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.’ ”


FDA Advisory Panel Calls for More Data on HIFU Treatment

“A US Food and Drug Administration (FDA) advisory committee voted October 1 not to recommend approval, at least for now, of a high-intensity focused ultrasound (HIFU) device to treat some patients with recurrent prostate cancer.

“Members of the Gastroenterology and Urology Devices Panel of the FDA’s Medical Devices Advisory Committee encouraged the device manufacturer to continue to conduct research but to come back when the ongoing pivotal trial at 20 sites in the United States and Canada is completed, not halfway through.

“SonaCare Medical Devices of Charlotte, North Carolina, had requested FDA approval of its Sonablate 450, a computer-controlled device with a probe that clinicians can use to deliver HIFU energy through thermal ablation to the prostate. The proposed indication was for treatment of biopsy-proven recurrent cancer in patients, low to high risk, who have failed primary external beam radiation therapy and have prostate-specific antigen (PSA) readings lower than 10 ng/mL.

“SonaCare based its request on an interim analysis of results from a nonrandomized, single-group clinical trial involving the first 100 of a planned 200 patients. Patient ages ranged from 53 to 83 years (mean, 69.7 years), they were mostly white (76%), and they had pretreatment PSA readings ranging from 0.4 to 14 (mean, 4.9).

“Of the first 100 patients enrolled at 16 sites, 78 completed 12 months of follow-up and had a biopsy, and 22 did not.”


The FDA Gives an Ariad Drug Candidate ‘Breakthrough Therapy Designation’

The gist: A new lung cancer treatment called AP26113 has shown promise for certain people with metastatic non-small cell lung cancer (NSCLC). Specifically, it is meant to treat people whose tumors have mutations in the ALK gene and who are resistant to the standard drug crizotinib. It has been tested in volunteer patients in clinical trials. The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for AP26113, meaning that review and approval will be accelerated so that the drug can more quickly reach patients in the U.S., outside of clinical trials.

“Ariad Pharmaceuticals Inc. of Cambridge said Thursday that federal regulators have granted “breakthrough therapy designation” to a drug candidate for a form of lung cancer.

“According to the Food and Drug Administration’s website, a breakthrough therapy designation means that the FDA will expedite the development and review of such drug. The designation is given to drug candidates designed to treat a serious or life threatening disease that have shown great promise based on preliminary clinical evidence.

“Ariad’s drug candidate, currently dubbed AP26113, is for the treatment of patients with anaplastic lymphoma kinase positive metastatic non-small cell lung cancer who are resistant to crizotinib, an existing treatment on the market.

“Ariad’s press release cited data from the American Cancer Society. According to the society, non-small cell lung cancer is the most common form of lung cancer, accounting for about 85 percent of the estimated 228,190 new cases of lung cancer diagnosed each year in the United States.”


Aldoxorubicin Receives FDA Orphan Drug Designations for Glioblastoma, Small Cell Lung Cancer, and Ovarian Cancer

The gist: When a newly developed drug for a rare (“orphan”) disease seems particularly promising for patients, the U.S. Food and Drug Administration (FDA) may choose to grant it “orphan drug designation.” The designation removes certain barriers that might otherwise keep a drug company from being able to successfully develop and profit from the drug in the U.S. A new drug called aldoxorubicin has just received an orphan drug designation for the treatment of small cell lung cancer (SCLC), glioblastoma multiforme, and ovarian cancer. Aldoxorubicin is a special version of the chemotherapy drug doxorubicin. It allows for higher doses of doxorubicin with fewer side effects.

“The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to aldoxorubicin in three indications: glioblastoma multiforme, small cell lung cancer, and ovarian cancer. Aldoxorubicin combines doxorubicin with a novel single-molecule linker that binds directly and specifically to albumin, allowing greater doses of the chemotherapeutic agent to be administered while reducing its toxic side effects.

“Aldoxorubicin is currently being studied in a global phase III clinical trial evaluating the efficacy and safety of aldoxorubicin as a second-line treatment for patients with soft-tissue sarcoma. CytRx, a biopharmaceutical research and development company, is also evaluating aldoxorubicin in two phase II clinical trials, one in patients with late-stage glioblastoma multiforme and the other in HIV-related Kaposi’s sarcoma. A global phase IIb trial in patients with relapsed small cell lung cancer is expected to commence later this month, and the company is undertaking a phase Ib combination study of aldoxorubicin plus gemcitabine as a potential precursor to a trial in relapsed ovarian cancer.”


UPDATE 1-U.S. FDA Approves Expanded Use of Medivation Prostate Cancer Drug

The gist: When a drug company creates a new cancer treatment, the treatment must be approved by the U.S. Food and Drug Administration (FDA) before doctors in the U.S. can prescribe it. An FDA approval for a new drug usually specifies the particular kinds of patients who are allowed to be treated. In 2012, drug called Xtandi was FDA-approved for treating people with metastatic castration-resistant prostate cancer (mCRPC) who have been previously but unsuccessfully treated with chemotherapy. Now, the FDA has also approved Xtandi for people with mCRPC who have not yet tried chemotherapy.

“U.S. health regulators approved the use of Medivation Inc’s and Astellas Pharma Inc’s advanced prostate cancer drug Xtandi in men who have not yet received chemotherapy, the companies said on Wednesday, significantly expanding the potential patient population for the oral medicine.

“The expanded Food and Drug Administration approval will also enable the drug to better compete with Johnson & Johnson’s Zytiga. The approval triggers $90 million in milestone payments to Medivation by Japan’s Astellas under a collaboration agreement.

“Xtandi, known chemically as enzalutamide, originally gained U.S. approval in 2012 for use in patients with castration-resistant prostate cancer that has spread beyond the prostate only after they had first received chemotherapy treatment.

” ‘The average duration of treatment should double and the addressable patient population triple in the pre-chemo setting,’ Sanford Bernstein analyst Geoffrey Porges said in a research note earlier this week.”


FDA Grants Fast Track Designation to PEGPH20 Program for Metastatic Pancreatic Cancer

Editor’s note: The U.S. Food and Drug Administration (FDA) has granted a “Fast-Track” designation to a new treatment for people with advanced pancreatic cancer. The treatment combines the drugs PEGPH20, gemcitabine, and nab-paclitazel. The Fast-Track designation means that the FDA will facilitate a faster approval process so that the treatment can soon be prescribed by oncologists in the U.S.

“The FDA granted fast track designation today to Halozyme Therapeutics for its PEGPH20 program in treating patients with pancreatic cancer, according to a company press release.

The program seeks to demonstrate improved overall survival in metastatic pancreatic cancer patients by studying the use of pegylated recombinant human hyaluronidase in combination with gemcitabine and nab-paclitaxel.

“The FDA’s fast track designation for our PEGPH20 program in pancreatic cancer underscores the significant need for new treatment options for pancreatic cancer patients with advanced disease,” Helen Torley, MD, Halozyme president and CEO, said in the release. “We look forward to continuing to work with the FDA on this program to explore whether patients with metastatic pancreatic cancer can benefit from this therapy.”