Wednesday, June 12, 2019

FDA Approves Kalydeco (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants with CF as Early as Six Months of Age

In continuation of my update on Kalydeco (ivacaftor) 
Ivacaftor.svg
Vertex Pharmaceuticals Incorporated  announced the U.S. Food and Drug Administration (FDA) approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages six months to less than 12 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data. Kalydeco is already approved in the U.S., Canada and EU for the treatment of CF in patients ages 12 months and older.
“Today’s approval for Kalydeco allows physicians to begin treating the underlying cause of CF in eligible infants as young as six months of age for the first time, with the potential to modify the course of the disease,” said Margaret Rosenfeld, M.D., MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.
This FDA approval is based on data from a 24-week Phase 3 open-label safety cohort (ARRIVAL) of 11 children with CF aged six months to less than 12 months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). The study demonstrated a safety profile similar to that observed in previous Phase 3 studies of older children and adults; most adverse events were mild or moderate in severity, and no patient discontinued therapy due to adverse events. The most common adverse events (≥30%) were cough (64%), nasal congestion (36%) and rhinorrhea (36%). Three serious adverse events, all considered unrelated to study treatment by the investigators, were observed in three patients.
Mean baseline sweat chloride for the children in this cohort was 101.5 mmol/L (n=11). Following 24 weeks of treatment with Kalydeco, the mean sweat chloride level was 43.1 mmol/L (n=6). In the six subjects with paired sweat chloride samples at baseline and week 24, there was a mean absolute change of -58.6 mmol/L (95% CI; -75.9, -41.3).
Results of this study were presented at the 32nd Annual North American Cystic Fibrosis Conference in October 2018.
“The manifestations of CF are often present at birth, which underscores our relentless commitment to reach the youngest CF patients possible in our clinical trials,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “As an important outcome of these efforts, we are now able to treat infants with cystic fibrosis as early as six months of age with Kalydeco.”
Kalydeco was first approved in 2012 in the U.S. and is now available in more than 40 countries with more than 5,000 patients on therapy. For more information on Kalydeco, prescribing information, or patient assistance programs, visit Kalydeco.com or VertexGPS.com.

https://en.wikipedia.org/wiki/Ivacaftor


Tuesday, June 11, 2019

Veggies, Fruits and Grains Keep Your Heart Pumping

Image result for vegetables fruits and grains

  



As if you needed any more proof that fruits, vegetables and whole grains are good for you, a new study finds they may cut your chances of heart As if you needed any more proof that fruits, vegetables and whole grains are good for you, a new study finds they may cut your chances of heart failure by 41%.

Conversely, the so-called Southern diet, which focuses on meats, fried and processed foods and lots of sweet tea, was tied to a 72% increased risk of heart failure.

"Eat more plants, limit red and processed meat," said lead researcher Dr. Kyla Lara, a cardiology fellow at the Mayo Clinic in Rochester, Minn.

Lara cautioned that this study cannot prove different diets cause or prevent heart failure, only that they are linked.

Image result for Veggies, Fruits and GrainsNearly 6 million American adults suffer from heart failure, and that number is expected to rise with the aging population. The condition occurs when the heart does not pump blood sufficiently to meet the body's needs.

Steps to prevent heart failure include not smoking, keeping blood pressure under control, maintaining a healthy weight and eating healthy foods.

Getting people to eat healthier requires that they be educated about the benefits of plant-based diets and have access to low-cost healthy foods, Lara said.

"Animal meat is not necessary for a nutritious diet, in terms of health promotion and quality of life," she said. "Now is the time to get on board with a plant-based diet -- it's going to be the future of health."

In the study, Lara and her colleagues collected data on more than 16,000 men and women, 45 and older, who took part in a large U.S. stroke study. None of the participants had heart disease at the start of the study. Participants completed a questionnaire that asked them about their diet.

The diets were classified into five types:

Convenience, which was heavy on meats, pasta, Mexican food, pizza and fast food.
Plant-based, which included vegetables, fruits, beans and fish.
Sweets and fats, which was heavy on desserts, bread, sweet breakfast foods, chocolate and other sugars.
Southern, which was heavy on fried foods, processed meats, eggs, added fats, and sugar-sweetened drinks.
Alcohol and salads, which was heavy on wine, liquor, beer, leafy greens and salad dressing.
After nearly nine years of follow-up, 363 participants developed heart failure.

The benefit of the plant-based diet was significant, but after taking into account factors such as weight, waist size, high blood pressure and high cholesterol, the negative effect of the Southern diet was no longer statistically significant, Lara said.

It might be that the increased risk for heart failure in this group was due to obesity and excess belly fat or other factors, she said.

None of the other diets showed a statistically significant association with heart failure, and no association was seen between any diet and the type of heart failure people developed, the researchers noted.

The findings were published April 22 in the Journal of the American College of Cardiology.

"Our lifestyle, such as what we eat, if we are physically active and smoking or vaping, can contribute significantly to a poorly functioning heart, which in turn affects our quality of life and ultimately how soon we die," said Samantha Heller, a senior clinical nutritionist at New York University Medical Center in New York City.
Plant-based diets have been shown to reduce the risk of cardiovascular disease, cognitive [thinking] decline, type 2 diabetes, several cancers, depression and obesity," said Heller, who wasn't involved with the study.
For optimal health, people need to cut back on fried foods, cheese, fast food and junk foods, and processed and red meats, she said.
"It is sad and frustrating when I see patients who could quite literally save their lives by making healthier choices, but instead opt for a burger and fries," Heller said.
A cheeseburger, fries and milkshake meal sounds innocent enough, but it can add up to more than 2,600 calories, 65 grams of saturated fat and 3,400 milligrams of salt, she said.
Heller advises people to include a minimum of one high-fiber food to every meal. "Dietary fiber is only found in plant foods, such as spinach, oranges, quinoa and lentils."
Also try having meatless dinners two or three nights a week, like a grilled vegetable and black bean burrito, pasta primavera or an edamame and fresh vegetable stir fry, she suggested.failure by 41%.
Conversely, the so-called Southern diet, which focuses on meats, fried and processed foods and lots of sweet tea, was tied to a 72% increased risk of heart failure.
"Eat more plants, limit red and processed meat," said lead researcher Dr. Kyla Lara, a cardiology fellow at the Mayo Clinic in Rochester, Minn.
Lara cautioned that this study cannot prove different diets cause or prevent heart failure, only that they are linked.
Nearly 6 million American adults suffer from heart failure, and that number is expected to rise with the aging population. The condition occurs when the heart does not pump blood sufficiently to meet the body's needs.
Steps to prevent heart failure include not smoking, keeping blood pressure under control, maintaining a healthy weight and eating healthy foods.
Getting people to eat healthier requires that they be educated about the benefits of plant-based diets and have access to low-cost healthy foods, Lara said.
"Animal meat is not necessary for a nutritious diet, in terms of health promotion and quality of life," she said. "Now is the time to get on board with a plant-based diet -- it's going to be the future of health."
In the study, Lara and her colleagues collected data on more than 16,000 men and women, 45 and older, who took part in a large U.S. stroke study. None of the participants had heart disease at the start of the study. Participants completed a questionnaire that asked them about their diet.
The diets were classified into five types:


  • Convenience, which was heavy on meats, pasta, Mexican food, pizza and fast food.
  • Plant-based, which included vegetables, fruits, beans and fish.
  • Sweets and fats, which was heavy on desserts, bread, sweet breakfast foods, chocolate and other sugars.
  • Southern, which was heavy on fried foods, processed meats, eggs, added fats, and sugar-sweetened drinks.
  • Alcohol and salads, which was heavy on wine, liquor, beer, leafy greens and salad dressing.


After nearly nine years of follow-up, 363 participants developed heart failure.
The benefit of the plant-based diet was significant, but after taking into account factors such as weight, waist size, high blood pressure and high cholesterol, the negative effect of the Southern diet was no longer statistically significant, Lara said.
It might be that the increased risk for heart failure in this group was due to obesity and excess belly fat or other factors, she said.
None of the other diets showed a statistically significant association with heart failure, and no association was seen between any diet and the type of heart failure people developed, the researchers noted.
The findings were published April 22 in the Journal of the American College of Cardiology.
"Our lifestyle, such as what we eat, if we are physically active and smoking or vaping, can contribute significantly to a poorly functioning heart, which in turn affects our quality of life and ultimately how soon we die," said Samantha Heller, a senior clinical nutritionist at New York University Medical Center in New York City.
Plant-based diets have been shown to reduce the risk of cardiovascular disease, cognitive [thinking] decline, type 2 diabetes, several cancers, depression and obesity," said Heller, who wasn't involved with the study.
For optimal health, people need to cut back on fried foods, cheese, fast food and junk foods, and processed and red meats, she said.
"It is sad and frustrating when I see patients who could quite literally save their lives by making healthier choices, but instead opt for a burger and fries," Heller said.
A cheeseburger, fries and milkshake meal sounds innocent enough, but it can add up to more than 2,600 calories, 65 grams of saturated fat and 3,400 milligrams of salt, she said.
Heller advises people to include a minimum of one high-fiber food to every meal. "Dietary fiber is only found in plant foods, such as spinach, oranges, quinoa and lentils."
Also try having meatless dinners two or three nights a week, like a grilled vegetable and black bean burrito, pasta primavera or an edamame and fresh vegetable stir fry, she suggested.
Veggies, Fruits and Grains Keep Your Heart Pumping 

Saturday, June 8, 2019

FDA Approves Mavenclad (cladribine) Tablets for Multiple Sclerosis


In continuation of my update on cladribine

Cladribine.svg

The U.S. Food and Drug Administration,    approved Mavenclad (cladribine) tablets to treat relapsing forms of multiple sclerosis (MS) in adults, to include relapsing-remitting disease and active secondary progressive disease. Mavenclad is not recommended for MS patients with clinically isolated syndrome. Because of its safety profile, the use of Mavenclad is generally recommended for patients who have had an inadequate response to, or are unable to tolerate, an alternate drug indicated for the treatment of MS.
“We are committed to supporting the development of safe and effective treatments for patients with multiple sclerosis,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “The approval of Mavenclad represents an additional option for patients who have tried another treatment without success.”
MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communications between the brain and other parts of the body. Most people experience their first symptoms of MS between the ages of 20 and 40. MS is among the most common causes of neurological disability in young adults and occurs more frequently in women than in men.
For most people, MS starts with a relapsing-remitting course, in which episodes of worsening function (relapses) are followed by recovery periods (remissions). These remissions may not be complete and may leave patients with some degree of residual disability. Many, but not all, patients with MS experience some degree of persistent disability that gradually worsens over time. In some patients, disability may progress independent of relapses, a process termed secondary progressive multiple sclerosis (SPMS). In the first few years of this process, many patients continue to experience relapses, a phase of the disease described as active SPMS. Active SPMS is one of the relapsing forms of MS, and drugs approved for the treatment of relapsing forms of MS can be used to treat active SPMS.
The efficacy of Mavenclad was shown in a clinical trial in 1,326 patients with relapsing forms of MS who had least one relapse in the previous 12 months. Mavenclad significantly decreased the number of relapses experienced by these patients compared to placebo. Mavenclad also reduced the progression of disability compared to placebo.
Mavenclad must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks. Mavenclad has a Boxed Warning for an increased risk of malignancy and fetal harm. Mavenclad is not to be used in patients with current malignancy. In patients with prior malignancy or with increased risk of malignancy, health care professionals should evaluate the benefits and risks of the use of Mavenclad on an individual patient basis. Health care professionals should follow standard cancer screening guidelines in patients treated with Mavenclad. The drug should not be used in pregnant women and in women and men of reproductive potential who do not plan to use effective contraception during treatment and for six months after the course of therapy because of the potential for fetal harm. Mavenclad should be stopped if the patient becomes pregnant.
Other warnings include the risk of decreased lymphocyte (white blood cell) counts; lymphocyte counts should be monitored before, during and after treatment. Mavenclad may increase the risk of infections; health care professionals should screen patients for infections and treatment with Mavenclad should be delayed if necessary. Mavenclad may cause hematologic toxicity and bone marrow suppression so health care professionals should measure a patient’s complete blood counts before, during and after therapy. The drug has been associated with graft-versus-host-disease following blood transfusions with non-irradiated blood. Mavenclad may cause liver injury and treatment should be interrupted or discontinued, as appropriate, if clinically significant liver injury is suspected.
The most common adverse reactions reported by patients receiving Mavenclad in the clinical trials include upper respiratory tract infections, headache and decreased lymphocyte counts. 

Ref : https://en.wikipedia.org/wiki/Cladribine



Friday, June 7, 2019

FDA Approves Avaclyr (acyclovir ophthalmic ointment) for the Treatment of Herpetic Keratitis


  Image

 The US Food and Drug Administration (FDA) has approved Fera Pharmaceuticals' New Drug Application (NDA) for Avaclyr (acyclovir ophthalmic ointment) 3% for the treatment of herpetic keratitis. Orphan drug exclusivity was also granted, providing seven years of marketing exclusivity for the product.

“Avaclyr approval is a major milestone for the company,” noted Frank DellaFera, Fera’s founder and President. “Working closely with the eye care community we were able to identify a need for additional options to treat herpetic keratitis. Acyclovir is the gold standard treatment in herpes virus infections so it made sense to provide this therapy in an ophthalmic dosage form1. Fera is thankful to FDA for approving Avaclyr, making it available to physicians and patients in the US.”
Fera launch plans include finalizing its selection of a commercialization partner to provide physicians and patients access to this important therapy.

About Acute Herpetic Keratitis

According to the Center for Disease Control and Prevention (CDC), HSV (Herpes Simplex Virus) keratitis is an infection of the cornea—the clear dome that covers the colored part of the eye—that is caused by HSV. The infection usually heals without damaging the eye, but more severe infections can lead to scarring of the cornea or blindness. HSV keratitis is a major cause of blindness worldwide2-3.

About Avaclyr

Avaclyr (acyclovir ophthalmic ointment) 3%, is a herpes simplex virus nucleoside analog DNA polymerase inhibitor, is indicated in the treatment of acute herpetic keratitis (dendritic ulcers) in patients with herpes simplex (HSV-1 and HSV-2) virus. The most common adverse reactions (2-10%) reported in patients were eye pain (stinging), punctate keratitis and follicular conjunctivitis

https://www.drugs.com/newdrugs/fda-approves-avaclyr-acyclovir-ophthalmic-ointment-herpetic-keratitis-4939.html?utm_source=ddc&utm_medium=rss&utm_campaign=FDA+Approves+Avaclyr+(acyclovir+ophthalmic+ointment)+for+the+Treatment+of+Herpetic+Keratitis


Thursday, June 6, 2019

FDA Approves Duaklir Pressair

In continuation of my update on formoterol 
Circassia Pharmaceuticals plc (“Circassia” or “the Company”; LSE: CIR), a specialty pharmaceutical company focused on respiratory disease,  announced the US Food and Drug Administration (FDA) has approved Duaklir for the maintenance treatment of chronic obstructive pulmonary disease (COPD). Duaklir is a fixed-dose combination of the long-acting muscarinic antagonist (LAMA) aclidinium bromide (400 mcg) and long-acting beta-agonist (LABA) formoterol fumarate (12 mcg) administered twice-daily via the breath-actuated inhaler Pressair®.  Circassia is on track to launch Duaklir in the United States in the second half of 2019 via its dedicated COPD sales force.
The Duaklir approval is based on a broad clinical database, including data from three phase III studies, ACLIFORM, AUGMENT and AMPLIFY. The label also includes clinical data from the phase IV ASCENT study, which shows aclidinium therapy is effective at reducing COPD exacerbations.  As a result, Duaklir® is the only twice-daily LAMA / LABA in the United States with COPD exacerbation data included in its prescribing information.
Steve Harris, Circassia’s Chief Executive, said: “We are delighted with the FDA approval of Duaklir, which we believe will provide a valuable treatment option for the significant number of patients with COPD in the United States. The addition of Duaklir to our portfolio further strengthens our range of marketed respiratory products and we look forward to launching it in the US in the coming months alongside our aclidinium monotherapy, Tudorza, as part of the significant LAMA / LABA market that is predicted to grow rapidly over the coming years.”
Michael Asmus, Circassia’s Vice President, US Medical Affairs, said: “With guidelines recommending combined LAMA and LABA therapy for a number of COPD patient groups, we believe Duaklir will make an important contribution to the treatment of this debilitating disease. Dukalir’s approval is based on a broad clinical database, including data demonstrating a reduction in the risk of COPD exacerbations driven by its aclidinium component, and we look forward to making this new therapeutic option available to patients across the United States.”
Aclidinium bromide.svg Aclidinium bromide
 Thumb Formoterol

https://en.wikipedia.org/wiki/Aclidinium_bromide
https://www.drugbank.ca/drugs/DB00983



Wednesday, June 5, 2019

FDA Approves Ibrance (palbociclib) for the Treatment of Men with HR+, HER2- Metastatic Breast Cancer

In continuation of my update on palbociclib

Palbociclib.svg

Pfizer  announced that the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) to expand the indications for Ibrance (palbociclib) in combination with an aromatase inhibitor or fulvestrant to include men with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer. The approval is based on data from electronic health records and postmarketing reports of the real-world use of Ibrance in male patients sourced from three databases: IQVIA Insurance database, Flatiron Health Breast Cancer database and the Pfizer global safety database.
“With this approval, we are now able to offer Ibrance to the underserved male breast cancer community and provide more patients with HR+, HER2- metastatic breast cancer the opportunity to access an innovative medicine,” said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology, Pfizer Global Product Development. “We appreciate that our partnership with the FDA has allowed us to take a significant step forward in the use of real-world data to bring medicines to patients who are most in need.”
Ibrance is now approved for adult patients with HR+, HER2- advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine based therapy in postmenopausal women or in men; or with fulvestrant in patients with disease progression following endocrine therapy. With today’s approval, Ibrance is the first and only CDK 4/6 inhibitor indicated in combination with an aromatase inhibitor for the first-line treatment of men with HR+, HER2- metastatic breast cancer in the U.S.
“Men with breast cancer have limited treatment options, making access to medicines such as Ibrance critically important,” said Bret Miller, founder of the Male Breast Cancer Coalition. “We applaud the use of real-world data, a new approach to drug review, to make Ibrance available to certain men with metastatic breast cancer and help address an unmet need for these patients.”
Real-world data is playing an increasingly important role in expanding the use of already approved innovative medicines.1 Due to the rarity of breast cancer in males, fewer clinical trials are conducted that include men resulting in fewer approved treatment options. In the U.S. in 2019, it is estimated that there will be 2,670 new cases of invasive breast cancer and about 500 deaths from metastatic breast cancer in males.2 The 21st Century Cures Act, enacted in 2016, was created to help accelerate medical product development, allowing new innovations and advances to become available to patients who need them faster and more efficiently.3 This law places additional focus on the use of real-world data to support regulatory decision-making.4
Detailed analysis of the use of Ibrance in men with HR+, HER2- advanced or metastatic breast cancer will be presented at an upcoming medical meeting. Based on limited data from postmarketing reports and electronic health records, the safety profile for men treated with Ibrance is consistent with the safety profile in women treated with Ibrance.5
About Ibrance (palbociclib) 125 mg capsules
Ibrance is an oral inhibitor of CDKs 4 and 6,6 which are key regulators of the cell cycle that trigger cellular progression.7,8 In the U.S., Ibrance is indicated for the treatment of adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine based therapy in postmenopausal women or in men; or with fulvestrant in patients with disease progression following endocrine therapy.
Ibrance currently is approved in more than 90 countries and has been prescribed to more than 200,000 patients globally.