Urinary tract infection

Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, System, Primary, Calcium oxalate, Enzyme, DSM-IV codes, Cell, Disease, Abdominal pain, Patient, Committee, Knowledge, Kidney failure, Organic acid, Regulation, EMA, IRIS, Glycine, Protein, Human, AGXT, Urine, European, COMP, Urinary tract infection, European Commission, Kidney, Safety, Blood, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Civil service commission, Diagnosis, Idiopathic pulmonary fibrosis, Disease, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Hydrogen, IPF, European, Collagen, COMP, Oxygen, Urinary tract infection, Sponsor, European Commission, Safety, Blood, Marketing, Medicine, Pharmaceutical industry

Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

U.S. FDA Approves Broad New Labels for NEXLETOL® and NEXLIZET® to Prevent Heart Attacks and Cardiovascular Procedures in Both Primary and Secondary Prevention Patients, Regardless of Statin Use

Retrieved on: 
Friday, March 22, 2024
Glucose, Urinary tract infection, Anaphylaxis, Myocardial infarction, Common, Bronchitis, Patient, European Medicines Agency, Ezetimibe, Therapy, Anemia, Pain, Sinusitis, Esperion Therapeutics, Bempedoic acid, Abdominal pain, Risk, Gout, Committee, Spasm, ESPR, Back pain, ATP, Safety, Membrane protein, Fatigue, CHMP, Arthralgia, Angioedema, Cardiovascular disease, Kidney, Constipation, Death, Adverse event, Pregnancy, CLEAR, Rash, Esperies, Rupture, FDA, Molina Healthcare, Hypersensitivity, Health, Conference, Tablet, CVD, Incidence, Diarrhea, Fetus, Gallstone, Diet, Excipient, Upper respiratory tract infection, Uric acid, Pharmaceutical industry

In addition, the enhanced labels support the use of NEXLETOL and NEXLIZET either alone or in combination with statins.

Key Points: 
  • In addition, the enhanced labels support the use of NEXLETOL and NEXLIZET either alone or in combination with statins.
  • They also include new indications for primary hyperlipidemia, alone or in combination with a statin, and are the only LDL-C lowering non-statin drugs indicated for primary prevention patients.
  • NEXLETOL and NEXLIZET are also the first oral non-statin LDL-C lowering drugs to be approved by the FDA to reduce the risk of CV events in both primary and secondary prevention patients.
  • CLEAR Outcomes is part of the CLEAR clinical research program for NEXLETOL® (bempedoic acid) Tablet and NEXLIZET® (bempedoic acid and ezetimibe) Tablet.

CHMP Issues Positive Opinions For Both Bempedoic Acid And The Bempedoic Acid / Ezetimibe Fixed-Dose Combination Tablet As Treatments For Hypercholesterolemia And Significantly Reducing Cardiovascular Events

Retrieved on: 
Friday, March 22, 2024
Urinary tract infection, Anaphylaxis, Bronchitis, European Commission, Tendon rupture, Patient, European Medicines Agency, Bempedoic acid, Ezetimibe, Therapy, Citrate synthase, Anemia, Pain, Sinusitis, Abdominal pain, Risk, Gout, Ageing, Committee, ACL, Spasm, EMA, Back pain, ATP, Safety, Fatigue, CHMP, Arthralgia, Angioedema, Tablet, Constipation, Agent handling, Death, Pregnancy, CLEAR, Rash, Kidney, Moyamoya disease, Rupture, Apolipoprotein, Hypercholesterolemia, Hypersensitivity, Health, CVD, Incidence, Diarrhea, Fetus, Esperion Therapeutics, Diet, Upper respiratory tract infection, FDC, Uric acid

ANN ARBOR, Mich. and MUNICH, Germany, March 22, 2024 (GLOBE NEWSWIRE) -- Daiichi Sankyo Europe GmbH (hereafter, ‘Daiichi Sankyo’) and Esperion Therapeutics, Inc. jointly announced today, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions for the label update of both bempedoic acid (marketed as NILEMDO®▼) and the bempedoic acid / ezetimibe fixed dose combination (FDC) (marketed as NUSTENDI®▼ ), recommending their approval as treatments to reduce low-density lipoprotein cholesterol (LDL-C) and cardiovascular risk.2 The existing label of bempedoic acid (NILEMDO®▼) provides authorisation for adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet:2

Key Points: 
  • “We are thrilled with the positive CHMP opinion, which reflects the significant cardiovascular risk reduction benefit that the bempedoic acid global franchise brings to patients worldwide,” said Sheldon Koenig, President and CEO, Esperion.
  • The CHMP is a scientific committee of the EMA that reviews medical product applications on their scientific and clinical merit.
  • Hyperuricemia: Bempedoic acid, a component of NEXLIZET and NEXLETOL, may increase blood uric acid levels which may lead to gout.
  • CLEAR Outcomes is part of the CLEAR clinical research program for NEXLETOL® (bempedoic acid) Tablet and NEXLIZET® (bempedoic acid and ezetimibe) Tablet.

Three Patients with Invasive Fusarium Infection in Matinas BioPharma’s Oral MAT2203 Compassionate/Expanded Use Access Program Achieve Complete Clinical Response

Retrieved on: 
Friday, March 22, 2024
Nephrotoxicity, Urinary tract infection, Fungal infection, Culture, Ventilator-associated pneumonia, Burns, Internal medicine, Amphotericin B, Emphysema, Azole, Coronary artery disease, Debridement, Carcinoma, Risk, Hyperlipidemia, Patient, Lung, LNC, Fusarium, Female, Malignancy, University, Doctor of Philosophy, MD, Aspergillosis, Echinocandin, Fever, Immune system, Division, Hypertension, Voriconazole, Physician, Infection, Pharmaceutical industry

The successful MAT2203 treatment outcome in these patients therefore adds to our confidence for the upcoming ORALTO Phase 3 trial in patients suffering from invasive aspergillosis with limited treatment options.

Key Points: 
  • The successful MAT2203 treatment outcome in these patients therefore adds to our confidence for the upcoming ORALTO Phase 3 trial in patients suffering from invasive aspergillosis with limited treatment options.
  • She was transitioned to oral MAT2203 for two weeks, which led to clinical resolution of her fungal infection.
  • She began to show clinical improvement following two weeks of oral MAT2203 treatment and her skin wounds completely healed following six months of MAT2203 treatment.
  • “MAT2203 continues to demonstrate its potential to effectively treat invasive fungal infections and help patients achieve complete clinical resolution.

Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy

Retrieved on: 
Wednesday, March 20, 2024
Encephalitis, ICER, Bone marrow, Patient, DSM-IV codes, Urinary tract infection, Travel, Child, Health technology assessment, Therapy, Children's Hospital of Philadelphia, Central nervous system, Risk, MLD, Qualification, Health, WAC, NBS, Nerve, Mutation, UCSF, Disease, Federation, Research, Acquisition, Genome, Rash, Stem cell, HSC, Hospital, Family, Swelling, FDA, Diagnosis, HTA, Caregiver, Traffic barrier, Viral, Fever, Kyowa Kirin, PSLI, Liver, Government, San Raffaele Hospital, ARSA, Pharmaceutical industry

TOKYO, LONDON and BOSTON, March 20, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the details of its U.S. commercial launch of Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, the first FDA-approved therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile, (PSEJ), or early symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

Key Points: 
  • Prior to Lenmeldy, there were no treatment options in the U.S. for early-onset MLD beyond supportive and end-of-life care.
  • This approach has the potential to restore enzymatic function to stop or slow disease progression with a single treatment.
  • Utilizing results from such studies, a multi-stakeholder working group is finalizing a nomination to add MLD to the U.S.
  • Based on current timelines and assumptions, Orchard Therapeutics expects the nomination will be submitted in mid-year 2024.

RespireRx Pharmaceuticals Inc. Reports Publication of Preclinical Research Results Demonstrating the Ability of CX1739, its Lead Clinical AMPAkine, to Improve Bladder Function After Spinal Cord Injury

Retrieved on: 
Wednesday, March 20, 2024
OTC Markets Group, Neurogenic bladder dysfunction, Death, Ampakine, DOI, Tetraplegia, Rat, Incidence, Bourbeau, Risk, Bruise, Patient, Research, University, Bladder cancer, Quality of life, Pleasure, EMG, Alom, Catheter, AMPA, Injury, Interim, CSO, Bangladesh Technical Education Board, Urinary tract infection, Respiration, Animal, Kidney, SCI, Rana, RespireRx

Restoration of bladder function is ranked as one of the highest priorities by individuals with SCI (Bourbeau et al., Spinal Cord 58:1216–1226; 2020).

Key Points: 
  • Restoration of bladder function is ranked as one of the highest priorities by individuals with SCI (Bourbeau et al., Spinal Cord 58:1216–1226; 2020).
  • Current treatment approaches usually require interventions such as catheterization for urinary voiding, which have their own set of risks and potentially significant set of complications.
  • The present paper, entitled “Acute ampakines increase voiding function and coordination in a rat model of SCI” (Rana, Alom et al.
  • We believe that this research has the potential to represent a novel, breakthrough in the treatment of SCI, which is badly needed.”

Recce Pharmaceuticals Completes Dosing Cohort in Phase I/II Trial of RECCE® 327 for Urinary Tract Infections and Urosepsis

Retrieved on: 
Tuesday, March 19, 2024
Urinary tract infection, Global health, Patient, ASX, RCE, Infection, Pyelonephritis, Pharmaceutical industry

“Completing dosing of our latest cohort marks another milestone in our journey toward advancing R327 as a potential frontline treatment for patients with UTIs or urosepsis,” said James Graham, Chief Executive Officer of Recce Pharmaceuticals.

Key Points: 
  • “Completing dosing of our latest cohort marks another milestone in our journey toward advancing R327 as a potential frontline treatment for patients with UTIs or urosepsis,” said James Graham, Chief Executive Officer of Recce Pharmaceuticals.
  • “We are dedicated to delivering impactful solutions to combat infectious diseases and improve global health outcomes.”
    An Independent Safety Committee will now review and evaluate the comprehensive data from the 4-subject cohort.
  • Data from this trial is expected to pave the way for a Phase II trial to establish the potential of R327 as a frontline treatment for patients with UTIs or urosepsis.
  • The administration of antibiotics via rapid intravenous infusions is a proven safe and effective method of administration, impacting patient treatment, wait times, and nursing workloads globally.

SPEVIGO® approved for expanded indications in China and the US

Retrieved on: 
Tuesday, March 19, 2024
Arthralgia, Urinary tract infection, Uncertainty, Human, NMPA, Psoriasis, Disease, Multiple organ dysfunction syndrome, Incidence, Risk, GPP, Boehringer Ingelheim, Patient, Ageing, Executive officer, Immunoglobulin G, Anxiety, Doctor of Philosophy, MD, Quality of life, Flare, Injection site reaction, Clinical trial, Skin, Fever, Immune system, Pain, Itch, Food, FDA, Yale University, Fear

In the trial with 123 patients, no flares were observed after week 4 of SPEVIGO® subcutaneous treatment in the high-dose group (n=30).13,14,15,16 In the Effisayil® 2 trial, SPEVIGO® was associated with an increased incidence (≥9 cases per 100 patient-years) of injection site reaction, urinary tract infection, arthralgia, and pruritus compared to placebo.

Key Points: 
  • In the trial with 123 patients, no flares were observed after week 4 of SPEVIGO® subcutaneous treatment in the high-dose group (n=30).13,14,15,16 In the Effisayil® 2 trial, SPEVIGO® was associated with an increased incidence (≥9 cases per 100 patient-years) of injection site reaction, urinary tract infection, arthralgia, and pruritus compared to placebo.
  • “Until now, people living with GPP have not had any approved options to treat their disease,” said Bruce Strober, MD, PhD, Clinical Professor, Dermatology, Yale University and Central Connecticut Dermatology.
  • “Experiencing GPP can be mentally and physically devastating, leaving those affected with uncertainty and fear of the next episode.
  • Therefore, expanding the treatment of GPP is a critical step towards addressing patients’ needs.”

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

Retrieved on: 
Monday, March 18, 2024
Gastroenteritis, Genomics, Metabolism, Neutropenia, Brain, Nerve, Mutation, RPD, Disease, Incidence, Partnership, Central nervous system, Genetics, GSK, Stomatitis, Patient, Research, Fats, PRV, Traffic barrier, Child, Viral, Gene therapy, SAN, Genome, Urinary tract infection, Rash, Hepatomegaly, Priority review, Stem cell, Infection, Therapy, MLD, Kyowa Kirin, Febrile neutropenia, Fever, PSLI, Prognosis, Health, Family, FDA, San Raffaele Hospital, ARSA, Orchard, Food, Pharmaceutical industry

“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.