Amgen Says FDA Approves Xgeva as a Treatment for Rare Bone-Destroying Cancer Complication

The gist: The drug denosumab (Xgeva) has been approved by the U.S. Food and Drug Administration (FDA) for treating a bone condition called hypercalcemia of malignancy. Doctors in the U.S. can now prescribe Xgeva to patients whose hypercalcemia of malignancy does not get better after treatment with drugs called bisphosphonates. Hypercalcemia of malignancy is a rare but potentially fatal complication in people with advanced cancer.

“Amgen’s Xgeva, which is used to treat bone disorders related to cancer, received an additional U.S. marketing approval.

“The Food and Drug Administration approved Xgeva as a treatment for hypercalcemia of malignancy, a condition in which a patient’s bones break down at an accelerated rate, the company said. It is related to advanced cancer and increases the risk of fractures. It can also lead to kidney failure, mental impairment, coma and death. Amgen said hypercalcemia of malignancy is considered an orphan disease, meaning there are fewer than 200,000 patients in the U.S.

“The biotech giant also sells Xgeva for the prevention of skeletal damage from solid tumors, and for inoperable cases of a rare condition called giant cell tumor of bone. The tumors destroy bones, which can cause painful fractures, joint problems, deformity and amputation. The drug is also marketed under the name Prolia as a treatment for osteoporosis.”


Drugs Home in on Bone Metastases in Prostate Cancer


Bone metastases are common in patients with metastatic castration-resistant prostate cancer (CRPC). They are associated with increased risk of death due to a number of complications such as bone fractures, compression of the spinal cord, and pain. Radiation of the affected bone sites is used as a palliative measure to relieve pain. The U.S. Food and Drug Administration (FDA) has also approved certain drugs for treatment of bone metastases in CRPC including the following: Continue reading…


Merck Receives FDA Breakthrough Therapy Designation for KEYTRUDA® (pembrolizumab) in Advanced Non-Small Cell Lung Cancer

The gist: The U.S. Food and Drug Administration (FDA) has granted “breakthrough therapy designation” to the drug Keytruda (aka pembrolizumab) for treating certain lung cancer patients. This designation means that review and approval will be accelerated so that the drug can more quickly reach patients in the U.S., outside of clinical trials. Keytruda is an immunotherapy drug, meaning that it boosts a patient’s own immune system to fight cancer. The breakthrough therapy designation specifically applies to patients with advanced non-small cell lung cancer (NSCLC) whose tumors have tested negative for EGFR mutation and ALK rearrangement, and whose disease worsened despite treatment with platinum-based chemotherapy.

“Merck (NYSE:MRK), known as MSD outside the United States and Canada, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to KEYTRUDA® (pembrolizumab), the company’s anti-PD-1 therapy, for the treatment of patients with Epidermal Growth Factor Receptor (EGFR) mutation-negative, and Anaplastic Lymphoma Kinase (ALK) rearrangement-negative non-small cell lung cancer (NSCLC) whose disease has progressed on or following platinum-based chemotherapy. This is the second Breakthrough Therapy Designation granted for KEYTRUDA.

“ ‘The FDA’s Breakthrough Therapy Designation of KEYTRUDA underscores that new treatment approaches for advanced non-small cell lung cancer continue to be needed,’ said Dr. Roger Perlmutter, president, Merck Research Laboratories. ‘Our data investigating the use of KEYTRUDA in this difficult-to-treat malignancy are very encouraging, and we look forward to working closely with the FDA to expedite our clinical program.’ ”


Lymphoseek Approved for All Solid-Tumor Cancers

The gist: Oncologists sometimes remove lymph nodes near a patient’s tumor. If the cancer is spreading, these so-called sentinel lymph nodes are likely to contain cancer cells. Therefore, it is important that oncologists be able to accurately detect sentinel lymph nodes so they can be removed and analyzed. A new system for sentinel lymph node detection has now been approved by the U.S. Food and Drug Administration (FDA) for all solid-tumor cancers, including breast cancer and melanoma. The system is called Lymphoseek.

“Approval of the Lymphoseek system for detecting sentinel lymph nodes has been extended to cover all solid-tumor cancers, its manufacturer said Wednesday.

“The FDA is also permitting the radiolabeled tracer system to also now be used with or without lymphoscintigraphy, according to Navidea Biopharmaceuticals.

“Previously, Lymphoseek had been approved in conjunction with melanomas, breast cancers, and head and neck tumors.

“The product uses a technetium-99 labeled tracer to identify lymph nodes serving areas near primary tumors, allowing oncologists to select for excision and analysis those nodes most likely to harbor emigrating cancer cells. The tracer is called tilmanocept, and it binds to CD206 receptors in lymph nodes.”


Pfizer Announces FDA Acceptance of Palbociclib New Drug Application with Priority Review

The gist: In the U.S., a drug must be approved by the U.S. Food and Drug Administration (FDA) in order for it to be prescribed to patients with specific diseases. Particularly promising drugs might be granted Priority Review, meaning that the FDA agrees to work with the drug manufacturer to accelerate the approval process. The FDA recently granted priority review to a drug meant to treat certain breast cancer patients. The drug is called palbociclib. It is meant to be combined with another drug called letrozole as a treatment for “postmenopausal women with estrogen receptor positive (ER+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer who have not received previous systemic treatment for their advanced disease.”  The FDA’s decision was based on promising results for the treatment in a clinical trial that tested it in volunteer patients. People who are interested in getting the treatment before it is approved can look into participating in Pfizer’s expanded access trial.

“Pfizer Inc. today announced the New Drug Application (NDA) for palbociclib has been accepted for filing and granted Priority Review by the United States Food and Drug Administration (FDA). This NDA requests FDA approval of palbociclib, in combination with letrozole, as a first-line treatment for postmenopausal women with estrogen receptor positive (ER+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer who have not received previous systemic treatment for their advanced disease. The submission is based on the final results of PALOMA-1, a randomized, Phase 2 trial comparing palbociclib plus letrozole versus letrozole alone in this population of patients.

“The FDA’s Priority Review status accelerates the review time from 10 months to a goal of six months from the day of acceptance of filing and is given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is April 13, 2015.

“Palbociclib received Breakthrough Therapy designation from the FDA in April 2013, for the first-line systemic treatment of women with advanced or metastatic ER+, HER2- breast cancer.

“ ‘If approved as a first-line therapy in combination with letrozole, palbociclib will be an important new option for the thousands of women in the U.S. who are living with metastatic breast cancer,’ said Garry Nicholson, president, Pfizer Oncology. ‘We look forward to continuing to work closely with the FDA through the review process.’

“Pfizer recently announced the initiation of a multi-center, open-label expanded access program (EAP) in the United States for palbociclib. Through the program, palbociclib is available to post-menopausal women with hormone receptor-positive (HR+), HER2- advanced breast cancer who are eligible for letrozole therapy and for whom enrolling in other palbociclib clinical trials is not an option. Healthcare professionals and patients can learn more about the palbociclib EAP by visiting www.clinicaltrials.gov (trial number: NCT02142868).”


FDA Approves Drug Combo for Nausea, Vomiting Caused by Chemo

The gist: With a stamp of approval from the U.S. Food and Drug Administration (FDA), doctors in the U.S. are now free to prescribe a new treatment to combat nausea and vomiting caused by chemotherapy. The treatment combines the drugs netupitant and palonosetron (Akynzeo). Akynzeo was approved on its own in 2008, and netupitant is a new drug. Clinical trials have shown that the combo works better than Akynzeo alone.


Anti–PD-1 Antibody Pembrolizumab Is Active in Ipilimumab-Refractory Advanced Melanoma

The gist: A drug called pembrolizumab (brand name Keytruda) is effective for treating people with melanoma that does not respond to treatment with the drug ipilimumab (Yervoy). That is the conclusion of a recent clinical trial—a research study with volunteer patients. Based on the study, the U.S. Food and Drug Administration (FDA) has already approved Keytruda for treating melanoma that is advanced or unresectable (can’t be removed by a surgeon) and is not responding well to other drugs.

“As reported in The Lancet by Robert et al, the anti–programmed-death receptor-1 (PD-1) antibody pembrolizumab (Keytruda) produced durable responses in a phase I trial in patients with ipilimumab (Yervoy)-refractory melanoma. The study provided the basis for the recent accelerated approval of pembrolizumab for treatment of patients with unresectable or metastatic melanoma with disease progression following treatment with ipilimumab and, in BRAF V600 mutation–positive patients, treatment with a BRAF inhibitor. Pembrolizumab is the first PD-1 inhibitor to be approved for use in the United States…

“In this open-label, international, multicenter expansion cohort of a phase I trial, 173 patients aged ≥ 18 years with advanced melanoma progressing after at least two ipilimumab doses were randomly assigned to receive pembrolizumab intravenously at 2 mg/kg (n = 89) or 10 mg/kg (n = 84) every 3 weeks until disease progression, intolerable toxicity, or consent withdrawal. The primary endpoint was overall response rate. Overall, 97% of patients were white, 17% had BRAF V600 mutant disease, and 73% had received at least two prior therapies for advanced or metastatic disease.”


FDA Eyes Faster Approval of Chemotherapy Treatments

“The Food and Drug Administration (FDA) is looking to speed up the approval process for chemotherapies and other cancer drugs to treat early-stage breast cancer, the agency announced Monday.

“In a guidance document, the FDA said it is looking to expedite the approval process to encourage industry to develop breakthrough chemotherapies, formally known as neoadjuvant treatment.

“The guidelines are intended to help pharmaceutical companies design trials that will get approved more quickly so they can begin marketing the breast cancer therapies before they’ve completed testing, the FDA noted.”


Abiraterone Acetate/Prednisone in Metastatic Castration-Resistant Prostate Cancer: Final Analysis of Early-Access Protocol Study

The gist: In 2012, the U.S. Food and Drug Administration (FDA) approved a drug called Zytiga for the treatment of metastatic castration-resistant prostate cancer. It is prescribed along with the drug prednisone. Before the FDA’s approval, the Zytiga/prednisone combination was being tested in patients in a clinical trial. The trial was an “early-access” trial, meaning that it gave patients access to a very promising treatment that had not yet been approved. All patients involved had metastatic castration-resistant prostate cancer that had worsened after chemotherapy. Recently reported results from the trial showed no new safety concerns for the treatment.

“Abiraterone acetate (Zytiga) is approved for use in combination with prednisone in the treatment of metastatic castration-resistant prostate cancer. As reported by Sternberg et al in The Lancet Oncology, results of an open-label, early-access protocol trial initiated prior to approval indicated no new safety signals with abiraterone acetate plus prednisone given after progression on chemotherapy…

“The study, conducted in 23 countries, included 2,314 patients with metastatic castration-resistant prostate cancer progressing after taxane chemotherapy enrolled between November 2010 and September 2013. Patients received abiraterone acetate 1,000 mg/day and prednisone 5 mg twice a day in 28-day cycles until disease progression, development of sustained side effects, or approval and availability of abiraterone acetate in the country of residence…

“The investigators concluded: ‘No new safety signals or unexpected adverse events were found in this early-access protocol trial to assess abiraterone acetate for patients with metastatic castration-resistant prostate cancer who progressed after chemotherapy. Future work is needed to ascertain the most effective regimen of abiraterone acetate to optimise patients’ outcomes.’ ”