News about Nplate seems to focus on the fact that the FDA missed the review deadline by about a month.
Oy vey. The drug industry in general is apparently pretty crabby about the idea that the FDA may be getting a little bit more cautious about approving new drugs after the Vioxx debacle. Heck, when you spend an average of 15 years developing a drug, you don’t want it sitting on the shelf for an extra month or two—regardless of any potential for deadly side effects.
Amgen’s platelet-stimulating drug may well have sat there for a reason.
Of course the FDA had questions. 14 of the 204 patients studied apparently had adverse reactions, eight patients developed a resistance, and there’s an increased risk of clotting or of serious bleeding when the patient stops taking the drug. The FDA was also concerned about abnormal marrow growth and malignant tumors.
“Serious adverse reactions associated with Nplate in clinical studies were bone marrow reticulin deposition and worsening thrombocytopenia after Nplate discontinuation. Additional risks include Bone Marrow Fibrosis, Thrombotic/Thromboembolic Complications, Lack or Loss of Response to Nplate, and Hematological Malignancies and Progression of Malignancy in Patients with a Pre-existing Hematological Malignancy or Myelodysplastic Syndrome (MDS),” reads Amgen’s disclaimer.
So the FDA had to figure out whether Nplate should be approved, and if so, under what—if any—conditions. Which turned out to include an ongoing long-term safety study.
Everything has consequences. Anyone with a platelet disorder that hasn’t responded to other treatments may well decide that the risks are worth it, and Amgen is to be applauded for developing a last-ditch drug. But patients deserve to be informed about what those risks are, and the drug industry should back off on criticism of the FDA’s requests for more safety data or a lengthy pause for thought.
What's your opinion? If you take Nplate, are you having any side-effects, and if so, are they what you expected? Do you think the FDA took too long to approve Nplate?
Monday, January 5, 2009
Thursday, December 18, 2008
Nplate—Top Drug Innovation for 2008?
Fred Cohen, M.D., editor of the Pharma’s Cutting Edge blog, considers both Nplate and its fellow thrombopoeitin receptor Promacta to be among the top drug innovations for 2008. Why?
“(The TPO-agonist class) represents a potential major departure in the treatment of this uncommon, costly disease.”
Patients suffering from chronic ITP, or immune thrombocytopenic purpara, suffer platelet destruction and are unable to produce enough new platelets to compensate. Ecessive bleeding may result. The condition can often resolve itself, particularly in children, and is sometimes mild enough to require monitoring rather than treatment. Chronic sufferers, however, have a tougher row to hoe. Roughly 140,000 patients are currently being treated for chronic ITP in the United States and Europe.
The treatment goal is to maintain enough platelets to avoid bleeding events.
Results from an ongoing open-label extension study were recently reported at the 2008American Society of Hematology meeting in San Francisco, California. The long-term interim data shows that treatment with Nplate, which is approved for adults that have not responded to other forms of treatment such as corticosteroids, maintains platelet levels for up to four years.
The most commonly reported side-effects included headaches (34%) and confusion (30%), fatigue (30%), and nasopharyngitis (30%).
Follow-up on Nplate’s long-tem effectiveness and safety is continuing. At this point, only some 20 patients have been followed for more than three years.
Do you suffer from chronic ITP? How are you treating it? And if you’re being treated with Nplate, how is it working for you?
“(The TPO-agonist class) represents a potential major departure in the treatment of this uncommon, costly disease.”
Patients suffering from chronic ITP, or immune thrombocytopenic purpara, suffer platelet destruction and are unable to produce enough new platelets to compensate. Ecessive bleeding may result. The condition can often resolve itself, particularly in children, and is sometimes mild enough to require monitoring rather than treatment. Chronic sufferers, however, have a tougher row to hoe. Roughly 140,000 patients are currently being treated for chronic ITP in the United States and Europe.
The treatment goal is to maintain enough platelets to avoid bleeding events.
Results from an ongoing open-label extension study were recently reported at the 2008American Society of Hematology meeting in San Francisco, California. The long-term interim data shows that treatment with Nplate, which is approved for adults that have not responded to other forms of treatment such as corticosteroids, maintains platelet levels for up to four years.
The most commonly reported side-effects included headaches (34%) and confusion (30%), fatigue (30%), and nasopharyngitis (30%).
Follow-up on Nplate’s long-tem effectiveness and safety is continuing. At this point, only some 20 patients have been followed for more than three years.
Do you suffer from chronic ITP? How are you treating it? And if you’re being treated with Nplate, how is it working for you?
Wednesday, December 3, 2008
Nplate Can Cause Bone Marrow Fibrosis
In a clinical study of nplate involving 271 patients, four patients had to discontinue treatment due to the formation of reticulin fiber deposition within the bone marrow. Six more patients were found to have reticulin progression after having a bone marrow biopsy.
Reticulin fiber deposition is the early stage of bone marrow fibrosis, also known as myelofibrosis. This is a bone disorder where fibrous scar tissue replaces bone marrow, inhibiting the patient from developing new blood cells. As fewer blood cells are generated, less platelets as well as red and white blood cells are able to form (a condition called pancytopenia). Symptoms include bone pain, bruising and easy bleeding, fatigue, shortness of breath due to anemia, and abdominal fullness caused by an enlarged spleen.
Although all the patients in the controlled studies were able to stop treatment of Nplate before their reticulin progressed to bone marrow fibrosis, one patient in an extension study did develop fibrosis. As this happens, the patients may experience very low blood cell counts.
What do you think? Nplate is a new drug with potentially unknown long term effects. Please share any opinions or experiences you have concerning Nplate.
Reticulin fiber deposition is the early stage of bone marrow fibrosis, also known as myelofibrosis. This is a bone disorder where fibrous scar tissue replaces bone marrow, inhibiting the patient from developing new blood cells. As fewer blood cells are generated, less platelets as well as red and white blood cells are able to form (a condition called pancytopenia). Symptoms include bone pain, bruising and easy bleeding, fatigue, shortness of breath due to anemia, and abdominal fullness caused by an enlarged spleen.
Although all the patients in the controlled studies were able to stop treatment of Nplate before their reticulin progressed to bone marrow fibrosis, one patient in an extension study did develop fibrosis. As this happens, the patients may experience very low blood cell counts.
What do you think? Nplate is a new drug with potentially unknown long term effects. Please share any opinions or experiences you have concerning Nplate.
Tuesday, November 25, 2008
European Medical Committee Endorses Nplate
The European Committee for Medicinal Products for Human Use (CHMP) has recommended Nplate for marketing in the European Union. Nplate (romiplostim) is currently the only long term drug on the market for treating chronic Idiopathic thrombocytopenic purpura (ITP), a condition of having low blood platelet counts.
Nplate is a fusion protein that contains characteristics of both antibodies and peptides. It works by stimulating the TPO receptor, causing bone marrow cells to grow, which make blood platelets.
There are an estimated 50,000 ITP patients in Europe, however not all of them are being recommended for Nplate. Only the patients who aren’t responding to current treatments are suggested to try Nplate. This could still be several thousands of people.
The CHMP is basing its recommendation on two different phase 3 studies where platelet counts were raised by 83% in patients from both splenectomised and non-splenectomised groups.
Before considering Nplate, patients should look at the potential side effects. Because Nplate works by stimulating bone marrow growth, there is the possibility of getting bone marrow fibrosis. Patients may also experience lower platelet counts after discontinuing Nplate compared to when started taking Nplate. Another risk is blood clot formation; as platelet counts increase, there may be a higher risk for strokes or heart attacks caused by blood clots.
What is your opinion? Please share your experiences and opinions about Nplate and ITP.
Nplate is a fusion protein that contains characteristics of both antibodies and peptides. It works by stimulating the TPO receptor, causing bone marrow cells to grow, which make blood platelets.
There are an estimated 50,000 ITP patients in Europe, however not all of them are being recommended for Nplate. Only the patients who aren’t responding to current treatments are suggested to try Nplate. This could still be several thousands of people.
The CHMP is basing its recommendation on two different phase 3 studies where platelet counts were raised by 83% in patients from both splenectomised and non-splenectomised groups.
Before considering Nplate, patients should look at the potential side effects. Because Nplate works by stimulating bone marrow growth, there is the possibility of getting bone marrow fibrosis. Patients may also experience lower platelet counts after discontinuing Nplate compared to when started taking Nplate. Another risk is blood clot formation; as platelet counts increase, there may be a higher risk for strokes or heart attacks caused by blood clots.
What is your opinion? Please share your experiences and opinions about Nplate and ITP.
Thursday, November 6, 2008
What is Idiopathic Thrombocytopenic Purpura (ITP)?
Idiopathic thrombocytopenic purpura, or immune thrombocytopenic purpura, is the condition of having low blood platelet counts. This can prevent blood from clotting, which may lead to severe, uncontrollable bleeding. Symptoms include getting bruised easily, bleeding under the skin causing red or purple spots, nosebleeds, bleeding in the gums, or urinating blood.
Broken into its parts, thrombocytopenia means having a low platelet count, idiopathic means that it has no known cause, and purpura describes the purple skin color caused from bleeding under the skin.
ITP can be acute, where it starts suddenly and improves spontaneously, or it can be chronic, where it comes on more slowly and lasts years. Acute ITP usually occurs in children after fighting a viral illness and often doesn’t return. Chronic ITP occurs more in adults and may not need treatment if platelet counts are not too low. While the cause is unknown, it seems that the immune system starts attacking blood platelets and destroys them. Antibodies erroneously coat the blood platelets, which signal the spleen to remove platelets from the body.
If your platelet count is below 20,000 you will probably need treatment. Between 20,000 and 50,000 treatment is considered on a case by case basis. And above 50,000 typically doesn’t require treatment.
Have you ever had acute or chronic ITP? If so, how have you treated it? Please share your experiences!
Broken into its parts, thrombocytopenia means having a low platelet count, idiopathic means that it has no known cause, and purpura describes the purple skin color caused from bleeding under the skin.
ITP can be acute, where it starts suddenly and improves spontaneously, or it can be chronic, where it comes on more slowly and lasts years. Acute ITP usually occurs in children after fighting a viral illness and often doesn’t return. Chronic ITP occurs more in adults and may not need treatment if platelet counts are not too low. While the cause is unknown, it seems that the immune system starts attacking blood platelets and destroys them. Antibodies erroneously coat the blood platelets, which signal the spleen to remove platelets from the body.
If your platelet count is below 20,000 you will probably need treatment. Between 20,000 and 50,000 treatment is considered on a case by case basis. And above 50,000 typically doesn’t require treatment.
Have you ever had acute or chronic ITP? If so, how have you treated it? Please share your experiences!
Tuesday, October 28, 2008
Nplate Side Effects
Nplate (romiplostim) was approved by the FDA on August 22, 2008 for treatment of chronic idiopathic thrombocytopenic purpura (ITP), a condition causing low blood platelet counts that can lead to severe, uncontrollable bleeding. Nplate works by stimulating bone marrow cells to increase the production of blood platelets. It is currently the only drug on the market that treats ITP long term and is intended to be used by patients who have not responded to other treatments. At the moment there are several serious, although uncommon, side effects to consider when beginning Nplate treatment.
Similar to thrombopoietin (TPO), a natural protein in the body, Nplate stimulates the TPO receptor, increasing bone marrow growth. This produces more platelets, which is the desired effect; however it may also cause unwanted changes in bone marrow like fibrosis. Bone marrow fibrosis (myelofibrosis) is a disorder where bone marrow is replaced with fibrous scar tissue and impairs the ability to generate new blood cells. As this happens, red and white blood cells decrease as well as the platelet count. Myelofibrosis has no known cure and eventually leads to bone marrow failure and life-threatening blood problems.
As platelet counts increase, there is also an increased risk that blood clots may form in the heart, lungs, or brain which can cause a heart attack or stroke. Additionally, if you are already taking Nplate and decide to stop treatment, you may be at a higher risk of bleeding than before you started taking Nplate. This is because your platelet count may drop below the level it was before starting Nplate treatment.
Further studies show that Nplate should not be used in patients who have blood cancer or have a precancerous blood condition called myelodysplasia because it can increase the risk of developing leukemia and other blood-related disorders.
The more common side effects of Nplate include the following:
Similar to thrombopoietin (TPO), a natural protein in the body, Nplate stimulates the TPO receptor, increasing bone marrow growth. This produces more platelets, which is the desired effect; however it may also cause unwanted changes in bone marrow like fibrosis. Bone marrow fibrosis (myelofibrosis) is a disorder where bone marrow is replaced with fibrous scar tissue and impairs the ability to generate new blood cells. As this happens, red and white blood cells decrease as well as the platelet count. Myelofibrosis has no known cure and eventually leads to bone marrow failure and life-threatening blood problems.
As platelet counts increase, there is also an increased risk that blood clots may form in the heart, lungs, or brain which can cause a heart attack or stroke. Additionally, if you are already taking Nplate and decide to stop treatment, you may be at a higher risk of bleeding than before you started taking Nplate. This is because your platelet count may drop below the level it was before starting Nplate treatment.
Further studies show that Nplate should not be used in patients who have blood cancer or have a precancerous blood condition called myelodysplasia because it can increase the risk of developing leukemia and other blood-related disorders.
The more common side effects of Nplate include the following:
- Headache
- Joint pain
- Dizziness
- Trouble sleeping
- Muscle tenderness or weakness
- Pain in arms and legs
- Abdominal pain
- Shoulder pain
- Indigestion
- Tingling or numbness in hands and feet
Do you have chronic ITP? Do you have experience with Nplate? Have you found any alternative treatments? Share your experiences!
Friday, October 17, 2008
Nplate’s Rival Drug Promacta Continues to Wait for FDA Approval
Promacta, a new drug by GlaxoSmithKline continues to wait for approval by the FDA so that it can compete with Amgen’s recently approved Nplate. Last May, the FDA advisory panel voted unanimously in favor of Promacta for short-term treatment and the FDA typically approves what their panels support. The FDA was supposed to make a decision by September 19, 2008 about whether to approve Promacta for the short term use of 6 weeks.
Both Nplate and Promacta treat Idiopathic Thrombocytopenic Purpura (ITP), a condition causing low platelet counts and problems with blood clotting. While Nplate provides long-term treatment for chronic ITP patients, Promacta will be the first to treat it short-term with a once a day oral pill.
Promacta, drug name eltrombopag, is a non-peptide thrombopoietin receptor agonist that stimulates the creation and separation of bone marrow cells that produce blood platelets. Because it is a non-peptide, it is hoped that it will raise platelet counts without suppressing the immune system. Possible side effects may include dry mouth, headache, nausea, flu-like illness, fatigue and chills.
Have you or someone you know experienced side effects from using Nplate? Please share your experiences and opinions about these drugs!
Both Nplate and Promacta treat Idiopathic Thrombocytopenic Purpura (ITP), a condition causing low platelet counts and problems with blood clotting. While Nplate provides long-term treatment for chronic ITP patients, Promacta will be the first to treat it short-term with a once a day oral pill.
Promacta, drug name eltrombopag, is a non-peptide thrombopoietin receptor agonist that stimulates the creation and separation of bone marrow cells that produce blood platelets. Because it is a non-peptide, it is hoped that it will raise platelet counts without suppressing the immune system. Possible side effects may include dry mouth, headache, nausea, flu-like illness, fatigue and chills.
Have you or someone you know experienced side effects from using Nplate? Please share your experiences and opinions about these drugs!
Friday, October 10, 2008
Rate A Drug
RateADrug.com is an interesting new site where users from the community rate their experience in using different prescription drugs. Currently there are over 3,000 medications to view and share experiences about. Detailed surveys include things such as drug effectiveness, how much and how long you have been taking the drug, changes in well being, side effects, and user comments about the drug.
This is a good way for patients to stay updated with the most current side effects as well as learn about new alternative treatments. Additionally, after you fill in your experiences with your drug, you will receive your personal risk/benefit analysis based from your question responses. This gives you a score from 1 to 10 for both your side effects and positive effects so that you can see how this drug is working for you and how that compares to other users. It's also great because you can forward all of your surveys along with comments to your doctor, family member or caregiver to keep them informed about any changes in your health caused from existing or new medications.
Go to RateADrug.com to take the Nplate drug survey and see what people are saying about it.
This is a good way for patients to stay updated with the most current side effects as well as learn about new alternative treatments. Additionally, after you fill in your experiences with your drug, you will receive your personal risk/benefit analysis based from your question responses. This gives you a score from 1 to 10 for both your side effects and positive effects so that you can see how this drug is working for you and how that compares to other users. It's also great because you can forward all of your surveys along with comments to your doctor, family member or caregiver to keep them informed about any changes in your health caused from existing or new medications.
Go to RateADrug.com to take the Nplate drug survey and see what people are saying about it.
Thursday, September 25, 2008
FDA Approves Nplate to Treat ITP
The FDA recently approved Amgen’s Nplate to treat Idiopathic Thrombocytopenic Purpura (ITP), a disorder causing low platelet counts and uncontrollable bleeding. Nplate is a fusion protein containing both peptide and antibody characteristics, which helps to raise and regulate platelet levels in chronic ITP patients. It is the first drug to provide a long-term treatment option for the estimated 60,000 chronic ITP patients in the U.S. The approval of Nplate was based on two clinical studies where chronic ITP patients successfully raised their platelet counts and sustained them for the 6 month study. Despite these positive results, there is evidence that Nplate may have some adverse side effects.
One concern is that Nplate may cause fibrous deposits in the bone marrow. Additionally, patients who discontinue using Nplate may have a lower platelet count after than before they started using it. Blood clots may also form due to excessive platelet count increases.
Nplate works by stimulating the thrombopoietin (TPO) receptor which is involved with the development of bone marrow cells that make platelets. One study showed that Nplate may increase risk of developing acute leukemia (a type of blood cancer) in patients who already have a condition called myelodysplasia. Myelodysplasia is a blood disorder connected to ineffective blood cell production. It puts patients at a higher risk of cell transformation leading to acute leukemia. In the study, 44 patients with myelodysplasia received the drug Nplate. Of them, 4 got leukemia. Further research is needed to determine if Nplate caused the development of this cancer.
Do you have chronic ITP? Do you have experience with Nplate? If so, by what means? Have you found any alternative treatments? Share your experiences!
One concern is that Nplate may cause fibrous deposits in the bone marrow. Additionally, patients who discontinue using Nplate may have a lower platelet count after than before they started using it. Blood clots may also form due to excessive platelet count increases.
Nplate works by stimulating the thrombopoietin (TPO) receptor which is involved with the development of bone marrow cells that make platelets. One study showed that Nplate may increase risk of developing acute leukemia (a type of blood cancer) in patients who already have a condition called myelodysplasia. Myelodysplasia is a blood disorder connected to ineffective blood cell production. It puts patients at a higher risk of cell transformation leading to acute leukemia. In the study, 44 patients with myelodysplasia received the drug Nplate. Of them, 4 got leukemia. Further research is needed to determine if Nplate caused the development of this cancer.
Do you have chronic ITP? Do you have experience with Nplate? If so, by what means? Have you found any alternative treatments? Share your experiences!
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