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 […]
Takeda’s ICLUSIG Receives FDA Approval for Frontline Treatment of Ph+ Acute Lymphoblastic Leukemia
Takeda recently announced a significant milestone in cancer treatment with the U.S. Food and Drug Administration (FDA) granting accelerated approval for the supplemental New Drug Application (sNDA) of ICLUSIG® (ponatinib). This approval marks a crucial advancement in the treatment landscape for adult patients newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). ICLUSIG targets […]
A for Acromegaly – Part I
Acromegaly is a rare, acquired and slowly progressive condition caused by excessive production of growth hormone (GH) by the pituitary gland, due to the presence of a “pituitary adenoma”, a benign tumor. The pituitary gland is a pea-sized endocrine gland located at the base of the skull, in charge of producing several essential hormones and […]
FDA Approves Inotuzumab Ozogamicin for Pediatric Acute Lymphoblastic Leukemia
The Food and Drug Administration (FDA) has granted approval for inotuzumab ozogamicin (Besponsa, Pfizer) for pediatric patients aged 1 year and older with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). This approval marks a significant advancement in the treatment landscape for this challenging condition. Inotuzumab ozogamicin functions by targeting CD22-positive B-cell precursor […]
Personal Genome Diagnostics
Personal Genome Diagnostics (PGDx) specializes in developing genomic products for cancer diagnosis. Combining next-generation sequencing, bioinformatics, and oncology expertise, PGDx pioneers advanced diagnostic approaches to identify genomic alterations that guide therapy selection and monitoring. Founded in 2010 by Luis Diaz and Victor Velculescu based on technology developed at the Johns Hopkins University, the company has […]
Care For Rare: Prologue
What are rare diseases? Being unique isn’t easy and Rare Diseases due to their low prevalence have not been an exception. In a law passed by the United States Congress in 1983 called the Orphan Drug Act, Rare Diseases are defined as conditions affecting less than 200,000 people in the United States. In contrast, the […]
FDA Approval of Amivantamab-vmjw: Advancing Treatment for EGFR Exon 20 Insertion-Mutated Non-Small Cell Lung Cancer
The recent approval by the Food and Drug Administration (FDA) of amivantamab-vmjw (Rybrevant, Janssen Biotech) marks a significant milestone in the treatment landscape for non-small cell lung cancer (NSCLC). This approval targets locally advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, a subgroup historically challenging to treat effectively. The […]
Versatile “Zepbound” by Eli Lilly Became the First Weight Loss Drug Approved by the FDA
Food and Drug Administration (FDA) approved ZepboundTM (Tirzepatide) for weight management in 2023. This twincertin compound is approved for controlling blood sugar in patients suffering from T2DM. Trizepatide is a precise amalgamation of the glucose-dependent insulinotropic polypeptide (GIP) and Glucagon-like peptide (GLP-1) receptors and has added advantage over Semaglutide, Liraglutide, Dulaglutide (agonist or activators of […]
Care For Rare! – Celebrating Rare Disease Day
MedNess is proudly celebrating the Rare Disease Day today. We also take this opportunity to announce a new column “*Care For Rare!*” on the MedNess platform by our newest author, Malini Gupta, PhD! We will come back tomorrow with more info on Malini, her educational background and her interest, but in the meantime, we hope […]
EpCAM CAR-T Therapy receives Dual IND from U.S. FDA and China CDE
Immunofoco, has achieved a significant milestone with the approval of Investigational New Drug (IND) applications by both the U.S. Food and Drug Administration (FDA) and the China Center for Drug Evaluation (CDE) for IMC001, an autologous CAR-T cell therapy targeting EpCAM. This groundbreaking approval marks a pivotal moment in the treatment landscape for advanced solid […]
STEMPeers’ Oncology digital news aggregator platform Onco-This-Week acquired by Rosenblatt Life Science Consultants
February 9, 2024: Erie, PA: US-based non-profit, PhD Career Support Group DBA STEMPeers, a 21,000+ member organization, announces the asset purchase agreement of their oncology news aggregator platform, Onco-This-Week, to Rosenblatt Life Science Consultants (RLS Consultants). Rosenblatt Life Science Consultants is a global consulting and training firm focusing exclusively on the healthcare and life sciences […]
Foundation Medicine
Foundation Medicine stands as an early innovator, harnessing genomics technologies for scalable, high-throughput cancer gene mutation profiling-based diagnoses. Established in 2010 in Cambridge, Massachusetts, the company’s first product, FoundationOne, was launched in 2012 as a tissue-based test. This was followed by FoundationOne Heme in 2013, an NGS based comprehensive genome profiling test catering to hematologic […]
Exact Sciences
Exact Sciences was founded in 1995 with the visionary, and perhaps ahead of its time goal of developing non-invasive colorectal cancer (CRC) testing. The company’s early and innovative stool-DNA tests like PreGen-Plus faced financial struggles and regulatory headwinds. The turning point was the 2009 collaboration with the Mayo Clinic for licensing the stool DNA test […]
Aquinnah’s potential Alzheimer’s drug capable of reducing ~70% brain tau pathology
Aquinnah Pharmaceuticals recently announced the preclinical findings on its novel therapeutic designed to slow or stop the progression of Alzheimer’s Disease (AD) and related disorders. Their drug discovery relies on identifying compounds capable of eliminating persistent stress granules. Stress granules are aggregates of proteins and RNA molecules formed as a cell survival process in response to […]