Archive for category: News

The US Food and Drug Administration (FDA) has approved the new birch triterpenes topical gel (Filsuvez) for treating 2 variants of epidermolysis bullosa (EB).Birch triterpenes topical gel is designed for partial thickness wounds in seen in individuals aged 6 months and older with a diagnosis of Junctional Epidermolysis Bullosa (JEB) and with Dystrophic Epidermolysis Bullosa (DEB).

FILSUVEZ is the first approved treatment for wounds associated with JEB, a rare, moderate-to-severe form of EB with blisters beginning in infancy. FILSUVEZ joined the Chiesi portfolio as part of the agreement reached during the acquisition of Amryt Pharma in January of this year.

EB is a debilitating inherited skin disease that causes a person’s skin to be so fragile it can be injured just from touch. This rare, chronic, and distressing disorder affects infants, children and adults and is intensely painful; recurrent blistering and chronic wounds can result in intolerable pain with limited mobility. Living with EB entails daily challenges to navigate, including slow-healing wounds at risk of infection and painful dressing changes.

FILSUVEZ is administered at home, allowing for integration into existing treatment routines. FILSUVEZ is applied topically to the wound at each dressing change.

“At Chiesi Global Rare Diseases we are driven by a need to alleviate the burdens faced in the rare disease community by providing innovative therapies and solutions that address debilitating unmet needs.” Giacomo Chiesi, Head of Chiesi Global Rare Diseases, said. “We are grateful for the support of those living with EB and their dedicated caregivers which allowed us to reach this landmark FDA approval and proudly provide FILSUVEZ as a solution for wound care management.”

Brett Kopelan, Executive Director, debra of America, added, “The FDA’s decision to approve FILSUVEZ provides those living with EB a safe and effective treatment option for the most prominent and difficult symptom of EB, open wounds that may not heal.” Kopelan also commented that, “today marks an important milestone for those living with junctional EB, as FILSUVEZ is the first FDA approved treatment for this variant of the disease. I want to thank Chiesi for their years of close collaboration with debra of America and their dedication and commitment to bringing a treatment option forward for those with dystrophic and junctional EB. I also want to thank the patients who participated in the clinical trials to bring this therapeutic option to fruition.”

FILSUVEZ was previously approved in June 2022 by the European Commission for the treatment of skin wounds in adults and children, ages 6 months and older with both JEB and DEB.

About FILSUVEZ topical gel

FILSUVEZ® (birch triterpenes) topical gel, is a medicine that is used in adults and children aged 6 months or older with epidermolysis bullosa (EB). FILSUVEZ contains a dry extract from two species of birch bark consisting of naturally occurring substances known as triterpenes, including betulin, betulinic acid, erythrodiol, lupeol and oleanolic acid. FILSUVEZ is available as a gel that should be applied to the wound surface at a thickness of approximately 1 mm and covered by a wound dressing. The medicine can also be applied directly to the wound dressing.

About EASE Trial

The EASE trial (NCT03068780) is the largest ever global Phase 3 trial conducted in patients with EB, performed across 58 sites in 28 countries. It comprises a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase. Patients with EB target wounds of between 10 and 50cm2 in size that were present for > 21 days and < 9 months were randomized in the double-blind phase to study treatment in a 1:1 ratio and wound dressings applied according to the standard of care. 223 patients were enrolled in the trial including 156 pediatric patients. Of those who completed the double-blind phase, 100% entered the open-label safety follow-up phase. The primary endpoint of the trial was to compare the efficacy of FILSUVEZ topical gel versus control gel according to the proportion of patients with complete closure of the target wound within 45 days of treatment. The primary endpoint was achieved with statistical significance (p-value = 0.013). While the key secondary endpoints did not achieve statistical significance, a number of favorable differences were observed.

New Delhi: JB Chemicals & Pharmaceuticals Ltd (JB Pharma) has announced that the Company has inked a trade mark licence agreement along with promotion and distribution pact with Novartis for select ophthalmology brands entailing a total sum of Rs 1,089 crore.

The acquired brands include Simbrinza, Travatan, Travacom, Azopt, Azarga, Vigamox, Nevanac, Ilevro, Vigadexa, Pataday.

The board of directors at its meeting held on December 19, 2023 approved the execution of a trade mark licence agreement with Novartis Innovative Therapies AG, which is perpetual in nature for the Indian market, for a portfolio of select ophthalmology brands which will be effective in January 2027, JB Pharma said in a regulatory filing.

USA: The American Diabetes Association (ADA) has released new screening and obesity management recommendations for diabetes patients. The updated Standards of Care in Diabetes—2024 is available online and is published as a supplement to the January 2024 issue of Diabetes Care.

The ADA’s Standards of Care for 2024 include new recommendations to screen for heart failure, type 1 diabetes risk, and peripheral arterial disease (PAD), along with new obesity management guidance. It also includes new recommendations related to bone health and liver disease. The Standards of Care are essentially the global guidelines for the care of individuals with diabetes and those at risk.

The ADA’s Professional Practice Committee developed the document via a scientific literature review. The panel comprised 21 professionals, including physicians from many specialities, certified diabetes care and education specialists, nurse practitioners, pharmacists, and dieticians.

This article will mainly focus on the ADA’s updates in obesity and weight management for the prevention and treatment of type 2 diabetes.

Obesity is a chronic, often relapsing disease with several physical, metabolic, and psychosocial complications, including a substantially increased type 2 diabetes risk. There is strong and consistent evidence that obesity management can delay the progression of prediabetes to type 2 diabetes (T2D) and is highly beneficial in T2D treatment.

The ADA aimed to provide evidence-based recommendations for obesity management, including pharmacologic, behavioural, and surgical intervention in people with, or at high risk of, type 2 diabetes.

Assessment and Monitoring of the Individuals with Overweight and Obesity

The use of person-centred, nonjudgmental language is recommended to foster collaboration between individuals and health care professionals, including person-first language.

To support the diagnosis of obesity, measure height and weight to calculate BMI and perform additional measurements of body fat distribution, like waist circumference, waist-to-hip ratio, and/or waist-to-height ratio.

At least annual monitoring of obesity-related anthropometric measurements is recommended to inform treatment considerations.

Accommodations should be made to provide privacy during anthropometric measurements.

In people with type 2 diabetes and overweight or obesity, weight management should represent a primary goal of treatment along with glycemic management.

People with diabetes and overweight or obese may benefit from any magnitude of weight loss. Weight loss of 3–7% of baseline weight improves glycemia and other intermediate cardiovascular risk factors.

Initial treatment approaches for obesity should be individualized.

Nutrition, Physical Activity, and Behavioral Therapy

Physical activity, nutrition, and behavioural therapy to achieve and maintain ≥5% weight loss are recommended for people with type 2 diabetes and overweight or obese.

Interventions including high frequency of counselling (≥16 sessions in 6 months) with a focus on nutrition changes, physical activity, and behavioural strategies to achieve a 500–750 kcal/day energy deficit be beneficial for weight loss and should be considered when available.

Structured programs delivering behavioural counselling (face-to-face or remote) to address barriers to access should be considered.

Nutrition recommendations should be individualized to the person’s preferences and nutritional needs.

When developing a plan of care, systemic, structural, and socioeconomic factors that may impact nutrition patterns and food choices should be considered, such as food access to healthful food options, insecurity and hunger, cultural circumstances, and other social determinants of health.

For those who achieve weight loss goals, long-term (≥1 year) weight maintenance programs are recommended, when available.

When short-term nutrition intervention using structured, very low-calorie meals (800–1,000 kcal/day) is considered.

Nutritional supplements are not effective for weight loss and are not recommended.

Pharmacotherapy

Whenever possible, minimizing medications for comorbid conditions that are associated with weight gain is recommended.

When choosing glucose-lowering medications for people with type 2 diabetes and overweight or obesity, prioritize medications with beneficial effects on weight.

Obesity pharmacotherapy should be considered for people with diabetes and overweight or obesity along with lifestyle changes. Potential benefits and risks must be considered.

In people with diabetes and overweight or obesity, the preferred pharmacotherapy should be a glucagon-like peptide 1 receptor agonist or dual glucose-dependent insulinotropic polypeptide and glucagon-like peptide 1 receptor agonist with greater weight loss efficacy (i.e., semaglutide or tirzepatide), especially considering their added weight-independent benefits.

To prevent therapeutic inertia, for those not reaching goals, reevaluate weight management therapies and intensify treatment with additional approaches.

Metabolic Surgery

Metabolic surgery should be considered as a weight and glycemic management approach in people with diabetes with BMI ≥30.0 kg/m2 who are otherwise good surgical candidates.

Metabolic surgery should be performed in high-volume centres with interprofessional teams knowledgeable about and experienced in managing obesity, diabetes, and gastrointestinal surgery.

People who undergo metabolic surgery should receive long-term medical and behavioural support and routine micronutrient, nutritional, and metabolic status monitoring.

In people who undergo metabolic surgery, routinely screen for psychosocial and behavioural health changes and refer to a qualified behavioural health professional as needed.

To sum up, new updates in managing obesity in people with diabetes, include approaches to reduce therapeutic inertia, support more personalization, and incorporate additional obesity measurements beyond body mass index (i.e., waist circumference, waist-to-hip ratio, and/or waist-to-height ratio).

Reference:

American Diabetes Association Professional Practice Committee; 8. Obesity and Weight Management for the Prevention and Treatment of Type 2 Diabetes: Standards of Care in Diabetes–2024. Diabetes Care 1 January 2024; 47 (Supplement_1): S145–S157. https://doi.org/10.2337/dc24-S008

China: Findings from a prospective cohort study have shown an association between serum glycated haemoglobin (HbA1c) levels at early gestation and the risk of subsequent pregnancy loss in pregnant women without diabetes mellitus.

USA: Warfarin use after bioprosthetic surgical aortic valve replacement (sAVR) was tied to lower all-cause mortality and decreased thromboembolism risk compared with not receiving warfarin, a recent retrospective study has revealed. The findings were published online in Mayo Clinic Proceedings. 

The use of bioprosthetic aortic valve replacement has increased significantly during the past decade. Among its advantages is that most patients can avoid warfarin for anticoagulation treatment. Even so, research has been conflicting on whether patients would benefit from more aggressive early postoperative anticoagulation treatment.

The researchers compared thromboembolic events and all-cause mortality in patients undergoing surgical aortic valve replacement receiving anticoagulation with warfarin versus patients with no systemic anticoagulation.

Mayo Clinic researchers analyzed nationwide data on more than 10,000 patients who underwent bioprosthetic aortic valve replacement. Warfarin use was associated with a 32% reduction in mortality risk. Patients treated with warfarin early postoperatively also had an increased risk of major bleeding events. 

“The findings support early warfarin use in appropriately selected patients, such as patients with low bleeding risk,” says Hartzell Schaff, M.D., a Mayo Clinic cardiovascular surgeon who contributed to the study. “There’s often reluctance to prescribe anticoagulant treatment early after surgery due to concerns about bleeding and uncertainty about benefits. Our research finds that the small increased hazard of bleeding (4% versus 2.3%) may be an acceptable risk given the benefits in terms of mortality risk as well as reduced risk of thromboembolism.”

The Mayo Clinic study analyzed deidentified patient data from 2007 to 2019 using OptumLabs Data Warehouse, which contains claims data of commercially insured and Medicare Advantage enrollees of all ages and races throughout the U.S.

Reference:

Huang Y, Schaff HV, Swarna KS, Sangaralingham LR, Nishimura RA, Dearani JA, Crestanello JA, Greason KL. Benefit of Anticoagulation Early After Surgical Aortic Valve Replacement Using Bioprosthetic Valves. Mayo Clin Proc. 2023 Dec;98(12):1797-1808. doi: 10.1016/j.mayocp.2023.08.012.