The approval follows positive results from the TATE trial.
A new 10 mg dose will be available to accommodate children who weigh less than 35 kg.
The FDA approved the use of Fasenra for the treatment of severe asthma in children aged 6 to 11 years, AstraZeneca announced in a press release.
Fasenra (benralizumab, AstraZeneca) was first approved in 2017 for additional maintenance treatment of severe eosinophilic asthma in patients aged at least 12 years. The approval for treatment of children aged 6 to 11 years was supported by evidence from the TATE study as well as trials in adult and adolescent populations, according to the release.
Fasenra is administered by subcutaneous injection every 4 weeks for the first 3 doses then once every 8 weeks.
In the TATE study, an open-label, multinational, phase 3 study of benralizumab in children aged 6 to 11 years with severe eosinophilic asthma, results were consistent with previous FASENRA trials in assessing maximum serum concentration, clearance, half-life and blood eosinophil count as well as safety and tolerability.
“These results add to Fasenra’s growing body of evidence, further characterizing its well-established safety profile, and represents a crucial step in offering children with [severe eosinophilic asthma] a wider choice of treatment in the future,” Donna Carstens, MD, senior director, medical affairs, Fasenra, AstraZeneca, told Healio.
Donna Carstens, MD
The recommended dose of Fasenra is 30 mg for patients aged 6 years or older who weigh 35 kg or more. A new 10 mg dose will be available for patients aged 6 to 11 years who weigh less than 35 kg. Fasenra is administered subcutaneously by injection every 4 weeks for the first three doses and then once every 8 weeks.
“Additional treatment options for children living with severe asthma are needed to help address the unmet need in this patient population and to reduce the burden of disease for the broader asthma community,” Carstens said. “The FDA’s decision to expand the approved use of Fasenra for the add-on maintenance treatment of patients aged 6 to 11 years old addresses this unmet need and represents a significant step in offering children with [severe eosinophilic asthma] a wider choice of treatment in the future.”
According to Carstens, the company plans to continue to evaluate Fasenra’s potential to treat different age groups across disease states.
“AstraZeneca’s mission is to revolutionize asthma care for all patient age groups,” she said.
The FDA will allow yogurt brands to make limited claims that the food can help reduce the risk of type 2 diabetes.
Yogurts will now be able to make qualified claims that they reduce type 2 diabetes risk.
The FDA states that there is scientific evidence for this claim, although it is limited.
It can be claimed that at least 2 cups (3 servings) weekly may provide this benefit.
However, nutritionists advise being cautious about eating yogurts with added sugar.
Other ways to reduce risk include lifestyle changes like diet and exercise.
On March 1, 2024, the Food and Drug Administration (FDA) released a statementTrusted Source saying that it has no intention to object to companies making certain qualified health claims related to how eating yogurt might reduce type 2 diabetes risk.
They will be allowed to do this, the agency states, as long as the claims are not worded in a misleading manner.
Per the FDA, a qualified health claim has scientific evidence behind it but does not meet the more rigorous requirements for an authorized health claim.
This announcement came in response to a 2018 petition from Danone North America, the maker of yogurt brands including Dannon, Activia, and Horizon Organics.
The company’s petition noted that the health effects appear to be due to yogurt as a food rather than any particular ingredient in yogurt. This means the claim is independent of fat or sugar content.
The qualified health claims that the FDA will be allowing include the following:
“Eating yogurt regularly, at least 2 cups (3 servings) per week, may reduce the risk of type 2 diabetes. FDA has concluded that there is limited information supporting this claim.”
“Eating yogurt regularly, at least 2 cups (3 servings) per week, may reduce the risk of type 2 diabetes according to limited scientific evidence.”
Miguel Freitas, PhD, Vice President of Health & Scientific Affairs at Danone North America, told Healthline that the claim was initially inspired by a studyTrusted Source out of the Harvard School of Public Health that found that higher yogurt intake was linked with a reduced risk for type 2 diabetes even though other forms of dairy were not.
Altogether, 32 studies, including 300,000 participants, were cited in Danone’s petition.
“Much of the research that exists in this area and was analyzed in review of this claim includes a variety of high quality prospective cohort studies, also called observational studies,” he added, “the majority of which provide direct or suggestive evidence that yogurt consumption is associated with a lower risk of developing type 2 diabetes when measured over time.”
Freitas went on to say that this type of data allows researchers to study large groups of people over longer periods of time in order to identify any possible associations between their habits and health outcomes such as the development of diabetes.
“While some consider randomized controlled trials, or studies that assign subjects to either a placebo or intervention group, to be stronger, they are challenging to apply to nutrition research such as that around yogurt and diabetes because they often lack suitable controls, don’t continue for a long enough period of time or are conducted in populations that aren’t representative of the average American,” he explained.
Freitas added that there have been some controlled studies looking at yogurt consumption and diabetes prevention. However, they have not been structured in a way that allows for yogurt to be compared to a suitable control.
Catherine Rall, Registered Dietitian at Happy V, said it really comes down to the fact that, while there is a correlation between yogurt consumption and not developing type 2 diabetes, that’s not the same as causation.
“While we can speculate that yogurt’s higher protein content gives it a lower glycemic index than higher-carb alternatives and that the probiotics in yogurt may play a role here, none of this can be conclusively proven,” she stated.
Regarding the sugar content of many yogurts, Freitas said, “… it’s important to note that much of the existing research on yogurt and type 2 diabetes does not distinguish between yogurts of varying sugar or fat content when illustrating a potential benefit, suggesting yogurt of any type could have a protective effect.”
Freitas further added that The Dietary Guidelines for Americans 2020-2025 does recommendTrusted Source limited added sugars to less than 10% of total calories while also stating that small amounts can be an acceptable part of an overall healthy diet, especially when it encourages people to select more nutritious foods like yogurt and allows people more flexibility in their diets.
However, Dan Gallagher, a Registered Dietitian with Aegle Nutrition, says not all yogurt is going to be created equal.
“Many yogurts end up being unhealthy because of the added sugars and preservatives added to make them more palatable,” he remarked.
“You’re going to be better off sticking with Greek yogurt or Skyr, which both have higher protein levels and very little added sugars or sweeteners,” said Gallagher.
Jenny Dobrynina, a Certified Nutritionist and Sports Dietetics Expert at Recipe from Chef, suggested several steps you can take outside of consuming more yogurt in order to reduce your risk for type 2 diabetes.
First, it’s important to eat a healthy diet. “I recommend a balanced diet rich in whole grains, fruits, vegetables, and lean proteins,” she said.
Additionally, Dobrynina suggests getting regular exercise in order to maintain healthy blood sugar levels.
Both diet and exercise can help with weight management, which she states can also reduce your risk for type 2 diabetes.
Dobrynina further suggests avoiding excess sugar and refined carbohydrates.
Dobrynina also advises against smoking and excessive alcohol use. “Both these vices contribute to increased chances of type 2 diabetes,” she said.
Finally, she says it’s important to manage stress. “Reduce your stress because chronic stress is one of the premier reasons impacting blood sugar levels,” she noted.
The FDA has announced that it will be allowing yogurt manufacturers to make qualified claims about yogurt’s ability to prevent type 2 diabetes.
They will be allowed to state that eating at least 2 cups (3 servings) of yogurt weekly may reduce the risk for type 2 diabetes.
However, they must specify that this is based on limited information.
While there is evidence of a link between eating more yogurt and protection against type 2 diabetes, it has not been proven that yogurt prevents type 2 diabetes.
Nutritionists say consumers should be cautious about consuming yogurts with added sugars and preservatives as these ingredients make yogurt less healthy.
Other ways to reduce your risk for type 2 diabetes include eating a nutritious diet, exercising, maintaining a healthy weight, limiting sugar and refined carbohydrates, avoiding smoking or drinking too much, and managing stress.
The FDA has approved Duvyzat for the treatment of Duchenne muscular dystrophy among patients aged 6 years and older, according to a press release.
Duvyzat (givinostat, Italfarmaco) is a histone deacetylase inhibitor that regulates the activity of histone deacetylase in dystrophic muscle to slow down muscle damage. This approval marks the first nonsteroidal drug available to treat patients with all genetic variants of Duchenne muscular dystrophy, according to the FDA.
The FDA approved givinostat for the treatment of Duchenne muscular dystrophy among patients aged 6 years and older.
The approval was based, in part, on data from the randomized, double-blind, placebo-controlled EPIDYS study of 179 boys aged 6 years and older who received givinostat twice daily or placebo alongside glucocorticosteroids. As Healio previously reported, results from the trial showed the drug reduced the decline of muscle function by 40%, as measured by a four stair climb test, after 18 months of treatment, meeting the study’s primary endpoint.
Diarrhea, abdominal pain, a reduction in platelets, nausea/vomiting, increased triglycerides and fever were the most commonly observed adverse events associated with givinostat.
“The FDA’s approval of Duvyzat for [Duchenne muscular dystrophy], based on our robust and successful clinical development program, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with [Duchenne muscular dystrophy],” Paolo Bettica, MD, PhD, chief medical officer at Italfarmaco Group, said in a company press release.“We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval. Our focus now is to make Duvyzat available as a treatment for [Duchenne muscular dystrophy] management in the U.S. as quickly as possible.”
The company said ITF Therapeutics, a new subsidiary based in the United States, will oversee the marketing and distribution of givinostat.
The US Food and Drug Administration (FDA) has approved aprocitentan (Tryvio) to lower blood pressure, in combination with other antihypertensive agents, in adults with treatment-resistant hypertension.
Aprocitentan is the first endothelin receptor antagonist approved for patients with hypertension. The recommended dose is 12.5 mg orally once daily, with or without food.
The efficacy and safety of aprocitentan were demonstrated in the phase 3 PRECISION study of 730 adults with systolic blood pressure ≥ 140 mmHg who were prescribed at least three antihypertensive medications.
Aprocitentan (12.5 mg daily) was well tolerated and statistically superior to placebo in lowering blood pressure at 4 weeks, with a sustained effect at 40 weeks.
Subgroup analyses showed that the blood pressure–lowering effect of aprocitentan appeared to be consistent among subgroups defined by age, sex, race, body mass index, baseline estimated glomerular filtration rate, baseline urine albumin-to-creatinine ratio, and medical history of diabetes, as well as between blood pressure measurement methods.
‘Transformational Progress’
“We have had to wait for over 30 years to see the approval of an oral anti-hypertensive agent that works on a new therapeutic pathway, so Tryvio provides transformational progress in the field of systemic hypertension,” study investigator Michael A. Weber, MD, State University of New York, commented in a news release.
“It is taken as a single daily oral dose that works in combination with whatever other drugs are being prescribed and without drug-drug interactions in patients with the burden of uncontrolled hypertension. Tryvio is easy for physicians to prescribe and easy for patients to use,” said Weber.
Aprocitenan is expected to be available in the second half of this year, the company notes. It will only be available through a Risk Evaluation and Mitigation Strategy (REMS) program owing to the risk for embryo-fetal toxicity.
Prescribers must be certified with the Tryvio REMS by enrolling and completing training. Pharmacies that dispense the drug must also be certified with the program.
Nonalcoholic Fatty Liver Disease may be represented by an increase in one bacteria, in a similar way the microbiome is a biomarker for obesity and end-stage liver fibrosis.
Patients suffering from scarring due to fatty liver disease now have a new treatment option: Rezdiffra, a medication recently approved by the U.S. Food and Drug Administration.
The main ingredient in Rezdiffra is resmetirom, and it is intended to be used in conjunction with diet and exercise to treat non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring. This approval brings a much-needed standard of care treatment for a condition that affects up to 15 million adults in the U.S., according to the American Liver Foundation.
“Previously, patients with NASH who also had notable liver scarring did not have a medication that could directly address their liver damage,” Dr. Nikolay Nikolov, acting director of the Office of Immunology and Inflammation at the FDA, said in a press release. “Today’s approval of Rezdiffra will, for the first time, provide a treatment option for these patients, in addition to diet and exercise.”
How Does Rezdiffra Work?
NASH occurs when fatty deposits accumulate in the liver, even in individuals who consume little to no alcohol. High-risk factors for non-alcoholic fatty liver disease, which can progress into NASH, include diabetes, high cholesterol, high triglycerides, poor diet, metabolic syndrome, polycystic ovary syndrome, sleep apnea, and hypothyroidism.
Inflammation in the liver causes NASH, leading to scarring of the organ’s muscle tissue due to fat deposits and buildup. When scarring occurs, the liver enters a potentially life-threatening condition known as cirrhosis.
Rezdiffra works by reducing the amount of fat that accumulates in the liver in the first place. To ensure the medication’s safety and efficacy, Madrigal Pharmaceuticals, the manufacturer of Rezdiffra, conducted a double-blind, placebo-controlled study involving 888 patients. Participants received either 80 or 100 milligrams of Rezdiffra once daily.
After one year, 26 to 27 percent of participants who received 80 milligrams of Rezdiffra and 24 to 36 percent of those who received 100 milligrams experienced no worsening of liver scarring, compared to only 9 to 13 percent of participants taking the placebo.
Additionally, 23 percent of subjects who received 80 milligrams of Rezdiffra and 24 to 28 percent of those who received 100 milligrams experienced an improvement in liver scarring and no worsening NASH symptoms, compared to 13 to 15 percent of those who received the placebo.
“Demonstration of these changes in a proportion of patients after just one year of treatment is notable, as the disease typically progresses slowly with a majority of patients taking years or even decades to show progression,” according to the FDA’s press release.
During the trial, the medication caused some side effects, including diarrhea and nausea. Rezdiffra can increase a person’s risk of drug-induced liver toxicity and gallbladder-related side effects, the FDA notes.
Not for Everyone
Rezdiffra is not suitable for everyone.
People with decompensated cirrhosis should not take this medication. Additionally, Rezdiffra may not mix well with certain other drugs; people taking particular statins for lowering cholesterol levels can potentially experience significant drug interactions.
Furthermore, anyone whose condition worsens while taking Rezdiffra should stop using it.
Rezdiffra received approval from the FDA through the agency’s accelerated approval process. This process allows for a shortened pathway to approval for drugs or medical devices that treat serious conditions or address an unmet medical need.
Rezdiffra is expected to be available on the U.S. market by April and will be distributed through a limited network of specialty pharmacies, according to Madrigal Pharmaceuticals.
An ingredient once commonly used in citrus-flavored sodas to keep the tangy taste mixed thoroughly through the beverage could finally be banned for good across the US.
The FDA proposed in November to revoke the registration of a modified vegetable oil known as BVO in the wake of recent toxicology studies that make it difficult to support its ongoing use.
“The proposed action is an example of how the agency monitors emerging evidence and, as needed, conducts scientific research to investigate safety related questions, and takes regulatory action when the science does not support the continued safe use of additives in foods,” James Jones, FDA deputy commissioner for human foods, explained when announcing the proposal.
BVO, or brominated vegetable oil, has been used as an emulsifying agent since the 1930s to ensure citrus flavoring agents don’t float to the top of sodas. Sticking a dozen bromine atoms to a triglyceride creates a dense oil that floats evenly throughout water when mixed with less dense fats.
Yet that’s not BVO’s only trick. Animal studies have strongly implied the compound can slowly build up in our fat tissues. With bromine’s potential ability to prevent iodine from doing its all-important work inside the thyroid, health authorities around the world have been suspicious of the emulsifier’s risks for decades.
In fact, BVO is already banned in many countries, including India, Japan, and nations of the European Union, and was outlawed in the state of California in October 2022 with legislation due to take effect in 2027.
Yet the FDA has been slow to convince. In the 1950s, the agency regarded the ingredient as generally recognized as safe (GRAS); an official classification afforded items that have either been appropriately tested or – for ingredients in common use prior to 1958 – don’t appear to be harmful.
That changed the following decade when questions were raised over its possible toxicity, prompting the FDA to overturn its GRAS classification for BVO and temporarily limit its use to relatively small concentrations of no more than 15 parts per million exclusively in citrus-flavored drinks.
Data on the risks posed by even these small amounts of BVO over time hasn’t been easy to collect, relying heavily on long-term studies that re-evaluate health effects in a significantly-sized sample of people. Yet the evidence has been slowly mounting.
It’s taken time, and a number of further studies, but on the back of more recent animal studies based on relative concentrations of BVO humans are likely to ingest, the FDA is finally convinced there is sufficient evidence to ban its use altogether.
“Over the years many beverage makers reformulated their products to replace BVO with an alternative ingredient, and today, few beverages in the US contain BVO,” said Jones.
The ban could be a sign of more things to come, with Jones announcing the agency is reviewing regulations that authorize the use of certain food additives, with a view to automatically prohibit the approval of any food coloring agents found to cause cancer in humans or animals, making for a more nimble bureaucratic process.
A final call on the FDA’s reclassification of BVO still needs to go through a lengthy review process that will take time to complete.
With suitable alternatives to BVO already being used to make citrus drinks around the world taste tangy down to the very last drop, the ingredient isn’t likely to be missed.
The FDA is warning people with diabetes and the public that noninvasive smartwatches or smart rings that do not pierce the skin should not be used to measure blood glucose, according to a press release.
The FDA issued a new safety communication on Feb. 21 to inform people with diabetes that glucose readings taken with smart devices that do not pierce the skin could be inaccurate. Those inaccurate measurements could lead a person to take the wrong dose of insulin or a diabetes medication and precipitate dangerously low glucose levels, which could result in mental confusion, coma or death within hours of the error.
The FDA is warning the public that smartwatches and smart rings that do not pierce the skin should not be used for measuring blood glucose.
The FDA has not authorized, cleared or approved any noninvasive smartwatch or smart ring for the measurement of glucose. While those devices claim to use noninvasive techniques to measure blood glucose, the FDA said the devices do not test blood glucose directly. The agency said standalone smartwatches or smart rings are different from smartwatch applications that display data from an FDA-approved device that measures blood glucose by piercing the skin, such as a continuous glucose monitoring device. The FDA’s safety communication applies to devices manufactured by dozens of companies and sold under multiple brand names.
Consumers, people with diabetes and their caregivers are advised not to purchase or use smartwatches or smart rings that claim to measure glucose levels on their own. People with diabetes are advised to speak with their health care provider about using an appropriate FDA-approved device. Providers are advised to warn patients about the risks of using unauthorized blood glucose measuring devices and to help patients select an FDA-approved device if needed.
The FDA has granted fast track designation to GSK’s bepirovirsen, a triple action investigational antisense oligonucleotide, for the treatment of chronic hepatitis B virus infection, according to a manufacturer release.
Developed jointly with Ionis Pharmaceuticals, bepirovirsen is intended to curb viral DNA replication, reduce the level of hepatitis B surface antigen and stimulate the immune system to boost the likelihood of a durable and sustained response.
The FDA based its decision on data from two phase 2b trials, B-Clear and B-Sure, which assessed the efficacy, safety and response durability of bepirovirsen in patients with chronic HBV.
The B-Clear trial included two patient cohorts consisting of those who received treatment with nucleoside/nucleotide analogues (NAs) and those who did not. Patients were randomly assigned to one of four treatment groups with bepirovirsen:
Bepirovirsen 300 mg with loading doses (LD) for 24 weeks;
Bepirovirsen 300 mg with LD for 12 weeks, then 150 mg for 12 weeks;
Bepirovirsen 300 mg with LD for 12 weeks, then placebo for 12 weeks;
Placebo with LD for 12 weeks, then bepirovirsen 300 mg without LD for 12 weeks.
According to the release, the B-Clear trial demonstrated that patients with low base hepatitis B surface antigen levels “were most likely to benefit from treatment with bepirovirsen.”
Compared with the NAs, bepirovirsen is “the only single agent in phase 3 development that has shown the potential to achieve clinically meaningful functional cure response when combined with oral nucleoside/nucleotide analogues,” the release noted. However, NAs can only hinder the virus itself, but do not lower hepatitis B surface antigen, which is critical for functional cure.
The B-Sure trial is currently investigating longer efficacy and response durability in bepirovirsen, following patients from the B-Clear trial who have stopped NA therapy for an additional 33 months. The B-Sure trial intends to assess “the potential for functional cure in patients who successfully stop all medication and continue to demonstrate no serologic evidence of hepatitis B surface antigen or HBV DNA,” the release stated.
Additionally, a confirmatory phase 3 trial, B-Well, is ongoing, the company noted.
FDA fast track designation is intended to expedite the development and review of drugs that fulfill an unmet need in serious diseases. Benefits of the designation include the potential for more frequent meetings with the FDA during clinical development, eligibility for accelerated approval or priority review, and the ability to submit sections of a biologics license application on a rolling basis.
The FDA approved irinotecan liposomeopens in a new tab or window (Onivyde) in combination with oxaliplatin, leucovorin, and fluorouracil (NALIRIFOX) for the first-line treatment of metastatic pancreatic adenocarcinoma, the agency announced on Tuesday.
Approval was based on resultsopens in a new tab or window from the phase III NAPOLI 3 trialopens in a new tab or window of 770 patients with metastatic pancreatic adenocarcinoma who had not previously received chemotherapy in the metastatic setting. Patients in the study were randomized to receive either NALIRIFOX or gemcitabine plus nab-paclitaxel (Abraxane).
Median overall survival — the study’s primary endpoint — reached 11.1 months in the NALIRIFOX arm versus 9.2 months in the gemcitabine plus nab-paclitaxel arm (HR 0.84, 95% CI 0.71-0.99, P=0.0403).
Treatment with NALIRIFOX improved median progression-free survival (7.4 vs 5.6 months, respectively; HR 0.70, 95% CI 0.59-0.85, P=0.0001), and led to a favorable overall response rate as well (41.8% vs 36.2%).
“These results support NALIRIFOX as a new reference regimen for the first-line treatment of patients with metastatic pancreatic cancer and, hopefully, something we can build off of in the future,” said study investigator Zev Wainberg, MD, of the UCLA Medical Center-Santa Monica in California, speaking at the 2023 Gastrointestinal Cancers Symposium, when the primary findings were first presented.
The most common adverse events with NALIRIFOX (≥20% with a difference between arms of ≥5% for all grades or ≥2% for grades 3/4 events) were diarrhea, fatigue, nausea, vomiting, decreased appetite, abdominal pain, mucosal inflammation, constipation, and decreased weight. The most common laboratory abnormalities (≥10% grade 3/4) were decreases in neutrophils, potassium, lymphocytes, and hemoglobin.
The recommended irinotecan liposome dose of 50 mg/m2 is administered by IV infusion over 90 minutes every 2 weeks, and should precede the other drugs in the regimen.
In 2015, the FDA approvedopens in a new tab or window irinotecan liposome in combination with fluorouracil and leucovorin to treat patients with metastatic pancreatic cancer whose disease has progressed after gemcitabine-based chemotherapy.
The health technology company DermaSensor Inc. announced it has received U.S. Food and Drug Administration (FDA) clearance for its real-time, noninvasive skin cancer evaluation system. Intended to enable primary care physicians to provide quantitative, point-of-care testing for various types of skin cancer (including melanoma, basal cell carcinoma, and squamous cell carcinoma), the wireless, handheld device uses artificial intelligence (AI)-powered spectroscopy to identify cellular and subcellular characteristics of lesions in question for skin cancer.
The device was evaluated in the DERM-SUCCESS study, which included more than 1,000 patients from 22 study centers led by the Mayo Clinic. In this study, the device had a sensitivity of 96% across all 224 skin cancers. In addition, negative results from the study reportedly had a 97% chance of being benign across all skin cancers. In a companion clinical utility study of its use by 108 physicians, the device was found to decrease the number of missed skin cancers by half (from 18%
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