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FDA approves Pomalyst for advanced multiple myeloma

Fri, 08 Feb 2013 05:00:00 GMT

FDA NEWS RELEASE
For Immediate Release: Feb. 8, 2013
Media Inquiries: Stephanie Yao, 301-796-0394, stephanie.yao@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA approves Pomalyst for advanced multiple myeloma

The U.S. Food and Drug Administration today approved Pomalyst (pomalidomide) to treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs.

Multiple myeloma is a form of blood cancer that primarily affects older adults and arises from plasma cells in the bone marrow. According to the National Cancer Institute, approximately 21,700 Americans are diagnosed with multiple myeloma and 10,710 die yearly from the disease.

Pomalyst is a pill that modulates the body's immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).

"Pomalyst is the third drug in a class of immunomodulatory agents that includes lenalidomide and thalidomide, and is the second drug approved in the past year to treat multiple myeloma," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "Treatment for multiple myeloma is tailored to meet individual patient's needs, and today's approval provides an additional treatment option for patients who have not responded to other drugs."

In July 2012, FDA approved Kyprolis (carfilzomib) to treat multiple myeloma. Similar to Kyprolis, Pomalyst is being approved under the agency's accelerated approval program, which provides patients earlier access to promising new drugs while the company conducts additional studies to confirm the drug's clinical benefit and safe use. The therapy was also granted orphan product designation because it is intended to treat a rare disease or condition.

Pomalyst's safety and effectiveness was evaluated in a clinical trial of 221 patients with relapsed or refractory multiple myeloma. The trial was designed to measure the number of patients whose cancer completely or partially disappeared after treatment (objective response rate, or ORR). Patients were randomly assigned to receive Pomalyst alone or Pomalyst with low-dose dexamethasone, a corticosteroid.

Results showed 7.4 percent of patients treated with Pomalyst alone achieved ORR. The median duration of response has not yet been reached in these patients. In patients treated with Pomalyst plus low-dose dexamethasone, 29.2 percent achieved ORR with a 7.4-month median duration of response.

Pomalyst carries a Boxed Warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can cause severe life-threatening birth defects, and that the drug can cause blood clots.

Because of Pomalyst's embryo-fetal risk, it is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the Pomalyst REMS Program by enrolling and complying with the REMS requirements. Patients must sign a Patient-Physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements. Pharmacies must be certified with the Pomalyst REMS Program, must only dispense to patients who are authorized to receive the drug and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.

Common side effects include a decrease in infection-fighting white blood cells (neutropenia), fatigue and weakness, low red blood cell count (anemia), constipation, diarrhea, low levels of platelets in the blood (thrombocytopenia), upper respiratory tract infections, back pain and fever.

Pomalyst, lenalidomide and thalidomide are marketed by Celgene, based in Summit, N.J. Kyprolis is marketed by South San Francisco, Calif.-based Onyx Pharmaceuticals.

FDA approves Iclusig to treat two rare types of leukemia

Fri, 14 Dec 2012 05:00:00 GMT

FDA approves Iclusig to treat two rare types of leukemia

December 14, 2012 - The U.S. Food and Drug Administration today approved Iclusig (ponatinib) to treat adults with chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL), two rare blood and bone marrow diseases.

Iclusig is being approved more than three months ahead of the product's prescription user fee goal date of March 27, 2013, the date the agency was scheduled to complete review of the drug application. The FDA reviewed the Iclusig drug application under the agency's priority review program, which provides for an expedited six-month review for drugs that may provide safe and effective therapy when no satisfactory alternative therapy exists, or offer significant improvement compared to marketed products.

Iclusig blocks certain proteins that promote the development of cancerous cells. The drug is taken once a day to treat patients with chronic, accelerated, and blast phases of CML and Ph+ ALL whose leukemia is resistant or intolerant to a class of drugs called tyrosine kinase inhibitors (TKIs). Iclusig targets CML cells that have a particular mutation, known as T315I, which makes these cells resistant to currently approved TKIs.

Talon Therapeutics Announces Publication of the Marqibo(R) RALLY Study in the Journal of Clinical Oncology

Tue, 27 Nov 2012 05:00:00 GMT

Talon Therapeutics Announces Publication of the Marqibo(R) (vinCRIStine sulfate LIPOSOME injection) RALLY Study in the Journal of Clinical Oncology (JCO)

Data supported FDA accelerated approval of Marqibo (http://jco.ascopubs.org/)
Data published online ahead of print as a Rapid Communication
Data represents the per protocol, planned analysis based on established endpoints

SOUTH SAN FRANCISCO, Calif., Nov. 27, 2012 (GLOBE NEWSWIRE) -- Talon Therapeutics, Inc. (OTCBB:TLON), a biopharmaceutical company dedicated to developing leading oncology products, today announced the publication of the complete data from its Phase 2 RALLY Study of Marqibo in the Journal of Clinical Oncology, the official journal of the American Society of Clinical Oncology (ASCO). The study results were published as a rapid communication and online ahead of in print. Marqibo received FDA accelerated approval in August of this year for treatment of adult patients with Philadelphia chromosome negative (Ph-) ALL in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies.
"This publication highlights the heavily pre-treated nature of this relapsed and refractory adult ALL population," said Steven R. Deitcher, MD, President and CEO of Talon. "The results of this study, in their totality, show that Marqibo facilitates vincristine dose-intensification and achievement of complete responses. Despite universal prior vincristine treatment and residual neurotoxicity in most patients, Marqibo's adverse event profile was comparable to that historically observed with non-liposomal vincristine."

About RALLY
The pivotal Phase 2 RALLY clinical trial enrolled a total of 65 evaluable patients at 22 sites in the U.S., Canada, Germany, and Israel. The primary objective of the RALLY clinical trial was to assess the efficacy of single-agent, once per week Marqibo 2.25 mg/m2 of actual patient body surface area as assessed by achievement of complete response (CR) or CR with incomplete blood count recovery (CRi). Secondary objectives included assessments of duration of CR/CRi, overall survival (OS), ability to undergo subsequent hematopoietic cell transplantation (HCT), safety, and pharmacokinetics. Marqibo was dosed weekly based on actual body surface area without any dose capping. The study population was defined as Philadelphia chromosome-negative adult patients in second or greater relapse, or those patients who relapsed following two lines of anti-leukemia chemotherapy, including those who have previously undergone HCT.

About Marqibo^®
Marqibo is a novel, sphingomyelin/cholesterol liposome-encapsulated, formulation of vincristine sulfate. Vincristine, a microtubule inhibitor, is FDA-approved for ALL and Non-Hodgkin's Lymphoma (NHL) and is widely used in combination regimens for treatment for a variety of adult and pediatric hematologic and solid tumor malignancies. The Optisome^TM nanoparticle encapsulation technology, utilized by Talon, has been shown to provide prolonged circulation of vincristine in the blood.

Marqibo has received orphan drug designation for the treatment of ALL from the FDA and from the European Medicines Agency (EMA). Talon intends to submit a Marketing Authorization Application to the EMA in 2013.

Important safety information and full prescribing information for Marqibo can be found at www.marqibo.com.

ABVD remains standard of care for advanced Hodgkin's lymphoma

Mon, 26 Nov 2012 05:00:00 GMT

Last Updated: 2012-11-26 17:45:20 -0400 (Reuters Health)

By Will Boggs, MD

NEW YORK (Reuters Health) - Stanford V, a combined modality approach for patients with advanced Hodgkin's lymphoma, does not improve outcomes over the established combination of doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD).

"There have not been major differences among regimens for Hodgkin's lymphoma in randomized trials recently, so I think our major thrust now should be to identify molecular or immunologic markers that predict outcome (gene expression profiles, macrophage markers, EBV, etc.) and introduction of new agents (e.g., brentuximab vedotin)," said Dr. Leo I. Gordon from Northwestern University in Chicago, in an email to Reuters Health.

Dr. Gordon and colleagues in the Eastern Cooperative Oncology Group, the Cancer and Leukemia Group B, the Southwest Oncology Group, and the Canadian NCIC Clinical Trials Group compared 12 weeks of Stanford V and six to eight cycles of ABVD, with or without radiation therapy, in 794 patients with advanced or locally extensive Hodgkin's lymphoma.

All patients received radiotherapy for bulky mediastinal adenopathy beginning 2 to 3 weeks after completion of chemotherapy. In addition, in the Stanford V group, 36 Gy was delivered to any pretreatment site larger than 5 cm and for macroscopic splenic disease.

Overall response rates did not differ between treatments, with complete and clinical complete remission rates of 72.7% for ABVD and 68.7% for Stanford V, the researchers reported today online in the Journal of Clinical Oncology.

After a median 6.4 years of follow-up, there was no significant difference in five-year failure-free survival for ABVD vs Stanford V (74% vs 71%) or in five-year overall survival (88% in each group).

Patients with locally advanced disease fared better than those with stages III and IV disease, and patients with high-risk disease had better failure-free survival with Stanford V than with ABVD (67% vs 57%; p=0.02).

Although toxicity was similar in the two arms, the Stanford V patients experienced significantly more grade 3 lymphopenia, grade 3 or 4 leukocytopenia, grade 3 and 4 sensory neuropathy, and grade 3 or 4 motor neuropathy.

Second cancers occurred with equal frequency after ABVD (15 instances) and Stanford V (19 instances).

"While there are options for treatment of advanced Hodgkin's lymphoma, ABVD remains the standard of care," Dr. Gordon concluded. "That said, we have not compared the BEACOPP regimens with ABVD in a large randomized trial. The current intergroup trial is looking at the value of early imaging (PET scan) as a predictor of outcome, and while not a randomized study, this trial should provide some useful information on early imaging with PET."

Dr. Dan L. Longo from Harvard Medical School in Boston wrote an editorial that accompanied this report. He told Reuters Health, "Standard ABVD is the regimen of choice for patients with advanced stage Hodgkin's lymphoma. The main decision is whether radiation therapy should be added following six cycles of chemotherapy."

"The optimal usage of radiation therapy has not been well defined," Dr. Longo said. "In general, most people should not receive any radiotherapy. If the patient had a large mediastinal mass at initial presentation, they may benefit from added radiation therapy, but they are put at risk of second cancers and premature coronary artery disease."

"New active agents are coming along, e.g. brentuximab vedotin, that may further improve the outcome of treatment when their optimal use is defined," Dr. Longo concluded. "In the meantime, ABVD is the treatment of choice and radiation therapy should be avoided if at all possible."

In his editorial, he wrote, "There is reason to hope that the next group of changes incorporated into the management of Hodgkin lymphoma will both enhance the fraction of patients cured from between 85% and 90% to between 95% and 100% and decrease the risk of late treatment-related toxicities that can interfere with and shorten the lives of patients who are cured."

SOURCE: http://bit.ly/XXhAqc

J Clin Oncol 2012.

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FDA approves Synribo for chronic myelogenous leukemia

Fri, 26 Oct 2012 04:00:00 GMT

The U.S. Food and Drug Administration today approved Synribo to treat adults with CML. Synribo is intended to be used in patients whose cancer progressed after treatment with at least two drugs from a class called tyrosine kinase inhibitors, also used to treat CML. Synribo blocks certain proteins that promote the development of cancerous cells. Read more.

Rituximab maintenance no help in relapsed diffuse large B-cell lymphoma

Fri, 26 Oct 2012 04:00:00 GMT

Last Updated: 2012-10-26 16:10:27 -0400 (Reuters Health)

By Robert Saunders

NEW YORK (Reuters Health) - After high-dose chemo and autologous stem-cell transplant for relapsed CD20-positive diffuse large B-cell lymphoma (DLBCL), maintenance with the anti-CD20 monoclonal antibody rituximab does not improve event-free survival, researchers say.

Not only did rituximab not prevent relapse in their international trial, but it was associated with higher toxicity. "Therefore, this treatment is not recommended in relapsed DLBCL," the researchers concluded in their report online Monday in the Journal of Clinical Oncology.

"Opposite of what was observed in follicular lymphoma, the efficacy of rituximab in DLBCL is limited and may even decrease due to prior exposure and drug resistance," lead author Dr. Christian Gisselbrecht commented in an email to Reuters Health.

Dr. Gisselbrecht, at Hopital Saint Louis in Paris, France, and colleagues explain that the standard approach to relapsed DLBCL is first salvage chemotherapy. Responders may then go on to consolidation with high-dose chemotherapy, followed by autologous stem-cell transplantation.

In the current study -- prompted by some encouraging results of phase II studies -- 242 such patients were randomly assigned post-transplantation to receive rituximab every two months or to observation alone for one year. Median follow-up was 44 months.

Four-year event-free survival -- i.e., freedom from progression, relapse, new treatment, or death from any cause -- was 52% in the rituximab group and 53% in the observation group, the team found.

Furthermore, after day 100, rituximab was associated with a 15% higher rate of serious adverse events, most of which were infections.

"One of the unexpected findings in this study was an apparent benefit for women submitted to rituximab maintenance," Dr. Gisselbrecht pointed out in his email. "Is this effect by chance? Or is it related to other mechanisms such as a lower clearance of rituximab? In that case, dose modifications could be recommended but more investigations are warranted."

Overall, however, he and his colleagues conclude that patients like those in this study require innovative approaches to treatment. "Obviously, there is a need for new drugs which can be used safely after transplantation," Dr. Gisselbrecht said. "Unfortunately, most of the new drugs in development are until now more active in low grade lymphoma."

SOURCE: http://bit.ly/UNvicx

J Clin Oncol 2012.

Corticosteroids increase infections in pediatric AML

Tue, 11 Sep 2012 04:00:00 GMT

Last Updated: 2012-09-11 14:25:18 -0400 (Reuters Health)

By Will Boggs MD

NEW YORK (Reuters Health) - Corticosteroids were associated with higher rates of infection, sepsis, and infectious death in a recent study of children with acute myeloid leukemia (AML).

"Systemic corticosteroids, even when used for short periods of time, may have important negative consequences, and thus, clinicians should try to avoid administering steroids whenever possible," Dr. Lillian Sung from The Hospital for Sick Children in Toronto told Reuters Health by email.

Her group reported online September 5 in Clinical Infectious Diseases on a population-based retrospective cohort study of children with newly diagnosed AML.

Nearly two-thirds of the children (218, 63.9%) experienced at least one sterile site microbiologically documented infection, 185 (54.3%) had bacteremia, and 263 (77.1%) had a clinically documented infection.

Among 1,277 courses of chemotherapy, 313 were associated with at least one sterile site infection (24.5%), 97 with sepsis (7.6%), and 16 with infectious deaths (1.3%). The median number of days with corticosteroid administration was two per course of chemo.

In multiple regression analysis, the duration of corticosteroid exposure was an independent risk factor for microbiologically documented sterile site infection, bacteremia, clinically documented infection, and sepsis.

Prolonged neutropenia and duration of corticosteroid exposure were both independent risk factors for sepsis, but only duration of corticosteroid use was associated with infectious death.

Cytarabine dose was also independently associated with sterile site infection and bacteremia, especially from Gram-positive organisms.

"While corticosteroid exposure has previously been determined to be a risk factor for invasive fungal infections, the relationship with other infection outcomes is less well recognized," the researchers note.

"Infections continue to be a major problem for children with AML in spite of our best supportive care, and children continue to die because of them," Dr. Sung said. "Further research will need to focus on reducing infections and better understanding why they occur in some children and not in others."

"We are currently conducting a large randomized controlled trial to determine if prophylactic caspofungin can reduce invasive fungal infection more than fluconazole," Dr. Sung said. "We will be able to evaluate how this effect is influenced by steroid exposure."

SOURCE: http://bit.ly/UHGd4V

Clin Infect Dis 2012.

US FDA approves Pfizer leukemia drug

Tue, 04 Sep 2012 04:00:00 GMT

Last Updated: 2012-09-04 18:45:06 -0400 (Reuters Health)

WASHINGTON (Reuters) - U.S. health regulators on Tuesday approved Pfizer Inc's Bosulif (bosutinib) to treat chronic myelogenous leukemia (CML), a disease that usually affects older adults.

Most people with CML have a specific type of genetic mutation called the Philadelphia chromosome. This mutation causes bone marrow to make an enzyme that triggers the abnormal growth of white blood cells. Bosulif blocks the enzyme's signal that causes the white blood cells to grow.

"We are seeing improvements in the treatment of CML based on a better understanding of the molecular basis of the disease," Dr. Richard Pazdur, head of the FDA's cancer drugs center, said in a statement.

Pfizer's drug is meant for people who have CML with the Philadelphia mutation who cannot tolerate other medicines, such as Novartis AG's Gleevec, or whose cancer has stopped responding to the older treatments.

The FDA gave Bosulif orphan status, which means it treats a condition or disease that affects fewer than 200,000 people in the United States. The designation comes with a seven-year period of marketing exclusivity.

About 26,000 Americans live with the cancer, and 5,430 people in the United States expected to be diagnosed with it annually, the U.S. Food and Drug Administration said.

US FDA approves Talon's Marqibo for rare leukemia

Thu, 09 Aug 2012 04:00:00 GMT

Last Updated: 2012-08-09 17:30:06 -0400 (Reuters Health)

WASHINGTON (Reuters) - The U.S. Food and Drug Administration said on Thursday it has approved Talon Therapeutics Inc's Marqibo treatment for adults with Philadelphia chromosome negative acute lymphoblastic leukemia.

Thursday's FDA approval is for acute lymphoblastic leukemia patients who have failed at least two other therapies.

The drug, a targeted version of generic chemotherapy drug vincristine, is injected once a week by a health care professional.

According to a statement from the FDA, the drug's effectiveness was evaluated in a single clinical trial in 65 adults whose leukemia had relapsed at least twice despite standard treatments, and who had at least one previous treatment response lasting at least 90 days. Ten patients (15.4%) responded with either a complete remission or a complete remission with incomplete blood count recovery. In those 10 patients, the median duration of documented remission was 28 days. The median time to the first event of relapse, death, or next therapy was 56 days.

In two single-arm safety trials of 83 patients, 76% had serious adverse events (e.g., low white blood cell counts with fever, hypotension, respiratory distress and cardiac arrest). The most common side effects included constipation, nausea, low blood cell counts, fever, nerve damage, fatigue, diarrhea, decreased appetite, and insomnia.

Marqibo will carry a Boxed Warning that the drug must be administered only intravenously because it is deadly if administered in other ways, such as into the spinal fluid, the FDA said.

The Boxed Warning also states that Marqibo has different dosage recommendations than vincristine sulfate injection alone.

The full announcement from the FDA is available here: http://1.usa.gov/MDg01R.

FDA approves Kyprolis for some patients with multiple myeloma

Fri, 20 Jul 2012 04:00:00 GMT

The U.S. Food and Drug Administration today approved Kyprolis (carfilzomib) to treat patients with multiple myeloma who have received at least two prior therapies, including treatment with Velcade (bortezomib) and an immunomodulatory therapy. Read more.