Hepatomegaly

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.

PANTHERx®Rare Selected by Edenbridge Pharmaceuticals as Specialty Pharmacy for Yargesa® (miglustat 100 mg capsules)

Retrieved on: 
Monday, April 1, 2024
Disease, Capsule, Patient, Pharmacy, Bone pain, Hepatomegaly, Enzyme replacement therapy, Anemia, Gaucher, Thrombocytopenia, Liver, Spleen, Pharmaceutical industry

Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.

Key Points: 
  • Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.
  • Through this process, Yargesa® reduces the buildup of harmful glycosphingolipids and symptoms of the disease.
  • This accumulation leads to manifestations including but not limited to enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.
  • "We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease.

Human medicines European public assessment report (EPAR): Mycamine, micafungin, Date of authorisation: 25/04/2008, Revision: 18, Status: Authorised

Retrieved on: 
Tuesday, January 9, 2024
Fungal infection, Liver, Marketing, Chills, INN, Risk, Leukopenia, Hypotension, Fungus, Hypertension, Nausea, DNA, Inflammation, Liver disease, Anatomy, IIA, CHMP, Diarrhea, Fever, Infection, ATC, Hepatomegaly, European, Neutrophil, Neoplasm, HIV, Tachycardia, Anemia, Hypocalcemia, Hypokalemia, Patient, Medical Subject Headings, Caspofungin, European Commission, Echinocandin, Kidney failure, International, Bilirubin, Neutropenia, Blood, Language, Thrombocytopenia, Magnesium, Select, Medicine, Vomiting, Phlebitis, Amphotericin B, Allergy, Fluconazole, Headache, Rat, Haematopoiesis, Committee, Checklist, Alkaline phosphatase, Rash, Medical device

Human medicines European public assessment report (EPAR): Mycamine, micafungin, Date of authorisation: 25/04/2008, Revision: 18, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Mycamine, micafungin, Date of authorisation: 25/04/2008, Revision: 18, Status: Authorised

Electra Therapeutics highlights ELA026 Phase 1b study in sHLH and presents preclinical data at ASH Annual Meeting

Retrieved on: 
Monday, December 12, 2022
Oncology, Health, Stem Cells, Clinical Trials, Pharmaceutical, Biotechnology, ADCP, Therapy, Histiocytosis, Lymphocyte, Primate, MD, Monocyte, Immunotherapy, Infection, Poster, PK, Pharmacology, Protein, Society, SIRP, Biomarker, Patient, Trial of the century, Cancer, Multiple organ dysfunction syndrome, Splenomegaly, Myelocyte, Death, Incidence, Autoimmune disease, Roger Hawkins (drummer), Clinical trial, Safety, Hepatomegaly, ADCC, Immunosuppression, ASH, PD, Cytopenia, Pharmaceutical industry, Vaccine, Medical device

Electra also announced today that a Phase 1b global study of ELA026 in sHLH patients has been initiated and is ongoing (ClinicalTrials.gov identifier: NCT05416307 ).

Key Points: 
  • Electra also announced today that a Phase 1b global study of ELA026 in sHLH patients has been initiated and is ongoing (ClinicalTrials.gov identifier: NCT05416307 ).
  • The Phase 1b study is highlighted as a clinical trial in progress poster at ASH, titled A Phase 1b study of ELA026 in patients with secondary hemophagocytic lymphohistiocytosis.
  • The Phase 1b study is an open-label, single-arm, multicenter study which will evaluate the safety and efficacy of ELA026, assess biomarkers and identify a dose for Phase 2/3 testing.
  • We have created a robust design for this Phase 1b study with the aim of progressing rapidly toward a Phase 2/3 study so we can accelerate ELA026 as a potential treatment option for sHLH patients in need.

Electra Therapeutics to Highlight ELA026 in Three Presentations at the American Society of Hematology (ASH) 2022 Annual Meeting

Retrieved on: 
Thursday, November 3, 2022
Biotechnology, Pharmaceutical, Health, Infection, Cytopenia, Multiple organ dysfunction syndrome, Society, Death, Hepatomegaly, Electra, ASH, Therapy, Splenomegaly, Autoimmune disease, SIRP, Toxicity, Immunotherapy, Patient, Cancer, Program, Vaccine

With ELA026, Electra is taking a first-in-class approach to targeting SIRP to enable precise depletion of pathological immune cells.

Key Points: 
  • With ELA026, Electra is taking a first-in-class approach to targeting SIRP to enable precise depletion of pathological immune cells.
  • ELA026 is currently in Phase 1 clinical studies, including a Phase 1b trial in patients with secondary hemophagocytic lymphohistiocytosis (sHLH), a life-threatening inflammatory disease.
  • Details of the poster presentations are as follows:
    Secondary hemophagocytic lymphohistiocytosis (sHLH) is a life-threatening hyperinflammatory condition for which there is no approved treatment.
  • ELA026 is in clinical development for secondary hemophagocytic lymphohistiocytosis (sHLH), a life-threatening hyperinflammatory condition for which there is no approved treatment.

Bristol Myers Squibb Announces Positive Topline Results of Phase 3 COMMANDS Trial

Retrieved on: 
Monday, October 31, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, People, Hepatomegaly, Beta thalassemia, Safety, Hypertension, Myelodysplastic syndrome, Deep vein thrombosis, DBP, History, MDS, Splenomegaly, SBP, EMH, Patient, The Bristol-Myers Squibb Children's Hospital, BMY, RBC, NYSE, Risk, Stroke, ESA, Spinal cord compression, Adult, Clinical trial, Anemia, Acquisition, Iron overload, Embolism, HB, Lymphatic system, Merck, Portal vein thrombosis, Bristol Myers Squibb, Microdeletion syndrome, Incidence, TEE, Splenectomy, Thrombosis, Hormone replacement therapy, Pharmaceutical industry, Blood product

We are pleased with the positive results of the COMMANDS study and look forward to presenting these important data.

Key Points: 
  • We are pleased with the positive results of the COMMANDS study and look forward to presenting these important data.
  • Bristol Myers Squibb will complete a full evaluation of the COMMANDS data and work with investigators to present detailed results at an upcoming medical meeting, as well as discuss these results with health authorities.
  • Bristol Myers Squibb thanks the patients and investigators who are participating in the COMMANDS clinical trial.
  • For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn , Twitter , YouTube , Facebook and Instagram .

First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 1/2 Trial for the Treatment of Gaucher Disease in China

Retrieved on: 
Thursday, July 21, 2022
Science, Biotechnology, Research, Pharmaceutical, Oncology, General Health, Health, Clinical Trials, Glycogen storage disease type II, University of Washington School of Medicine, Death, ERT, Degenerative disease, Treatment, III, University, GBA, Glioblastoma, WuXi Biologics, Doctor of Philosophy, CEO, Haemophilia, Research, Fracture, Gaucher, Partnership, Bone pain, Peking Union Medical College Hospital, Safety, Gaucher's disease, University of Massachusetts, Glycogen storage disease, Cell, Quality of life, Hematology, Clinical trial, GD, Lysosomal storage disease, Disease, Hepatomegaly, Splenomegaly, Patient, Pharmacokinetics, Hunter syndrome, Department, Frost & Sullivan, Thrombocytopenia, Anemia, Diagnosis, Lysosome, Lists of diseases, Population, Enzyme replacement therapy, Pharmaceutical industry

CANbridge Pharmaceuticals Inc. (HKEX:1228), a China and US-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the first patient has been dosed in the CAN103 Phase 1/2 trial in treatment-nave patients with Gaucher disease (GD) Types I and III in China.

Key Points: 
  • CANbridge Pharmaceuticals Inc. (HKEX:1228), a China and US-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the first patient has been dosed in the CAN103 Phase 1/2 trial in treatment-nave patients with Gaucher disease (GD) Types I and III in China.
  • CAN103 is an enzyme replacement therapy (ERT) under development by CANbridge, as part of its rare disease partnership with WuXi Biologics (2269.HK), for the long-term treatment of adults and children with Gaucher disease Types I and III.
  • Part A (Phase 1) is an open-label study to evaluate the safety, tolerability and pharmacokinetics of different dose levels of CAN103 ERT in a small number of treatment-nave subjects with Gaucher disease Type I.
  • Dosing the first patient in the CAN103 Gaucher disease trial demonstrates the CANbridge commitment to developing rare disease solutions.

CANbridge Pharmaceuticals CAN108 New Drug Application/Orphan Drug Registration (NDA/ORD) for Alagille Syndrome Accepted by the Taiwan Food and Drug Administration

Retrieved on: 
Tuesday, March 29, 2022
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, IBAT, Heart, MPS II, Haemophilia, Therapy, Liver injury, University of Massachusetts Medical School, University, 2-Bromo-LSD, Glioblastoma, Patient, GBM, Genetic distance, Excoriation disorder, Liver, Research, Biliary atresia, Serum, Sleep, WuXi Biologics, Taiwan, Hepatomegaly, Itch, Food, Cholestatic pruritus, Enterohepatic circulation, Hunter syndrome, CEO, Drug discovery, Frost & Sullivan, MPS, Glycogen storage disease, Breakthrough therapy, PFIC, Liver disease, US Foods, University of Massachusetts, Jaundice, Disorder, Xanthoma, NDA, Alagille syndrome, Primary biliary cholangitis, Lists of diseases, FDA, Spiroplasma mirum, University of Washington School of Medicine, Disease, Pharmaceutical industry, Generic drug, ALAGILLE SYNDROME (ALGS), MARALIXIBAT

CANbridge recently received an acceptance letter for a CAN108 NDA the same indication from Chinas National Medical Products Administration.

Key Points: 
  • CANbridge recently received an acceptance letter for a CAN108 NDA the same indication from Chinas National Medical Products Administration.
  • CANbridge and Mirum Pharmaceuticals signed an exclusive license agreement for the development, commercialization and manufacture, under certain conditions, of maralixibat (CAN108) in Greater China last year.
  • As our first therapeutic under development for rare liver disease, CAN108 deepens our rare disease portfolio and highlights the swiftness of our regulatory progress.
  • Alagille syndrome (ALGS) is a rare autosomal dominant disorder that affects multiple organs, including the liver, heart, bones and eyes.

VONJO™ (pacritinib) Now Approved for the Treatment of Adults with Intermediate or High-Risk Primary or Secondary Myelofibrosis with a Platelet Count Below 50 x 109/L

Retrieved on: 
Friday, March 4, 2022
Oncology, Health, Genetics, General Health, Radiology, Pharmaceutical, Biotechnology, Thrombocytopenia, Specialty pharmacy, Bone marrow, Regulation of tobacco by the U.S. Food and Drug Administration, Vera, Lymphatic system, Patient, Adult, Food, Aplastic anemia, Biopharma LLC, Myeloproliferative neoplasm, FDA, United, Survival, Splenomegaly, Haematopoiesis, Fatigue, Ruxolitinib, Leukemia, NCT, Hepatomegaly, Physician, Platelet, CTI, Medical imaging

Onco360 is honored to become a specialty pharmacy provider for VONJO patients, said Benito Fernandez, Chief Commercial Officer, Onco360.

Key Points: 
  • Onco360 is honored to become a specialty pharmacy provider for VONJO patients, said Benito Fernandez, Chief Commercial Officer, Onco360.
  • We are committed to supporting the highly specialized needs of myelofibrosis patients with thrombocytopenia and their physicians across the United States.
  • Approximately 1.5 myelofibrosis cases per 100,000 people are diagnosed annually in the United States.
  • The FDAs approval of VONJO is based upon the results of the PERSIST-2 clinical trial (NCT 02055781).

CANbridge Announces Approval of CAN108 for Rare Liver Disease, Alagille Syndrome, Under the Early and Pilot Implementation Policy in Boao Lecheng International Medical Tourism Pilot Zone

Retrieved on: 
Friday, February 25, 2022
Biotechnology, General Health, Pharmaceutical, Health, MPS II, IBAT, Policy, Lists of diseases, Disorder, Tuba mirum, Glycogen storage disease, Dysplasia, Liver, MPS, NDA, Research, Incidence, University, National Medical Products Administration, Therapy, Biliary atresia, University of Massachusetts Medical School, Growth, GBM, Haemophilia, Time-invariant system, Liver transplantation, University of Massachusetts, Disease, NMPA, New Drug Application, Degenerative disease, Hunter syndrome, Hepatomegaly, Glioblastoma, Cholestatic pruritus, Quality of life, CEO, Jaundice, Alagille syndrome, LSD, Serum, Fatty liver disease, Excoriation disorder, Kidney, Bile, WuXi Biologics, Patient, Enterohepatic circulation, Liver injury, PFIC, Death, Itch, Ageing, Liver disease, FDA, Sleep deprivation, Pharmaceutical industry, Fine chemical, Alagille Syndrome (ALGS), CAN108 (maralixibat), CANbridge Pharmaceuticals Inc., Biotechnology, General Health, Pharmaceutical, Health, Alagille Syndrome (ALGS), CAN108 (maralixibat), CANbridge Pharmaceuticals Inc., ALAGILLE SYNDROME (ALGS), CAN108 (MARALIXIBAT), CANBRIDGE PHARMACEUTICALS INC.

The Early and Pilot Implementation Policy of Boao Lecheng International Medical Tourism Pilot Zone enables Chinese patients to access therapeutics that are available in other parts of the world, thereby improving the quality of life of patients, especially children.

Key Points: 
  • The Early and Pilot Implementation Policy of Boao Lecheng International Medical Tourism Pilot Zone enables Chinese patients to access therapeutics that are available in other parts of the world, thereby improving the quality of life of patients, especially children.
  • CANbridge has the exclusive license to develop and commercialize CAN108 in Greater China for three rare liver disease indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA).
  • The National Medical Products Administration (NMPA) has accepted a New Drug Application (NDA) for CAN108 for Alagille syndrome in China under priority review.
  • Alagille syndrome (ALGS) is an autosomal dominant multisystem disorder which can lead to end-stage liver disease and death.