FDA Grants Accelerated Approval to Adagrasib with Cetuximab for KRAS G12C-Mutated Colorectal Cancer
The Food and Drug Administration (FDA) has granted accelerated approval for the combination of adagrasib (Krazati; Mirati Therapeutics, Inc.) and cetuximab for the treatment of adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC). This approval applies to patients who have previously been treated with chemotherapy regimens based on fluoropyrimidine, oxaliplatin, and irinotecan, […]
I for I-Cell Disease- Part I
I-cell disease, also known as Mucolipidosis type II, is a severe rare lysosomal storage disorder caused by mutations in the GNPTAB gene located on chromosome 12q23.2. This gene encodes the alpha and beta subunits of N-acetylglucosamine-1-phosphotransferase, which plays a critical role in processing and packaging lysosomal enzymes by tagging them with mannose-6-phosphate in the Golgi […]
Iqirvo Clears FDA Hurdle, Offering New Relief for Primary Biliary Cholangitis Patients
The FDA approves Iqirvo (elafibranor) for primary biliary cholangitis (PBC) treatment, offering a new option for patients intolerant to UDCA. What is Primary Biliary Cholangitis (PBC)? A case study from December 2023 reports a 19-year-old male with multiple congenital abnormalities and an intellectual disability suffered from pruritus for 5 years. Tests revealed elevated levels of […]
H for Huntington’s Disease- Part II
Huntington’s disease (HD) is a debilitating neurodegenerative condition caused by an abnormal expansion of CAG triplets in the huntingtin (HTT) gene, leading to the progressive deterioration of GABAergic neurons in the brain’s central regions. As of now, there is no cure for HD, and treatment focuses on symptom management and improving quality of life. The […]
FDA Approves Blinatumomab as Consolidation Therapy for CD19-Positive Philadelphia Chromosome-Negative B-Cell Precursor Acute Lymphoblastic Leukemia
The Food and Drug Administration (FDA) has granted approval for blinatumomab (Blincyto, Amgen) for use in adult and pediatric patients aged one month and older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (Ph-negative BCP ALL) during the consolidation phase of multiphase chemotherapy. This decision marks a significant advancement in the treatment regimen for […]
H for Huntington’s Disease – Part I
Huntington’s disease (HD) is a rare, inherited neurodegenerative disorder affecting the central nervous system. Characterized by involuntary movements, behavioral disturbances, and dementia, HD typically manifests between ages 30 and 50, though juvenile cases also occur. Symptoms include chorea, dystonia, cognitive decline, psychiatric issues, and difficulties with speech and swallowing. The disease leads to complete dependency […]
FDA Grants Accelerated Approval to Repotrectinib for NTRK Fusion-Positive Tumors
The Food and Drug Administration (FDA) has granted accelerated approval to repotrectinib (AUGTYRO, Bristol-Myers Squibb; BMS) for the treatment of adult and pediatric patients aged 12 and older with solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. This approval covers patients with locally advanced or metastatic tumors for whom surgical resection would […]
Breaking Barriers in Rare Disease Treatment: FDA Approves Bkemv as First Interchangeable Biosimilar
The US Food and Drug Administration (FDA) has approved Amgen’s Bkemv as the first interchangeable biosimilar to Soliris for treating rare diseases, such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The FDA has approved Bkemv, a biosimilar, as the first interchangeable treatment for two rare and debilitating diseases: paroxysmal nocturnal hemoglobinuria […]
US FDA Approves Breyanzi for Relapsed or Refractory Mantle Cell Lymphoma
The U.S. Food and Drug Administration (FDA) has approved lisocabtagene maraleucel (Breyanzi), developed by Juno Therapeutics, Inc., for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have undergone at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor (BTKi). This approval marks a significant advancement […]
G for Gaucher Disease – Part II
Gaucher disease results from mutations in the GBA gene, disrupting the breakdown of glucocerebroside and causing its accumulation, leading to cellular damage. Efforts to repair this faulty “switch” have long been pursued in medical research. This disease though currently incurable, can be managed effectively with available therapies. Type 1 Gaucher patients can lead full lives, […]
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 […]
