte antigens (HLAs). HLAs are proteins on the surface of cells that act as a unique identifier for each person. Even with a close HLA match, some differences can exist, triggering an immune response.
Factors that increase the risk of GVHD include:
Degree of HLA mismatch: The less the donor and recipient's HLAs match, the higher the risk.
Donor relatedness: Unrelated donors carry a higher risk than related donors, even with close matches.
Age of donor and recipient: Older age in both increases the risk.
Sex differences between donor and recipient.
Donor previously being pregnant.
Source of stem cells: Using stem cells from the bloodstream rather than bone marrow may increase risk in some cases.
Types of GVHD
GVHD can manifest in two main forms:
Acute GVHD
Acute GVHD typically develops within the first 100 days after a transplant, though it can sometimes occur later. It commonly affects the:
Skin: Rashes (often resembling a sunburn), itching, redness, blistering, and peeling.
Gastrointestinal (GI) tract: Nausea, vomiting, abdominal pain, diarrhea (which can be severe and bloody), and loss of appetite.
Liver: Jaundice (yellowing of the skin and eyes), dark urine, and elevated liver enzymes.
Chronic GVHD
Chronic GVHD usually starts more than 100 days after a transplant, but it can also develop years later or progress from acute GVHD. It can affect almost any part of the body, leading to more widespread and long-term issues. Symptoms can be mild to very severe and may include:
Skin: Dryness, tightening, hardening, darkening, itching, hair loss, and brittle nails.
Mouth: Dryness, sores, pain, sensitivity to certain foods, and difficulty eating.
Eyes: Dryness, irritation, sensitivity to light, and blurred vision.
Lungs: Persistent cough, shortness of breath, and wheezing.
Muscles and joints: Stiffness, pain, and limited movement.
GI tract: Persistent diarrhea, weight loss, and difficulty absorbing nutrients.
Genitals: Dryness, irritation, and painful intercourse.
Treatment of GVHD
The primary goal of GVHD treatment is to suppress the donor's immune response to prevent or reduce the attack on the recipient's tissues.
Corticosteroids: These are the most common first-line treatment for both acute and chronic GVHD. They work by weakening the immune system and reducing inflammation. Examples include prednisone and methylprednisolone.
Other Immunosuppressants: If steroids aren't effective or cause too many side effects, other drugs like cyclosporine, tacrolimus, mycophenolate mofetil (MMF), or sirolimus may be used.
Extracorporeal Photopheresis (ECP): This treatment involves removing a patient's blood, treating the white blood cells with a light-sensitive drug and UV light, and then returning them to the body. It's often used for chronic GVHD, especially skin involvement.
Targeted Therapies: Newer medications specifically approved for steroid-refractory chronic GVHD include ruxolitinib, ibrutinib, belumosudil, and axatilimab.
Treatment for GVHD can be prolonged, sometimes lasting years. Early diagnosis and consistent treatment are crucial to manage symptoms and prevent permanent organ damage.
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