FDA Approved Drugs
The following drugs have been approved since the year 2000 for the treatment of various leukemias:
Tasigna (nilotinib)
FDA approved October 2007
This drug is approved for treatment of Philadelphia chromosome positive chronic myeloid leukemia (CML) in adult patients whose disease has progressed on or who cannot tolerate other therapies that Gleevec (imatinib) is approved for the treatment of new diagnosed patients with Philadelphia chromosome positive CML.
FDA's approval includes a black box warning for possible life-threatening heart problems that may lead to an irregular heartbeat and possible sudden death.
CML accounts for 15 percent of all leukemias in adults. Approximately 4,500 new cases of CML will be diagnosed in 2007. An abnormal chromosome, called the Philadelphia chromosome, is located in the leukemic cells and is present in the majority of CML patients.
The effectiveness of Tasigna is based on response rates observed in an ongoing clinical trial. Responses are associated with normalization of blood counts and bone marrow examinations. Further follow-up of patients is needed to determine how long these responses will last.
Patients may lower their chances for the heart problems by taking Tasigna without food, and by avoiding grapefruit products. Patients should also consult with their physician or other health care professional about avoiding other medications that can cause heart problems when taking Tasigna.
Patients with low blood potassium or magnesium should not use Tasigna.
The most common side effects include low blood counts, rash, headache, nausea and itching. Other possible serious side effects include liver damage, fluid accumulation and pancreas inflammation.
Women are advised to avoid becoming pregnant while taking Tasigna. Women who become pregnant are advised that Tasigna can harm their unborn child. Nursing mothers are advised not to breastfeed their child while taking the drug.
Oncaspar
FDA approved July 2006
This drug is approved to treat children and adults with newly diagnosed acute lymphoblastic leukemia (ALL) as part of a multiple drug chemotherapy regimen. FDA previously approved Oncaspar in 1994 only for patients with ALL who were unable to receive the cancer drug L-asparaginase because they were allergic to that drug.
The use of Oncaspar in place of the currently used drug, L-asparaginase, markedly reduces the number of drug injections required, from 21 injections of Elspar (L-asparaginase), which has been the standard of care, to three injections with Oncaspar over the 20-week course of treatment.
The approval is based on a randomized multi-center trial conducted by the Children's Cancer Group, a National Cancer Institute-funded cooperative oncology group , in 118 pediatric patients. In the trial, researchers demonstrated that Oncaspar could be safely and effectively substituted for Elspar as part of a multi-drug cancer regimen.
Serious side effects with Oncaspar include anaphylaxis (allergic shock), other serious allergic reactions, blood clots, stroke, pancreatitis, glucose intolerance and bleeding problems.
Sprycel (dasatinib)
FDA approved June 2006
This drug is approved for treatment of chronic myelogenous leukemia.
Dacogen (decitabine)
FDA approved May 2006
This drug is approved for the treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.
Arranon (nelarabine)
FDA approved October 2005
This drug is approved to treat adults and children with T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL), whose disease has not responded to or has relapsed following at least two chemotherapy regimens. Arranon is the first drug to treat this limited population of patients.
Arranon is a cancer chemotherapy drug that kills cancer cells by blocking the cell's ability to reproduce. Rapidly dividing cancer cells are more sensitive to cancer chemotherapy drugs than are more slowly dividing normal cells.
The safety and efficacy of this product were demonstrated in two clinical studies, one conducted in children and the other in adults. Both studies enrolled patients with relapsed or refractory T-ALL/T-LBL. All patients received Arranon. Among the 39 pediatric patients treated, 23 percent had a complete disappearance of their cancer. Complete disappearance lasted from 3.3 to 9.3 weeks. Of the 28 adult patients treated, the rate of complete disappearance was 21 percent and lasted from 4 to more than 195 weeks.
Common side effects reported with Arranon treatment are fatigue, nausea, vomiting and diarrhea.
Clolar (clofarabine)
FDA approved December 2004
This drug is approved for the treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens.
Vidaza (azacitidine)
FDA approved May 2004
For use for the treatment of patients with the following myelodysplastic syndrome subtypes: refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia and requiring transfusions), refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia.
Elspar (asparaginase)
FDA approved August 2003
This drug is approved for the therapy of patients with acute lymphocytic leukemia. This agent is useful primarily in combination with other chemotherapeutic agents in the induction of remissions of the disease in pediatric patients.
Gleevec (imatinib mesylate)
FDA approved May 2003
The Food and Drug Administration (FDA) today announced the approval of tablets for the treatment of pediatric patients with Philadelphia chromosome positive(Ph+)chronic myeloid leukemia (CML) in chronic phase — a rare, life-threatening form of cancer that accounts for approximately two percent of all leukemias in children.
Gleevec is indicated for children whose disease has recurred after stem cell transplant or who are resistant to interferon alpha therapy.
This drug was approved under the accelerated approval program. The program helps make products for serious or life-threatening diseases available earlier in the development process by allowing approval to be based on a promising effect of the drug, such as tumor shrinkage. As of yet, there are no controlled trials demonstrating
clinical benefit, such as improvement in disease-related symptoms or increased survival. Subsequent studies after approval will be conducted to confirm that the drug has improved survival or other clinical benefits in pediatric patients.
In addition to its original approved indication for CML refractory to other treatments in adults, and expansion
to use as a first line treatment for CML, Gleevec was also previously granted accelerated approval for the treatment of gastrointestinal stromal cancer in February, 2002.
The most frequently reported adverse events reported with the use of Gleevec are nausea, vomiting, diarrhea, edema(sometimes severe), and muscle cramps. A considerable reduction in white blood cells and platelets was also reported with Gleevec treatment.
The recommended dosage for pediatric populations is 260mg/m2/day. In children, Gleevec treatment can be given as a once daily dose or, alternatively, the daily dose may be split in two-once in the morning and once in the evening.
Gleevec (imatinib mesylate)
FDA approved December 2002
This drug is approved for the first-line treatment of patients with chronic myeloid leukemia (CML), an uncommon life-threatening form of cancer-- affecting about 40,000 people in the United States.
Gleevec was first approved in May 2001 for the advanced stages of CML under FDA's accelerated approval regulations. The drug was also indicated for use as a second-line treatment for chronic phase patients after failure of interferon-alpha therapy. At that time, further studies were needed to evaluate whether the drug provided an actual clinical benefit, such as improved survival, as well as to examine its effect when used in early stage disease.
Gleevec, a specific inhibitor of the translocation-created enzyme, works by blocking the rapid growth of white blood cells. Approval was based on a clinical trial of 1106 patients with newly diagnosed CML (chronic phase). Five hundred and fifty three patients were treated with Gleevec and 553 were given standard CML therapy with a combination of interferon-alpha and cytarabine. The patients treated with Gleevec after one year had significantly fewer cancerous cells in their blood and bone marrow. The rate of progression of disease was also decreased in the patients treated with Gleevec. Because patients with CML often live for up to 10 years with the disease, the 14-month median length of follow-up was too short to measure long term clinical benefits such as improved survival.
Gleevec is now approved for the treatment of patients with all three stages of CML -- CML myeloid blast crisis, CML accelerated phase, and CML in chronic phase, either before or after use of other therapy. The only known cure for CML is still by a stem cell (bone marrow) transplant.
The most common side effects reported with use of Gleevec include nausea, vomiting, edema (fluid retention), muscle cramps, fatigue, skin rash, and headache.
Campath (Alemtuzumab)
FDA approved May 2001
This drug is approved for the treatment of B-cell chronic lymphocytic leukemia (B-CLL) in patients who have been treated with alkylating agents and who have failed fludarabine therapy.
Trisenox (arsenic trioxide)
FDA approved September 2000
This drug is used to treat acute promyelocytic leukemia.
Mylotarg (gemtuzumab ozogamicin)
FDA approved May 2000
Treatment of CD33 positive acute myeloid leukemia in patients in first relapse who are 60 years of age or older and who are not considered candidates for cytotoxic chemotherapy.