Enzyme

Orphan designation: imatinib Treatment of pulmonary arterial hypertension, 21/06/2021 Positive

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Tuesday, April 9, 2024
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Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of pulmonary arterial hypertension (PAH) in the European Union on 21 June 2021.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1[...]

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Failure, Civil service commission, Enzyme, Protease, Disease, Infection, Inflammation, Patient, Committee, Knowledge, Netherton syndrome, Regulation, EMA, IRIS, Life, Protein, Kallikrein, European, Skin, COMP, Water, Safety, European Commission, MDC, Calcineurin, Moisturizer, Steroid, Syndrome, Medicine, Pharmaceutical industry

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive

Key Points: 


Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive

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5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn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: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Radiation, Brain, Ornithine decarboxylase, Enzyme, Growth, Weakness, Survival, Cell, Neuroblastoma, Disease, Patient, Tablet, Committee, Knowledge, Abdomen, Regulation, Child, Cancer, EMA, IRIS, Bone pain, Mouth, European, Polyamine, Fever, COMP, Sponsor, European Commission, Spine, Safety, Marketing, Limited, Medicine, Pharmaceutical industry

Orphan designation: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

Key Points: 


Orphan designation: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

Orphan designation: Veliparib Treatment of ovarian cancer, 17/12/2010 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Ovarian cancer, Enzyme, DNA repair, Abbott Laboratories, Survival, Disease, Patient, Committee, Knowledge, DNA, Cancer, Female, Regulation, Laboratory, EMA, IRIS, Ludwigshafen, Woman, PARP, European, COMP, Sponsor, European Commission, Fibroadenoma, Safety, Marketing, Medicine, Pharmaceutical industry

Orphan designation: Veliparib Treatment of ovarian cancer, 17/12/2010 Withdrawn

Key Points: 


Orphan designation: Veliparib Treatment of ovarian cancer, 17/12/2010 Withdrawn

Eisbach and Cancer Focus Fund Announce $4.5 Million Investment to Support First-in-Human Phase 1/2 Trial of EIS-12656 for Refractory Advanced Solid Tumors

Retrieved on: 
Thursday, March 21, 2024
Cancer, Principal, Enzyme, DNA repair, Biochemistry, Survival, Professor, Monroe Dunaway Anderson, Patient, Neoplasm, DNA, History, Breast, Prostate, Protein, University, MD Anderson Cancer Center, PARP, Therapy, ALC1, Chromatin, CSO, LP, Pharmaceutical industry

EIS-12656 is a small molecule designed to treat tumors that are refractory or resistant to PARP inhibitors.

Key Points: 
  • EIS-12656 is a small molecule designed to treat tumors that are refractory or resistant to PARP inhibitors.
  • By hindering DNA repair, PARP inhibitors cause tumor cell death, and they have been effective therapies for many ovarian, breast, prostate, and pancreatic cancers.
  • EIS-12656 inhibits the chromatin remodeling enzyme ALC1, which is critical to the DNA repair process associated with PARP activation.
  • EIS-12656 was developed by Eisbach to disrupt DNA-damage-dependent PARP activation at an early stage of the repair process.

CAMP4 Therapeutics Announces Dosing of First Participant in Phase 1 Clinical Study of CMP-CPS-001, a Potential First-in-Class Therapeutic for Urea Cycle Disorders

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Thursday, March 21, 2024
Disability, Gene, Enzyme, Seizure, Patient, DSM-IV codes, RNA, Messenger, Protein, Trial of the century, CPS1, UCD, Clinical trial, Therapy, Safety, Pharmacokinetics, Ammonia, Brain damage, Disorder, Blood, Pharmaceutical industry

“Most importantly, the clinical development of CMP-CPS-001 is a step toward potentially bringing a new, disease-modifying treatment to individuals living with UCDs.

Key Points: 
  • “Most importantly, the clinical development of CMP-CPS-001 is a step toward potentially bringing a new, disease-modifying treatment to individuals living with UCDs.
  • People with urea cycle disorders accumulate excessive ammonia in their blood, which may cause irreversible brain damage, disability, and seizures, and may be fatal.
  • CMP-CPS-001 targets carbamoyl phosphate synthetase 1 (CPS1), a key enzyme that catalyzes the first step of the urea cycle.
  • For more information about the Phase 1 clinical study of CMP-CPS-001, please visit clinicaltrials.gov ( NCT06247670 ).

DepYmed Receives Clearance from U.S. Food and Drug Administration to Initiate Phase 1 Clinical Trial for DPM-1003 for the Treatment of Rett Syndrome

Retrieved on: 
Tuesday, March 19, 2024
PTP, Obesity, Enzyme, DSM-IV codes, Patient, Cancer, IND, Clinical trial, Safety, Family, Rett syndrome, Food, Pharmaceutical industry

Today, DepYmed announces it has received clearance from the U.S. Food and Drug Administration to initiate its phase 1 clinical trial for DPM-1003 for the treatment of Rett Syndrome.

Key Points: 
  • Today, DepYmed announces it has received clearance from the U.S. Food and Drug Administration to initiate its phase 1 clinical trial for DPM-1003 for the treatment of Rett Syndrome.
  • “This is a foundational achievement for DepYmed.
  • This includes Rett Syndrome, cancer, metabolic disease and obesity, and Alzheimer’s Disease.
  • We look forward to beginning to assess the safety and tolerability of DPM-1003 and continue our development of this compound for Rett Syndrome and our other ongoing programs.”

Terragia Secures $6M to Develop Cost-Competitive, Low-Carbon Biofuel Technology

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Thursday, March 28, 2024
Chemicals, Plastics, Alternative Energy, Energy, Green Technology, Manufacturing, Sustainability, Alternative Vehicles, Fuels, Start-Up, University, Education, Professional Services, Environment, Biotechnology, Automotive, Health, Energy Centre, Ethanol, Biofuel, Research, Enzyme, EIP, National Science Foundation, Partnership, Chairperson, Distinguishing, Aviation, Dartmouth College, Carbon, Biology, Education minister, CO2, Ship, Fermentation, Multimedia, SAF, Renewable energy

Terragia Biofuel , a technology startup aiming to drive the next generation of biofuels, today announced it has raised a $6 million seed round led by Engine Ventures and Energy Impact Partners (EIP).

Key Points: 
  • Terragia Biofuel , a technology startup aiming to drive the next generation of biofuels, today announced it has raised a $6 million seed round led by Engine Ventures and Energy Impact Partners (EIP).
  • “Terragia has an exciting opportunity to succeed where others in the cellulosic biofuel industry have not.
  • Brief joins co-founders Lee Lynd, who transitioned to Chief Technology Officer, Bill Brady, Board Chair, and Chris Herring, Vice President of Technology Development.
  • We have a unique opportunity to make a transformational impact on climate outcomes, in addition to generating substantially higher revenues for biofuel producers.”

ELIQUENT Life Sciences Announces Appointment of Former FDA Senior Officials

Retrieved on: 
Thursday, March 21, 2024
Public Policy, Government, Biotechnology, Pharmaceutical, Health, FDA, Other Policy Issues, Medical Devices, College, Principal, Federation, Enzyme, Commissioner, Georgetown University, Regulatory affairs, BIO, National Cancer Institute, OBP, Biotechnology Innovation Organization, Laboratory, Quality, Divisional patent application, Biosimilar, Pennsylvania State University, Cell, Multimedia, Food, Pharmaceutical industry

ELIQUENT Life Sciences, a global regulatory consulting firm for the life sciences industry, today announced the appointment of John Taylor as head of the firm’s Quality & Compliance Practice and Michele Dougherty as head of the firm’s Regulatory Affairs Practice.

Key Points: 
  • ELIQUENT Life Sciences, a global regulatory consulting firm for the life sciences industry, today announced the appointment of John Taylor as head of the firm’s Quality & Compliance Practice and Michele Dougherty as head of the firm’s Regulatory Affairs Practice.
  • View the full release here: https://www.businesswire.com/news/home/20240321127987/en/
    John Taylor, Head of Quality & Compliance Practice, ELIQUENT Life Sciences (Photo: Business Wire)
    ELIQUENT’s integrated suite of services deliver the answers and solutions that global life sciences innovators need to gain and maintain market authorization for their products.
  • Most recently, John served for 10-years as President and Principal of Compliance & Regulatory Affairs at Greenleaf Health, a legacy brand of ELIQUENT Life Sciences.
  • A 10-year veteran of FDA, Michele’s career includes more than 15-years of regulatory experience across the product quality lifecycle, including her most recent role as President of DataRevive, a legacy brand of ELIQUENT Life Sciences.