Haematopoiesis

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Death, Glycosaminoglycan, B cell, Enzyme, Mutation, Medicine, Cell, Mental disability, Disease, Patient, Committee, Knowledge, Scheie syndrome, Regulation, EMA, IRIS, Periodic breathing, Mucopolysaccharidosis, European, COMP, Enzyme replacement therapy, European Commission, Safety, DSM-IV codes, IQVIA, Marketing, Haematopoiesis, Liver, RDS, FGK, Hurler syndrome, MPS, Pharmaceutical industry

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Key Points: 


Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in ha[...]

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Graft-versus-host disease, Medicine, Cell, Bone marrow, Risk, Haematopoiesis, Patient, Committee, Knowledge, Regulation, HSCT, EMA, IRIS, European, Leukemia, COMP, Immune system, European Commission, Consultant, Safety, Haematopoietic system, Blood, Marketing, Umbilical cord, CATS, Pharmaceutical industry

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Key Points: 


Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Keros Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, February 28, 2024
Therapy, Research, Syndrome, Investment, Obesity, Technology transfer, Growth, KROS, PAH, Patient, Disorder, Protein, Ruxolitinib, Cytopenia, Orthostatic hypertension, Haematopoiesis, Keros, Pharmaceutical industry

Keros did not generate any revenue for the year ended December 31, 2022.

Key Points: 
  • Keros did not generate any revenue for the year ended December 31, 2022.
  • Research and development expenses were $37.5 million for the fourth quarter and $135.3 million for the year ended December 31, 2023, as compared to $24.9 million for the fourth quarter and $87.3 million for the year ended December 31, 2022.
  • General and administrative expenses were $9.1 million for the fourth quarter and $34.8 million for the year ended December 31, 2023, as compared to $7.1 million and $27.5 million for the fourth quarter and year ended December 31, 2022.
  • Keros’ cash and cash equivalents as of December 31, 2023 was $331.1 million compared to $279.0 million as of December 31, 2022.

Ryvu Therapeutics to Present Preclinical Data on RVU120 and Synthetic Lethality Programs at the 2024 AACR Annual Meeting

Retrieved on: 
Wednesday, March 6, 2024
WBC, WRN, Lung cancer, RNA, Malignancy, Microsatellite instability, Proteome, Intellectual property, MMR, MPN, Glioblastoma, Neoplasm, Splenomegaly, DMPK, Phosphorylation, HDAC, STAT5, RNA-Seq, WES, Standard of care, Therapy, RecQ helicase, Phenotype, Ruxolitinib, DNA, DNA repair, Pancreatic serous cystadenoma, CDKN2A, Cancer, CRC, PIM, Arginine, MTA, Abstract, PDC, MTAP, Colorectal cancer, CRISPR, PV, MF, MSI-H, Cell, JAK, DNA mismatch repair, Medicine, Patient, Gene, PRMT5, Haematopoiesis, Mesothelioma, Pharmaceutical industry

Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.

Key Points: 
  • Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.
  • "We are excited to present our latest preclinical data at the AACR Annual Meeting, showcasing our significant progress in advancing novel small molecule therapies for oncology.
  • This year, we will present data from our most advanced preclinical project on MTA-cooperative PRMT5 inhibitors, the lead program within Ryvu's synthetic lethality pipeline.
  • Ryvu has developed potentially best-in-class MTA-cooperative PRMT5 inhibitors showing favorable drug-like properties and effective PRMT5 inhibition dependent on MTA binding.

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Retrieved on: 
Sunday, February 4, 2024
Hearing, Inflammation, Civil service commission, Knowledge, European, Cell, Haematopoiesis, COMP, IRIS, Bone marrow, Mitochondrion, Adrenal insufficiency, Regulation, Gland, ABCD1, Patient, Safety, Diagnosis, EMA, ALD, European Commission, Adrenoleukodystrophy, Seizure, B cell, PPAR, Brain, Disease, Therapy, Eurostat, Tissue, Dementia, Medicine, Committee, Hormone, DSM-IV codes, Spinal cord, Pharmaceutical industry

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Key Points: 


Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Orphan designation: Allogeneic peripheral blood-derived haematopoietic stem and progenitor cells, regulatory T cells and conventional T cells Treatment in haematopoietic stem cell transplantation, 25/07/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, Haematopoiesis, European Commission, Allotransplantation, Committee, EMA, Pharmaceutical industry

EU/3/23/2815 - orphan designation for treatment in haematopoietic stem cell transplantation

Key Points: 
  • EU/3/23/2815 - orphan designation for treatment in haematopoietic stem cell transplantation
    Allogeneic peripheral blood-derived haematopoietic stem and progenitor cells, regulatory T cells and conventional T cells
    OrphanHuman
    Phortas GmbH
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

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

Human medicines European public assessment report (EPAR): Carmustine medac (previously Carmustine Obvius), carmustine, Date of authorisation: 18/07/2018, Revision: 10, Status: Authorised

Retrieved on: 
Friday, January 5, 2024
RNA, Medical Subject Headings, Brain, Marketing, Agency, Health, INN, Risk, Stomach, Ependymoma, ATC, Medulloblastoma, Radiation, International, Blood, Language, DNA, Literature, Hodgkin lymphoma, Chemotherapy, Cancer, Skin cancer, Glioblastoma, Astrocytoma, Haematopoiesis, European Medicines Agency, Neoplasm, Transplant, Select, Cell, Patient, Anatomy, IIA, Medical device

Human medicines European public assessment report (EPAR): Carmustine medac (previously Carmustine Obvius), carmustine, Date of authorisation: 18/07/2018, Revision: 10, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Carmustine medac (previously Carmustine Obvius), carmustine, Date of authorisation: 18/07/2018, Revision: 10, Status: Authorised

Human medicines European public assessment report (EPAR): Posaconazole Accord, posaconazole, Date of authorisation: 25/07/2019, Revision: 8, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Coccidioidomycosis, Patient, Medical Subject Headings, Spore, Marketing, Amphotericin B, Chromoblastomycosis, Failure, Acute leukemia, INN, Immune system, Canker, Stomach, Risk, Infection, Bone marrow, Itraconazole, ATC, Refractory, Fungus, B cell, International, Fluconazole, Disease, Blood, Language, Neutropenia, MDS, Haematopoiesis, European Medicines Agency, Aspergillosis, Mycetoma, Syndrome, Tablet, Select, HSCT, Anatomy, AML, Ergosterol, IIA, Skin, Medical device

Human medicines European public assessment report (EPAR): Posaconazole Accord, posaconazole, Date of authorisation: 25/07/2019, Revision: 8, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Posaconazole Accord, posaconazole, Date of authorisation: 25/07/2019, Revision: 8, Status: Authorised

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Retrieved on: 
Tuesday, January 2, 2024
SCD, Medical Subject Headings, Marketing, Febrile neutropenia, European Commission, Sickle cell disease, INN, Limited, Hematology, Physician, ATC, EMA, B cell, International, ATMP, HLA, Vomiting, Headache, Thalassemia, Nausea, Tissue, Disease, Civil service commission, HSC, CD34, Cas9, Haematopoiesis, Vertex Pharmaceuticals, Public, Gene, Transplant, Medication package insert, Volatile organic compound, Committee, Patient, Liver disease, TDT, HBF, Mouth ulcer, Cell, Anatomy, Medicine, Hemoglobinopathy, BCL11A, Health, CHMP

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Key Points: 


Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion