FDA Greenlights mRESVIA: Moderna’s Breakthrough in RSV Prevention
Moderna’s mRESVIA, an mRNA vaccine, has received FDA approval, a significant milestone in the fight against respiratory syncytial virus (RSV). June 9th, 2024: In a significant development for public health, Moderna has received approval from the U.S. Food and Drug Administration (FDA) for its new respiratory syncytial virus (RSV) vaccine, mRESVIA. This marks a major […]
Eyeing a Brighter Future: Two New Biosimilars Approved for Macular Edema
FDA approves biosimilars Yesafili and Opuviz for Diabetic Macular Edema, offering affordable treatment options. Breakthrough in Diabetic Macular Edema Treatment May 25th, 2024: The FDA’s recent approval of two new biosimilars, Yesafili (aflibercept-jbvf) and Opuviz (aflibercept-yszy), marks a significant breakthrough in the treatment of Diabetic Retinopathy. These biosimilars are interchangeable with Eylea (Aflibercept), a biologic […]
G for Gaucher Disease – Part I
Gaucher disease is a rare, genetic metabolic disorder where glucocerebrosidase deficiency lead to accumulation of fat-laden “Gaucher cells” in areas like the spleen, liver and bone marrow. Gaucher cells are normal macrophages which become full of unprocessed glucocerebroside and accumulate in the organs causing dysfunction and inflammation. This is the second most severe type of […]
Follicular Lymphoma Treatment Option Gains FDA Approval: Lisocabtagene Maraleucel
The Food and Drug Administration (FDA) granted accelerated approval to lisocabtagene maraleucel (Breyanzi, Juno Therapeutics). Lisocabtagene maraleucel is a chimeric antigen receptor (CAR) T-cell therapy. CAR T-cell therapies are a type of immunotherapy that involves engineering a patient’s T cells to recognize and attack cancer cells. Lisocabtagene maraleucel T cells are specifically designed to target […]
Insulin Efsitora Alfa-Substitute for Continual Insulin Administration
Eli Lilly Efistora has showed positive outcomes from QWINT-2 and QWINT-4 phase 3 clinical trials. The trials discerned impact of the once weekly insulin efsitora in patients with T2 diabetes using insulin for the first time. The trials compared results with patients requiring multiple daily insulin injections. Although results of efsitora consumption are not superior […]
F for Fanconi Anemia – Part II
Fanconi anemia (FA) is a rare genetic disorder characterized by bone marrow failure, birth defects, and a heightened risk of cancer. Symptoms typically emerge around age 7, though diagnosis may occur later when cytopenia is detected. The disorder’s average lifespan is between 20 and 30 years, with most patients developing bone marrow failure (BMF) by […]
F for Fanconi Anemia – Part I
Fanconi Anemia (FA) is a rare autosomal recessive disorder caused due to mutations in any of the known 23 genes that play a role in the DNA repair pathway. Defect in the DNA repair in all cells of the body results in accumulation of errors in these cells over time, thus making people living with […]
FDA Grants Accelerated Approval to Tarlatamab-dlle for ES-SCLC
The Food and Drug Administration (FDA) has granted accelerated approval to tarlatamab-dlle (Imdelltra, Amgen, Inc.) for the treatment of extensive stage small cell lung cancer (ES-SCLC) in patients with disease progression following platinum-based chemotherapy. Tarlatamab-dlle is an innovative bi-specific T-cell engager designed to direct the body’s immune response specifically towards cancer cells. This mechanism allows […]
E for Erdheim-Chester Disease- Part II
Diagnosing Erdheim-Chester Disease (ECD), presents significant challenges due to its rarity and varied clinical presentations. Successful diagnosis typically involves integrating descriptive pathology, clinical observations, and radiographic findings. Patients often undergo multiple biopsies, leading to delays in diagnosis and treatment initiation, with the average time from symptom onset to diagnosis ranging from months to years. Determining […]
Sanofi & Novavax Partner on COVID-19 and Flu Combination Vaccines
Sanofi and Novavax, two leading companies in the field of vaccine development, have recently announced a significant co-exclusive licensing agreement aimed at broadening access to COVID-19 vaccines and exploring novel combination vaccines targeting both COVID-19 and influenza. This collaboration underscores the companies’ commitment to advancing public health by leveraging their respective strengths in vaccine research, […]
Lixisenatide has added benefits for treating Parkinson disease
New Diabetic Drug Lixisenatide has found its new purpose to treat Parkinson disease. The studies published in New England Journal of Medicine suggested that lixisentide therapy attenuated the progression of motor disability in patients diagnosed with Parkinson disease in less than 3 years (Meissner et al., 2024). These benefits were associated with gastrointestinal side effects. […]
E for Erdheim-Chester Disease Part-1
Erdheim-Chester Disease (ECD) is an extremely rare multisystem adult-onset histiocytic disorder which is a type of a slow-growing blood cancer. Histiocytes are large phagocytic cells, typically tasked with combatting infections. In this disease, there is an excessive accumulation of histiocytes which infiltrate the body’s loose connective tissue, triggering inflammation and thickening of the affected areas. […]
Accelerated Approval of Tovorafenib for Pediatric Low-Grade Glioma: A Breakthrough in BRAF-Altered Therapy
Pediatric low-grade glioma (LGG) with BRAF alterations presents a significant therapeutic challenge, lacking approved systemic treatments. However, a recent milestone has been achieved with the Food and Drug Administration’s (FDA) accelerated approval of tovorafenib (Ojemda, Day One Biopharmaceuticals, Inc.) for patients aged 6 months and older with relapsed or refractory pediatric LGG harboring BRAF fusions, […]
The US FDA approves lutetium Lu 177 dotatate for pediatric patients with GEP-NETS
The Food and Drug Administration has recently approved lutetium Lu 177 dotatate (Lutathera, Advanced Accelerator Applications USA, Inc., a Novartis company) for pediatric patients aged 12 and above diagnosed with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), which include foregut, midgut, and hindgut neuroendocrine tumors. This approval, granted in 2018 for adults, marks the first […]
D for Duchenne Muscular Dystrophy- Part II
One of the most prevalent forms of muscle dystrophy affecting males, Duchenne Muscular Dystrophy (DMD) is a rare progressive muscle wasting disease which leads to challenges with mobility, progressive muscle weakness, respiratory difficulties, and ultimately premature death. There have been significant strides in gene-based treatments aimed at combating DMD as summarized in Figure 1. Among […]
D for Duchenne Muscular Dystrophy-Part I
Although a rare muscle disorder, Duchenne Muscular Dystrophy (DMD) is one of the frequent genetic disorders affecting male births globally. More than 30 different muscle dystrophies are known, of which DMD is classified as Dystrophinopathy, a progressive muscle degeneration disease caused by the deficiency of Dystrophin. DMD is caused due to mutations in DMD gene […]
FDA Approval of Nogapendekin Alfa inbakicept-pmln: A Breakthrough in Bladder Cancer Treatment
Bladder cancer presents a significant healthcare challenge, particularly non-muscle invasive bladder cancer (NMIBC), which can be challenging to treat effectively. The recent approval of nogapendekin alfa inbakicept-pmln (Anktiva, Altor BioScience, LLC) by the Food and Drug Administration (FDA) offers a promising new option for adult patients with BCG-unresponsive NMIBC with carcinoma in situ (CIS) with […]
Exciting Leadership Change Announcement!
Dear MedNess Readers, we hope you are enjoying the great articles our writers are bringing to you but today, this is a different announcement – we want to share with you some exciting leadership changes at MedNess! Effective April 2024, our beloved and long-time Social Media Manager, Siftjit Kaur, has been promoted to our new […]
Advancing Alzheimer’s Disease Diagnosis
Roche recently announced a significant milestone in Alzheimer’s disease diagnosis with the FDA granting Breakthrough Device Designation to its Elecsys pTau217 assay. Developed in collaboration with Eli Lilly, this blood test aims to identify amyloid pathology, a crucial marker for Alzheimer’s disease. The Elecsys pTau217 assay promises a faster and more accurate diagnosis of Alzheimer’s […]
C For Crigler- Najjar Syndrome – Part II
Crigler-Najjar Syndrome (CNS) is often characterized by jaundice at birth leading to kernicterus in which the accumulated unconjugated bilirubin reaches the brain and affects the nervous system. With the elevated unconjugated bilirubin levels, the primary therapeutic goal is to reduce the blood bilirubin to prevent kernicterus development. Phenobarbital is often given to patients with type […]
Mission Therapeutics starts a clinical trial on its Parkinson’s Disease lead, MTX325
Mission Therapeutics recently announced the commencement of Phase I first-in-human clinical trial of MTX325 as a potential disease-modifying treatment for Parkinson’s Disease (PD). The potent selective central nervous system-penetrant compound is designed to improve mitochondrial quality and function by enhancing mitophagy. It targets a deubiquitylating enzyme, USP30 known to inhibit mitophagy. While normal cells can […]
Accelerated Approval Granted to Enhertu for HER2-Positive Solid Tumors
The Food and Drug Administration (FDA) has recently granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu). This approval is significant for adult patients suffering from unresectable or metastatic HER2-positive (IHC3+) solid tumors who have exhausted prior systemic treatment options. Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly […]
Wegovy is the New Weight Loss Superstar to Win FDA Approval
Novo Nordisk, already popular weight loss med Wegovy, added another feather in its cap. NOVO’s SELECT cardiovascular outcome showed a 20% reduction in CV events. The study was conducted for five years on 17,604 patients who were obese or overweight. The trial was initiated in 2018. The FDA nod was based on the results of […]
Supplemental Biologics License Application for intravenous maintenance dosing of Alzheimer’s drug, LEQEMBI
A Supplemental Biologics License Application (sBLA) was filed to the U.S. Food and Drug Administration (FDA) by Eisai for Lecanemab-irmb (U.S. brand name: LEQEMBI®), the approved drug for treating mild cognitive impairment or early Alzheimer’s Disease (AD). Alzheimer’s Disease (AD) is the most common cause of dementia where aggregates of amyloid beta (Aβ) plaques and […]
C For Crigler-Najjar Syndrome
Crigler-Najjar Syndrome (CNS) is a rare, autosomal inherited disorder characterized by a loss or deficiency of the liver enzyme Uridine Diphosphate glucuronosyltransferase (UGT), resulting in the inability to convert and clear bilirubin from the liver. Bilirubin is an orange-yellow pigment produced by the normal breakdown of worn-out RBCs, by a process called hemolysis. To be […]
B for Beta Thalassemia – Part II
Treatment options for Beta Thalassemia involve alleviating symptoms, managing complications, and improving quality of life. The specific treatment approach depends on the type and severity of Thalassemia. Standard therapy for this disease includes repeated lifelong blood transfusions to compensate for the lack of enough functional red blood cells. Patients with transfusion-dependent Thalassemia (TDT)require transfusions every […]
Advancing Cancer Treatment: FDA Approval of Mirvetuximab Soravtansine-Gynx for Platinum-Resistant Epithelial Ovarian Cancer
Image Source: Vinoth Khandelwal In a recent development for the treatment of platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, the Food and Drug Administration (FDA) has recently approved mirvetuximab soravtansine-gynx (Elahere, ImmunoGen, Inc. [now a part of AbbVie]). This approval marks a milestone in addressing the medical needs of adult patients with FRα […]
B for Beta Thalassemia
Beta Thalassemia (BT) is an inherited autosomal rare recessive blood disorder caused by reduced levels of functional hemoglobin (Hb), the red iron heavy oxygen-carrying pigment of the blood. Hemoglobin is a tetramer of globin chains each bound to a heme group which is bound to iron which accommodates oxygen. The four types of globin chains […]
Novo Weight Loss Drug Semaglutide (Ozempic’s) Reduces Kidney Disease Progression
Novo Semaglutide, aka Ozempic, has added another feather to the cap besides reducing body weight. Ozempic has been shown to reduce the progression of chronic kidney disease (CKD). The FLOW trial has shown that administration of Semaglutide (1.0 mg) reduces the risk of kidney disease-related problems in patients suffering from type 2 diabetes. This reduction is 24 […]
A for Acromegaly – Part II
On March 5th, 2024, Camurus AB, a Swedish biopharmaceutical company, announced FDA approval for the review of their New Drug Application (NDA) for OclaizTM (CAM2029) for the treatment of Acromegaly. The FDA has also set a Prescription Drug User Fee Act (PDUFA) target action date for October 21, 2024. The NDA submission is supported by […]
