RUVICA (ibrutinib) capsules approved for treatment-naïve CLL patients

In continuation of my update on ibrutinib

The U.S. Food and Drug Administration (FDA) has approved IMBRUVICA® (ibrutinib) capsules for treatment-naïve patients with chronic lymphocytic leukemia (CLL). The approval is based on data from the Phase 3 RESONATE-2 (PCYC-1115) study, the first head-to-head clinical trial comparing IMBRUVICA to a chemotherapy agent. Results showed IMBRUVICA significantly extended progression-free survival (PFS; the primary endpoint) and increased overall response rate (ORR; a key secondary endpoint) compared to chlorambucil in previously untreated patients with CLL age 65 or older. IMBRUVICA is now approved for use in all lines of CLL therapy, considerably expanding the number of patients who may benefit from this treatment. This broadens the indication beyond the initial CLL approval in February 2014 for the treatment of patients with CLL who have received at least one prior therapy and in July 2014 for CLL patients with del 17p, a genetic mutation typically associated with poor treatment outcomes. IMBRUVICA is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.

On a related front, the National Comprehensive Cancer Network® (NCCN) published an update on February 17 to its Clinical Practice Guidelines for non-Hodgkin's lymphomas recommending IMBRUVICA for certain first-line CLL patients.

"People living with CLL who have not been previously treated now have an option that significantly improved progression-free survival when compared to the oral chemotherapy used in the RESONATE-2 trial," said Jan Burger, M.D., Ph.D., Associate Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX and RESONATE-2 study lead investigator. "The results seen in the RESONATE-2 clinical trial are truly compelling and make this medicine an attractive first-line treatment option for clinicians in the hematology space."

RUVICA (ibrutinib) capsules approved for treatment-naïve CLL patients:

The U.S. Food and Drug Administration has approved IMBRUVICA (ibrutinib) capsules for treatment-naïve patients with chronic lymphocytic leukemia (CLL).

Novartis announces FDA approval of Afinitor for progressive, nonfunctional neuroendocrine tumors of GI

 

In continuation of my update  on Everolimus 

Novartis today announced that the United States Food and Drug Administration (FDA) approved Afinitor® (everolimus) tablets for the treatment of adult patients with progressive, well-differentiated, nonfunctional neuroendocrine tumors (NET) of gastrointestinal (GI) or lung origin that are unresectable, locally advanced or metastatic. Afinitor received a priority review designation providing a shortened review period for drugs that treat serious conditions and offer a significant improvement in safety or effectiveness.

"Afinitor is the first treatment approved for progressive, nonfunctional NET of lung origin, and one of very few options available for progressive, nonfunctional GI NET, representing a shift in the treatment paradigm for these cancers," said Bruno Strigini, President, Novartis Oncology. "We are proud of our Afinitor development program, which has translated to meaningful benefits for patients with several different cancers and rare diseases."

Neuroendocrine tumors are a rare type of cancer that originate in neuroendocrine cells throughout the body, and are most often found in the GI tract, lungs or pancreas. NET can be defined as functional or nonfunctional. Functional NET are characterized by symptoms caused by the oversecretion of hormones and other substances. Nonfunctional NET may be characterized by symptoms caused by tumor growth, such as intestinal obstruction, pain and bleeding for GI NET, and asthma, chronic obstructive pulmonary disease and pneumonia for lung NET. More than 70% of patients with NET have nonfunctional tumors. At the time of diagnosis, 5%-44% (depending on site of tumor origin) of patients with NET in the GI tract and 28% of patients with lung NET have advanced disease, meaning the cancer has spread to other areas of the body, making it difficult to treat. Progression, or the continued growth or spread of the tumor, is typically associated with poor outcomes.

Novartis announces FDA approval of Afinitor for progressive, nonfunctional neuroendocrine tumors of GI

New drug shows promise against Huntington's disease

A drug that would be the first to target the cause of Huntington's disease (HD) is effective and safe when tested in mice and monkeys, according to data released today that will be presented at the American Academy of Neurology's 68th Annual Meeting in Vancouver, Canada, April 15 to 21, 2016. A study to test the drug in humans has begun.

Huntington's disease is a rare, hereditary disease that causes uncontrolled movements, loss of intellectual abilities, emotional problems and eventually death. The disease is passed from parent to child through a mutation in the huntingtin gene. The mutation results in the production of a disease-causing huntingtin protein. Each child has a 50/50 chance of inheriting the gene mutation. Everyone who inherits the mutated gene will eventually develop the disease.

The new drug, called IONIS-HTTRx, is an antisense drug that acts as a "gene silencer" to inhibit the production of huntingtin protein in people with Huntington's disease.

"It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease," said clinical study principal investigator Blair R. Leavitt, MD, of the University of British Columbia in Vancouver. "Right now we only have treatments that work on the symptoms of the disease." Leavitt notes the drug is still years away from being used in human clinical practice.

Earlier studies in mouse models of Huntington's disease showed that treatment with antisense drugs delays disease progression and results in sustained reversal of the disease phenotype. In YAC128 mice, a transgenic model of HD, motor deficits improved within one month of initiating antisense treatment and were restored to normal at two months after treatment termination. Motor skills of antisense-treated BACHD mice, another transgenic model of HD, improved eight weeks after initiation of treatment and persisted for at least nine months after treatment termination. In monkeys, dose-dependent reductions in HTT mRNA and Htt protein throughout the central nervous system were observed after intrathecal administration of an antisense drug. Reduction of cortical huntingtin levels by 50 percent was readily achieved in monkeys and correlated with 15 to 20 percent reduction in the caudate. In further tests in rodents and monkeys, IONIS-HTTRx was found to be well-tolerated without any dose-limiting side effects.

New drug shows promise against Huntington's disease

PCSK9-inhibitor drugs: A game-changer for individuals with extremely high cholesterol levels

A 59-year-old heart patient with dangerously high levels of cholesterol that could not be adequately reduced by statin drugs now has near-normal cholesterol levels, thanks to a new class of drugs that grew out of work done by UT Southwestern Medical Center researchers.

Two of these drugs, in a category known as PCSK9 inhibitors, were approved by the Food and Drug Administration last summer for use by some individuals with extremely high cholesterol levels.

"If you take the core patients who are at highest risk, it makes you appreciate how important this drug class is," said Dr. Amit Khera, Director of the Preventive Cardiology Program and Associate Professor of Internal Medicine at UT Southwestern.

Frank Brown of Dallas, grandfather of six and the owner of Frank's Wrecker Service in Dallas, has familial hypercholesterolemia, an inherited condition that causes high levels of cholesterol, especially low-density lipoprotein (LDL) cholesterol or "bad cholesterol." High levels of LDL cholesterol are strongly associated with heart disease.

Mr. Brown, with a history of two heart attacks, had been aggressively treated with multiple drugs to reduce his cholesterol levels, but they remained stubbornly high.

"When I first met Mr. Brown, he had a strong family history of heart disease, he had a cholesterol level that was ridiculously high with an LDL of 384, and he was having chest pains," said Dr. Amit Khera, who is Mr. Brown's cardiologist.

Dr. Khera, who holds the Dallas Heart Ball Chair in Hypertension and Heart Disease at UT Southwestern, was treating Mr. Brown with three cholesterol-lowering medications: a statin, which is a class of drugs that works by blocking a substance the body needs to make cholesterol; ezetimibe, a drug that blocks absorption of cholesterol in the intestine; and colesevelam, which sequesters bile acids. Even with this trio of medicines, Mr. Brown's LDL cholesterol level hovered around 200.

PCSK9-inhibitor drugs: A game-changer for individuals with extremely high cholesterol levels

Steroid May Be Safe, Effective Gout Treatment, Study Finds

A steroid pill may be as good as a nonsteroidal anti-inflammatory drug (NSAID) for treating painful gout, new research suggests.

Researchers who compared the steroid prednisolone with the arthritis medication indomethacin found both drugs offered a similar degree of pain reduction. And while indomethacin (Indocin) appeared to cause more minor side effects, neither treatment prompted serious complications, the researchers said.

        (Prednisole) (Indomethacin)

Smaller investigations have pointed in the same direction, said study lead author Dr. Timothy Rainer, a professor of emergency medicine at Cardiff University in Wales. But because the new findings are the product of a "larger and better-designed" effort, Rainer said steroid pills may gain standing among gout experts who usually stick with NSAIDs as their first-line treatment.

The bottom line is that there are choices, said Dr. Philip Mease, a rheumatologist with the Swedish Medical Center in Seattle.

"That is the key message - that there are options," said Mease, who wasn't involved in the study. "Sometimes ER docs don't think about giving a tapering dose of prednisone, but it can be very effective at helping with gout, which can be damn painful."

Steroid May Be Safe, Effective Gout Treatment, Study Finds 

New molecule has potential to prolong life of cystic fibrosis patients

New molecule has potential to prolong life of cystic fibrosis patients: Scientists at Queen's University Belfast have discovered a new molecule which has the potential to prolong the life of individuals with cystic fibrosis (CF).

Pfizer Receives Expanded FDA Approval For Ibrance (palbociclib) In HR , HER2- Metastatic Breast Cancer

In continuation of my updates on palbociclib

Pfizer Inc. (NYSE:PFE) announced that the U.S. Food and Drug Administration (FDA) has approved a new indication expanding the use of Ibrance (palbociclib) 125mg capsules, Pfizer’s metastatic breast cancer therapy. Now Ibrance also is approved for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy.1 Pfizer’s supplemental New Drug Application (sNDA) for Ibrance was reviewed and approved under the FDA’s Breakthrough Therapy designation and Priority Review programs based on results from the Phase 3 PALOMA-3 trial in pre-, peri- and post-menopausal women with HR+, HER2- metastatic breast cancer whose disease progressed on or after prior endocrine therapy in the adjuvant or metastatic setting.

Ibrance first was approved in February 2015 and also is indicated for the treatment of HR+, HER2- advanced or metastatic breast cancer in combination with letrozole as initial endocrine-based therapy in postmenopausal women.1 The indication in combination with letrozole is approved under accelerated approval based on progression-free survival (PFS). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.1 The confirmatory Phase 3 trial, PALOMA-2, is fully enrolled.

Ibrance is the first and only cyclin-dependent kinase 4/6 (CDK 4/6) inhibitor approved by the FDA.

Pfizer Receives Expanded FDA Approval For Ibrance (palbociclib) In HR , HER2- Metastatic Breast Cancer

FDA Approves Briviact (brivaracetam) to Treat Partial Onset Seizures

The U.S. Food and Drug Administration yesterday approved Briviact (brivaracetam) as an add-on treatment to other medications to treat partial onset seizures in patients age 16 years and older with epilepsy.

Epilepsy is a brain disorder that causes people to have recurring seizures. A seizure is an episode, usually of relatively short duration, of abnormal brain activity. Seizures can cause a variety of symptoms, including uncontrolled movements or spasms, abnormal thinking and behavior, and abnormal sensations. Muscle spasms can be violent, and loss of consciousness can occur. Seizures occur when clusters of nerve cells (neurons) in the brain undergo uncontrolled activation. A partial onset seizure begins in a limited area of the brain.

“Patients can have different responses to the various seizure medicines that are available,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “With the approval of Briviact, I am pleased that patients with epilepsy have a new treatment option.”

FDA Approves Briviact (brivaracetam) to Treat Partial Onset Seizures

Pfizer Receives Expanded FDA Approval For Ibrance (palbociclib) In HR , HER2- Metastatic Breast Cancer

In continuation of my updates on palbociclib

Pfizer Inc. (NYSE:PFE) announced that the U.S. Food and Drug Administration (FDA) has approved a new indication expanding the use of Ibrance (palbociclib) 125mg capsules, Pfizer’s metastatic breast cancer therapy. Now Ibrance also is approved for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy.1 Pfizer’s supplemental New Drug Application (sNDA) for Ibrance was reviewed and approved under the FDA’s Breakthrough Therapy designation and Priority Review programs based on results from the Phase 3 PALOMA-3 trial in pre-, peri- and post-menopausal women with HR+, HER2- metastatic breast cancer whose disease progressed on or after prior endocrine therapy in the adjuvant or metastatic setting.

Ibrance first was approved in February 2015 and also is indicated for the treatment of HR+, HER2- advanced or metastatic breast cancer in combination with letrozole as initial endocrine-based therapy in postmenopausal women.1 The indication in combination with letrozole is approved under accelerated approval based on progression-free survival (PFS). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.1 The confirmatory Phase 3 trial, PALOMA-2, is fully enrolled.

Ibrance is the first and only cyclin-dependent kinase 4/6 (CDK 4/6) inhibitor approved by the FDA.

Pfizer Receives Expanded FDA Approval For Ibrance (palbociclib) In HR , HER2- Metastatic Breast Cancer

FDA-approved Alzheimer's medications may help people quit smoking

Despite several safe drug therapies available to help smokers quit, three-quarters report relapsing within six months of a quit attempt. University of Pennsylvania researchers Rebecca Ashare and Heath Schmidt saw potential for a permanent cessation solution in a class of FDA-approved medications used to improve cognitive impairments from Alzheimer's disease.

In a study consisting of a rat trial and a human trial, Ashare and Schmidt studied the effects of two acetylcholinesterase inhibitors, or AChEIs, called galantamine and donepezil on overall nicotine intake. The rat component showed that pretreating the rodents with an AChEI decreased their nicotine consumption. Consistent with these effects, clinical trial participants taking the AChEI, not the placebo, smoked 2.3 fewer cigarettes daily, a 12 percent decrease, and noted feeling less satisfied with the cigarettes they did smoke.

 (Galantamine )  (Donepezil) 

"We're very interested in screening potential efficacy of anti-addiction medications in our models," said Schmidt, a professor in Penn's School of Nursing and Perelman School of Medicine. "For this study, we looked at potential smoking-cessation medications."

The research itself took a translational approach, what Ashare, a professor in Penn Medicine's psychiatry department, calls bi-directional. In other words, the preclinical data informed the clinical study and vice versa.

At Penn's Center for Interdisciplinary Research on Nicotine Addiction, work on smoking cessation has been ongoing since 2001. Specifically, research from Caryn Lerman, CIRNA's director and the Mary W. Calkins Professor in Psychiatry, concluded that ...

FDA-approved Alzheimer's medications may help people quit smoking