Lipodystrophy

Chiesi Global Rare Diseases Announces Health Canada Approval of MYALEPTA™ (metreleptin for injection) for the Treatment of Patients with Lipodystrophy

Retrieved on: 
Monday, February 5, 2024
DSM-IV codes, Metabolic disorder, Acquired generalized lipodystrophy, Research, LD, Patient, Congenital generalized lipodystrophy, Western University, Diet, Lipodystrophy, Hope, Health Canada, Barraquer–Simons syndrome, Pharmaceutical industry

BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, is pleased to announce the Health Canada approval of MYALEPTA™ (metreleptin for injection).

Key Points: 
  • BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, is pleased to announce the Health Canada approval of MYALEPTA™ (metreleptin for injection).
  • As an adjunct to diet, MYALEPTA is indicated as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children two years of age and above.
  • MYALEPTA was developed by Amryt Pharma, which was acquired in April 2023 by the Chiesi Group, an international, research focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare disease and specialty care.
  • “Being a lipodystrophy patient myself and having lost my mother and sister to complications from this devastating disorder, our work is helping to advocate on behalf of patients and caregivers.”

Theratechnologies Receives Complete Response Letter (CRL) from the FDA for the F8 Formulation of Tesamorelin sBLA

Retrieved on: 
Wednesday, January 24, 2024
Senior, CMC, CRL, Chemistry, Symantec Endpoint Protection, F4, Lipodystrophy, Risk, Connective tissue, Food, TSX, FDA, HIV, Microbiology

The Company will address the FDA’s request and intends to pursue approval of this newer formulation of tesamorelin.

Key Points: 
  • The Company will address the FDA’s request and intends to pursue approval of this newer formulation of tesamorelin.
  • The questions outlined in the CRL are largely related to chemistry, manufacturing and controls (CMC) concerning the microbiology, assays, impurities and stability for both the lyophilized product and the final reconstituted drug product.
  • In addition, the FDA requested further information to understand the potential impact of the proposed formulation on immunogenicity risk.
  • “While we are disappointed to receive a Complete Response Letter from the FDA for the F8 formulation of tesamorelin containing questions that were not raised during the review process, we plan to address these new comments as swiftly as possible,” said Christian Marsolais, Ph.D., Senior Vice President and Chief Medical Officer at Theratechnologies.

Catalyst Pharmaceuticals Appoints Richard J. Daly as Chief Executive Officer

Retrieved on: 
Thursday, October 19, 2023
CORAL, AstraZeneca, SVKM's NMIMS, TAP Pharmaceuticals, Rich, Catalyst (nonprofit organization), Notre Dame, Rare, Biotechnology, Takeda, Laboratory, Neuralstem, Inc., Diabetes, MBA, Catalyst Pharmaceuticals, Abbott Laboratories, Senior, Executive, University, Kellogg School, Metreleptin, Culture, Patient, Marketing, Bachelor of Science in Human Biology, Central nervous system, Management, Pharmaceutical industry, Science, Catalysis, Lipodystrophy

Mr. Daly is a seasoned pharmaceutical executive bringing more than three decades of experience to Catalyst.

Key Points: 
  • Mr. Daly is a seasoned pharmaceutical executive bringing more than three decades of experience to Catalyst.
  • "Today's announcement paves the way for a seamless transition for the new CEO at Catalyst.
  • "I am honored to serve as Catalyst's new CEO at such a pivotal point in the Company's evolution," said Richard J. Daly.
  • Prior to his appointment at Catalyst, Mr. Daly served as President of CARsgen Therapeutics Corporation since January 2022.

Theratechnologies Receives January 2024 PDUFA Goal Date for Tesamorelin F8 Formulation sBLA

Retrieved on: 
Wednesday, October 4, 2023
Connective tissue, Lipodystrophy, HIV, Prescription Drug User Fee Act, PDUFA, TSX, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, News, Food, F4, Medical device, Pharmaceutical industry

MONTREAL, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 22, 2024 to the Company’s supplemental Biologics License Application (sBLA) of the F8 formulation of tesamorelin.

Key Points: 
  • MONTREAL, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 22, 2024 to the Company’s supplemental Biologics License Application (sBLA) of the F8 formulation of tesamorelin.
  • Tesamorelin is the only medication approved in the U.S. for the reduction of excess abdominal fat in adults with HIV who have lipodystrophy.
  • Once approved, the F8 formulation is set to replace the current F4 formulation, which is sold in the U.S. under the trade name EGRIFTA SV®.
  • The proposed proprietary name for the F8 formulation of tesamorelin, EGRIFTA MDVTM, is already under review by the FDA.

Theratechnologies Submits Tesamorelin F8 Formulation sBLA for FDA Review

Retrieved on: 
Monday, September 25, 2023
Connective tissue, Lipodystrophy, Senior, HIV, Ageing, F1, Patient, MDV, PDUFA, TSX, FDA, Letter, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, F4, Pharmaceutical industry

MONTREAL, Sept. 25, 2023 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the Company has filed a supplemental Biologics License Application (sBLA) for the F8 formulation of tesamorelin to the U.S. Food and Drug Administration (FDA) for review.

Key Points: 
  • MONTREAL, Sept. 25, 2023 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the Company has filed a supplemental Biologics License Application (sBLA) for the F8 formulation of tesamorelin to the U.S. Food and Drug Administration (FDA) for review.
  • Tesamorelin is the only medication approved in the U.S. for the reduction of excess abdominal fat in adults with HIV who have lipodystrophy.
  • Pharmacokinetic studies have shown bioequivalence of the F8 formulation to the original F1 formulation of tesamorelin (previously sold under the trade name EGRIFTA®).
  • The proposed proprietary name for the F8 formulation, EGRIFTA MDVTM, is already under review by the FDA.

World Orphan Drug Alliance Announces Agreement with Theratechnologies for the Distribution of EGRIFTA SV

Retrieved on: 
Thursday, January 5, 2023
CEE, WODA, HIV, Degenerative disease, LATAM, Lipodystrophy, Tissue (biology), MENAT, Central and Eastern Europe, Pharmaceutical industry, Airline, Medis

Under the agreement, the World Orphan Drug Alliance (WODA) has obtained exclusive distribution rights to EGRIFTA SV® (tesamorelin for injection) to be made available under a Named Patient Program.

Key Points: 
  • Under the agreement, the World Orphan Drug Alliance (WODA) has obtained exclusive distribution rights to EGRIFTA SV® (tesamorelin for injection) to be made available under a Named Patient Program.
  • The multi-territorial agreement covers 41 countries in the regions of Latin America (LATAM), Middle East, North Africa & Turkey (MENAT) and Central & Eastern Europe (CEE).
  • DUBAI, United Arab Emirates and SÃO PAULO, Brazil and LJUBLJANA, Slovenia, Jan. 05, 2023 (GLOBE NEWSWIRE) -- The World Orphan Drug Alliance (WODA), a global alliance of commercial distributors focused on providing access to treatments for rare diseases and specialty medicines in complex and underserved markets around the world, today announced a long-term, multi-territorial distribution agreement with Theratechnologies Inc. ("Theratechnologies") for EGRIFTA SV® in 41 countries.
  • "We are excited to partner with Theratechnologies and support them in addressing markets outside of North America.

Amryt Announces New Patent for Mycapssa®

Retrieved on: 
Wednesday, November 30, 2022
COVID-19, Acromegaly, Birth, Lipodystrophy, Environment, Metreleptin, Degenerative disease, Lomitapide, EMA, Patient, GL, Octreotide, European, Diet, CHMP, Patent, Orange Book, Investment, FDA, Pharmaceutical industry, Dietary supplement, EU

The USPTO has issued to Amryt US Patent No.

Key Points: 
  • The USPTO has issued to Amryt US Patent No.
  • With the addition of this patent, Amryt has ten Orange Book-listed patents for Mycapssa with patent protection through December 2040.
  • Amryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases.
  • Mycapssa has also been submitted to the EMA and has received a positive opinion by the CHMP recommending the approval of Mycapssa in theEuropean Union(EU).

OrsoBio Acquires Phase 2-Ready, Selective ACC2 Inhibitor from Shionogi & Co., Ltd. for the Treatment of Type 2 Diabetes

Retrieved on: 
Wednesday, November 2, 2022
Health, Diabetes, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, MACP, Liver, Heart, MD, Yale school, Health, Pain, Insulin resistance, Doctor of Philosophy, Shionogi, Hypertriglyceridemia, Type 2 diabetes, CNS, MACE, Program, Infection, Respiration (physiology), LDL, Thrombocytopenia, Yale School of Medicine, Safety, Institute of Liver and Biliary Sciences, FRCP, Cirrhosis, Fatty liver disease, Foundation, Abstract, Disorder, FAO, Pharmacokinetics, Patient, Organic acid, Poster, Mitochondrion, Gilead Sciences, Lipodystrophy, Dyslipidemia, Sarcopenia, Pharmaceutical industry, Medicine

TLC-3595 also has potential for treating other conditions characterized by impaired FAO, including cirrhosis with sarcopenia and genetic, long-chain FAO disorders.

Key Points: 
  • TLC-3595 also has potential for treating other conditions characterized by impaired FAO, including cirrhosis with sarcopenia and genetic, long-chain FAO disorders.
  • TLC-3595 treatment led to improvements in LDL and total cholesterol, confirming the compounds pharmacodynamic effects, and its pharmacokinetics support once daily oral administration.
  • Insulin resistance is the major predisposing factor in the pathogenesis of type 2 diabetes.
  • Selectively inhibiting ACC2 with TLC-3595 offers promise as a therapy for type 2 diabetes and other disorders characterized by impaired FAO.

OrsoBio Acquires ACMSD Inhibitor Program to Address Mitochondrial Dysfunction from Mitobridge, an Astellas Company

Retrieved on: 
Wednesday, November 2, 2022
Health, Diabetes, Clinical Trials, General Health, Pharmaceutical, Biotechnology, Pharmacology, Oxidative stress, Liver, MD, Steatosis, Lipotoxicity, Acquisition, Hepatocyte, Mouse, Doctor of Philosophy, Degenerative disease, Complementarity, Program, IPSC, Respiration (physiology), Chemistry, Institute of Liver and Biliary Sciences, Maintenance, Fatty liver disease, Rail profile, Foundation, Abstract, De novo synthesis, NAD, Patient, Poster, Biology, De novo, Gilead Sciences, Tryptophan, Dyslipidemia, Lipodystrophy, Pharmaceutical industry

OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced its acquisition of an ACMSD inhibitor program from Mitobridge, an Astellas Company.

Key Points: 
  • OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced its acquisition of an ACMSD inhibitor program from Mitobridge, an Astellas Company.
  • The program includes a range of proprietary compounds being evaluated for their potential to address mitochondrial dysfunction in metabolic and inflammatory liver and kidney disorders.
  • ACMSD is highly expressed in the liver and kidneys and NAD+ is reduced in patients with advanced hepatic and renal disease.
  • TLC-065 and other compounds within the ACMSD inhibitor portfolio are being studied for their potential to replenish NAD+ and improve mitochondrial function.

OrsoBio Initiates IND-Enabling Activities for Liver-Targeted Mitochondrial Protonophore TLC-6740 for the Treatment of Severe Metabolic Disorders

Retrieved on: 
Wednesday, November 2, 2022
Health, Diabetes, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Weight loss, MACP, Liver, MD, Yale school, Plasma, Hepatocyte, Methanol toxicity, Insulin resistance, Doctor of Philosophy, Obesity, MACE, Intellectual property, Respiration (physiology), AMPK, Yale School of Medicine, Dyslipidemia, FRCP, NASH, Institute of Liver and Biliary Sciences, Fatty liver disease, Foundation, Abstract, Primate, Patient, Lipodystrophy, De novo, Gilead Sciences, TCA, Diabetes, Yale University, Pharmaceutical industry, Medicine

OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced initiation of IND-enabling studies of TLC-6740, a liver-targeted mitochondrial protonophore.

Key Points: 
  • OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced initiation of IND-enabling studies of TLC-6740, a liver-targeted mitochondrial protonophore.
  • TLC-6740 will be initially advanced for the treatment of patients with severe lipodystrophies with a first-in-human study planned to initiate in 2023.
  • TLC-6740 is a novel, liver-targeted mitochondrial protonophore in development for the treatment of severe lipodystrophies and other metabolic disorders.
  • OrsoBio, Inc. is a privately held, clinical-stage biopharmaceutical company dedicated to developing therapies to treat severe metabolic disorders, including type 2 diabetes, severe dyslipidemias, lipodystrophies, and nonalcoholic steatohepatitis.