PPAR

Gilead’s Seladelpar Demonstrated a Sustained and Consistent Long-Term Efficacy and Safety Profile in Primary Biliary Cholangitis

Retrieved on: 
Mittwoch, Juni 5, 2024
Biotechnology, Hospitals, Health, Pharmaceutical, Clinical Trials, Biomarker, Inflammation, Cirrhosis, Liver disease, Itch, Associate, PPAR, Doctor of Philosophy, Quality of life, Food, Gilead Sciences, Alkaline phosphatase, Science, MD, NDA, TB, European Medicines Agency, Safety, Therapy, Female, UDCA, NRS, Elastography, Ursodeoxycholic acid, Month, Bachelor of Science, MHRA, Master of Science, Cholestasis, Interim, University of Birmingham, ULN, MBBS, Acquisition, FDA, University, Congress, MRCP, Liver injury, EMA, ALP, CP, EASL, PBC, Primary biliary cholangitis, Pharmaceutical industry

“The data presented at EASL further support the sustained efficacy and safety profile of seladelpar observed across its robust development program, including a capacity to normalize ALP values for many of the people studied with PBC.

Key Points: 
  • “The data presented at EASL further support the sustained efficacy and safety profile of seladelpar observed across its robust development program, including a capacity to normalize ALP values for many of the people studied with PBC.
  • Participants received 10 mg seladelpar, once daily, for up to 155 weeks in the current analysis of the ASSURE cohort.
  • “The long-term efficacy and safety interim results from ASSURE demonstrate that seladelpar may meaningfully raise the bar in PBC.
  • Medicines and Healthcare Products Regulatory Agency (MHRA) and the European Medicines Agency (EMA) have also accepted seladelpar for review.

Investigational Seladelpar Demonstrates Significant Improvements in Liver Disease Progression and Reduced Itching in Primary Biliary Cholangitis

Retrieved on: 
Samstag, Mai 18, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Hospitals, Clinical Trials, Cholestasis, Itch, Health care, Seladelpar, Patient, Primary biliary cholangitis, University of Miami, Month, Comorbidity, ALT, Ursodeoxycholic acid, Liver disease, GGT, Food, Liver, NDA, European Medicines Agency, University, FDA, Therapy, Safety, Conference, NRS, PBC, ULN, Gilead Sciences, EMA, Child, Cirrhosis, Biomarker, Acquisition, MHRA, Alkaline phosphatase, Data analysis, UDCA, Observation, TB, Liver injury, Gastrointestinal disease, Inflammation, PPAR, Quality of life, ALP, Female, Pharmaceutical industry

Additional findings demonstrate that seladelpar can help reduce pruritus (itch) in people living with primary biliary cholangitis (PBC).

Key Points: 
  • Additional findings demonstrate that seladelpar can help reduce pruritus (itch) in people living with primary biliary cholangitis (PBC).
  • This data will be shared in an oral presentation during the Presidential Plenary of the Digestive Disease Week® 2024 Conference in Washington, DC.
  • Enrolled patients received an open-label oral dose of 10 mg seladelpar once daily, with the majority (97%) also receiving UDCA treatment.
  • Seladelpar has also been accepted for review by the UK Medicines and Healthcare products Regulatory Agency (MHRA) and the European Medicines Agency (EMA).

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Retrieved on: 
Donnerstag, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Atrophy, Brain, Enzyme, DSM-IV codes, Cell, Patient, Committee, Knowledge, Mitochondrion, PPAR, Regulation, Fatigue, EMA, IRIS, Eye, Diet, European, Retina, COMP, Heart, Sponsor, European Commission, Hypoglycemia, Safety, LCHAD, Dicarboxylic acid, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Cardiol Therapeutics Announces Year-End 2023 Update on Operations

Retrieved on: 
Dienstag, April 2, 2024
Hypertrophy, Heart, CRDL, Patient, Mayo Clinic, PPAR, Agent handling, Cleveland Clinic, Fibrosis, European, Therapy, Myocarditis, Analysis, American Heart Association, Human, Massachusetts General Hospital, Pericarditis, MMT, Heart failure, American College, Safety, ODD, Cardiology, Clinical professor, Disease, Pericardium, Research, World Heart Federation, Management, University, Death, Pericardial effusion, Maverick, Reactive oxygen species, TSX, Priority review, FDA, Conversation, Financial statement, Inflammation, Fast Track, Calcium, Wound healing, API, Pharmaceutical industry

"Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.

Key Points: 
  • "Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.
  • In January 2023, Cardiol announced the first patient had been enrolled in the Company's Phase II open-label pilot study ("MAvERIC-Pilot") investigating the tolerance, safety, and efficacy of CardiolRx™ in patients with recurrent pericarditis.
  • In November 2023, Cardiol announced that it had exceeded 50% of the patient enrollment target for the MAvERIC-Pilot study.
  • In January 2024, Cardiol announced that ARCHER had exceeded 50% patient enrollment and was progressing ahead of the original study timeline.

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

Retrieved on: 
Donnerstag, Februar 29, 2024
Rare disease, CALD, Central nervous system, Adrenoleukodystrophy, Committee, Leukodystrophy, Hospital, X-ALD, PPAR, Therapy, CHMP, CNS, European Medicines Agency, Patient, Disease, Pharmaceutical industry

The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.

Key Points: 
  • The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.
  • Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated.
  • We hope that this film will raise awareness and understanding of the condition."
  • Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD.

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

Retrieved on: 
Donnerstag, Februar 29, 2024
Rare disease, CALD, Central nervous system, Adrenoleukodystrophy, Committee, Leukodystrophy, Hospital, X-ALD, PPAR, Therapy, CHMP, CNS, European Medicines Agency, Patient, Disease, Pharmaceutical industry

The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.

Key Points: 
  • The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.
  • Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated.
  • We hope that this film will raise awareness and understanding of the condition."
  • Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD.

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: 
Sonntag, Februar 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

Non-Alcoholic Steatohepatitis Treatment Drug Market In Japan is Expected to Showcase Impressive Growth by 2032 | DelveInsight

Retrieved on: 
Mittwoch, Januar 17, 2024
Obesity, Merck & Co., MRI, LAS, Weight loss, Fatty liver disease, Type 2 diabetes, Magnetic resonance imaging, Insulin resistance, NASH, FFAR4, Liver, Liver disease, Diabetes, Alcoholic liver disease, Therapy, Hypertension, PPAR, Eli Lilly and Company, Gilead Sciences, Chronic pain, Disease, Inflammation, FASN, AstraZeneca, Novo Nordisk, FXR, Prevalence, CCR5, FGF, Physical examination, Weakness, Bronchial thermoplasty, Cilofexor, Metabolic syndrome, Mitsubishi Tanabe Pharma, Development, NAFLD, Risk, Fibrosis, Fatigue, Dyslipidemia, USD, Non-alcoholic fatty liver disease, Merck, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Non-Alcoholic Steatohepatitis Market Insights report includes a comprehensive understanding of current treatment practices, non-alcoholic steatohepatitis emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into Japan.

Key Points: 
  • LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Non-Alcoholic Steatohepatitis Market Insights report includes a comprehensive understanding of current treatment practices, non-alcoholic steatohepatitis emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into Japan.
  • According to DelveInsight's analysis, the market size for NASH reached USD 200 million in 2022 in Japan and is expected to grow with a significant CAGR by 2032.
  • DelveInsight's analysis reveals that the overall diagnosed prevalent population of NASH in Japan was reported as 2.5 million in 2022.
  • Among the forecasted emerging therapies, MGL-3196 (Resmetirom) is expected to capture the highest market in Japan by 2032.

Non-Alcoholic Steatohepatitis Treatment Drug Market In Japan is Expected to Showcase Impressive Growth by 2032 | DelveInsight

Retrieved on: 
Mittwoch, Januar 17, 2024
Obesity, Merck & Co., MRI, LAS, Weight loss, Fatty liver disease, Type 2 diabetes, Magnetic resonance imaging, Insulin resistance, NASH, FFAR4, Liver, Liver disease, Diabetes, Alcoholic liver disease, Therapy, Hypertension, PPAR, Eli Lilly and Company, Gilead Sciences, Chronic pain, Disease, Inflammation, FASN, AstraZeneca, Novo Nordisk, FXR, Prevalence, CCR5, FGF, Physical examination, Weakness, Bronchial thermoplasty, Cilofexor, Metabolic syndrome, Mitsubishi Tanabe Pharma, Development, NAFLD, Risk, Fibrosis, Fatigue, Dyslipidemia, USD, Non-alcoholic fatty liver disease, Merck, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Non-Alcoholic Steatohepatitis Market Insights report includes a comprehensive understanding of current treatment practices, non-alcoholic steatohepatitis emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into Japan.

Key Points: 
  • LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Non-Alcoholic Steatohepatitis Market Insights report includes a comprehensive understanding of current treatment practices, non-alcoholic steatohepatitis emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into Japan.
  • According to DelveInsight's analysis, the market size for NASH reached USD 200 million in 2022 in Japan and is expected to grow with a significant CAGR by 2032.
  • DelveInsight's analysis reveals that the overall diagnosed prevalent population of NASH in Japan was reported as 2.5 million in 2022.
  • Among the forecasted emerging therapies, MGL-3196 (Resmetirom) is expected to capture the highest market in Japan by 2032.

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

Retrieved on: 
Donnerstag, Dezember 7, 2023
Liver, Ascending cholangitis, Ipsen, Food, Itch, Health care, Woman, FDA, EMA, MAA, PDUFA, Primary biliary cholangitis, Bile, Fatigue, NDA, Elafibranor, MHRA, ADR, New Drug Application, European Medicines Agency, Common, IPN, PPAR, Liver failure, Patient, Medicine, EVP, PBC, CHMP, Pharmaceutical industry

New Drug Application granted priority review with PDUFA date set for June 10, 2024

Key Points: 
  • New Drug Application granted priority review with PDUFA date set for June 10, 2024
    Investigational elafibranor is the first novel second-line treatment for primary biliary cholangitis (PBC) to be filed in E.U.
  • An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.
  • The target FDA PDUFA date under priority review is June 10, 2024.
  • “This is a condition where many patients are living with worsening disease and debilitating symptoms despite being on treatment.