PPAR

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Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn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: 
Thursday, 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Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn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Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn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: 
Tuesday, 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: 
Thursday, February 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: 
Thursday, February 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: 
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

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

Retrieved on: 
Wednesday, January 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: 
Wednesday, January 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: 
Thursday, December 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.

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: 
Thursday, December 7, 2023
Liver, UDCA, Chapter Two, Woman, FDA, MAA, Fatigue, Nausea, Primary biliary cholangitis, Inflammation, ADR, Patent, Liver disease, Diagnosis, AMF, CHMP, Ascending cholangitis, UCD, Business, ACLF, PDUFA, Itch, Risk, New Drug Application, OA, Common, EASL, MASH, Liver failure, Safety, Ammonia, Elafibranor, Therapy, Epidemiology, History, Patient, Cholangiocarcinoma, Food, Health care, EMA, Ursodeoxycholic acid, MHRA, Cirrhosis, Medicine, NTZ, EVP, Cholestasis, NASH, Research, Diarrhea, Bile, NDA, PBC, Abdominal pain, Clinical trial, European Medicines Agency, IPN, PPAR, P10, CCA, Fibrosis, Pharmaceutical industry

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.

Key Points: 
  • 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 trial enrolled 161 patients who were randomized 2:1 to receive either elafibranor 80mg once daily or placebo.
  • ELATIVE also investigated the effect of treatment with elafibranor on pruritus (severe itch), a significant symptom burden amongst people living with PBC.
  • Findings from the secondary endpoint using the PBC Worst Itch NRS score, showed a reduction in pruritis for elafibranor, which was not statistically significant.

Reneo Pharmaceuticals Reports Third Quarter 2023 Financial Results

Retrieved on: 
Monday, November 13, 2023
STRIDE, Three Months, DNA, Therapy, Treatment, PMM, Investment, PPAR, Patient, Research, G&A, R, Patent, A-DNA, Pharmaceutical industry, Nursing, Disease

IRVINE, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today reported financial results for the third quarter ended September 30, 2023 and provided a business update.

Key Points: 
  • IRVINE, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today reported financial results for the third quarter ended September 30, 2023 and provided a business update.
  • “We are looking forward to sharing topline results of our pivotal STRIDE study in December,” said Gregory J. Flesher, President and CEO of Reneo Pharmaceuticals.
  • Research and development (R&D) expenses were $13.6 million during the third quarter of 2023, compared to $9.9 million for the same period in 2022.
  • General and administrative (G&A) expenses were $7.3 million during the third quarter of 2023, compared to $3.9 million for the same period in 2022.