Acquired generalized 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

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.”

OrsoBio Announces Presentation of Five Abstracts at AASLD’s The Liver Meeting® 2022 Highlighting Programs for Severe Metabolic Disorders

Retrieved on: 
Thursday, November 3, 2022

OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced the presentation of five abstracts from the companys programs at AASLDs The Liver Meeting from November 4-8.

Key Points: 
  • OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced the presentation of five abstracts from the companys programs at AASLDs The Liver Meeting from November 4-8.
  • There remains tremendous unmet medical need for patients with severe metabolic disorders, said Mani Subramanian, MD, PhD, Chief Executive Officer of OrsoBio.
  • I am truly excited about translating the data on OrsoBios compounds in HLOs derived from genetic subsets of patients to clinical studies in patients with severe metabolic disorders.
  • The following abstracts will be presented as poster presentations at The Liver Meeting, in Washington, D.C., November 4-8, 2022.

OrsoBio Announces Acquisition of LXR Inverse Agonist Program for Treatment of Severe Dyslipidemias from Phenex Pharmaceuticals

Retrieved on: 
Wednesday, November 2, 2022

OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced its acquisition of assets relating to the Liver X Receptor (LXR) inverse agonist program from Phenex Pharmaceuticals, including lead candidate TLC-2716 and associated intellectual property.

Key Points: 
  • OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced its acquisition of assets relating to the Liver X Receptor (LXR) inverse agonist program from Phenex Pharmaceuticals, including lead candidate TLC-2716 and associated intellectual property.
  • Our Phase 1 study is progressing well, and we look forward to the continued development of TLC-2716 for the treatment of severe dyslipidemias in 2023.
  • I am truly excited that OrsoBio is progressing TLC-2716 into the clinic to unlock the potential of this first-in-class compound for patients with severe dyslipidemias.
  • 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.

OrsoBio Announces Exclusive License to Intellectual Property Relating to Mitochondrial Protonophores for the Treatment of Severe Metabolic Disorders

Retrieved on: 
Wednesday, November 2, 2022

OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced that it has acquired exclusive worldwide rights to intellectual property from Yale University to enable the development of novel mitochondrial protonophores.

Key Points: 
  • OrsoBio, Inc., a clinical-stage biopharmaceutical company developing treatments for severe metabolic disorders, today announced that it has acquired exclusive worldwide rights to intellectual property from Yale University to enable the development of novel mitochondrial protonophores.
  • The SBIR grant will enable OrsoBio to advance TLC-1235 as a potential treatment to improve insulin sensitivity, NASH, and dyslipidemia in patients with severe lipodystrophies.
  • TLC-1235 is a novel, controlled-release mitochondrial protonophore that has been shown to be both safe and effective in extensive preclinical models.
  • 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.

Amryt Receives Reimbursement Approval from the Dutch Ministry of Health for Myalepta® (metreleptin)

Retrieved on: 
Monday, October 17, 2022

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.

Key Points: 
  • 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.
  • Amryt comprises a strong and growing portfolio of commercial and development assets.
  • Amryt also intends to develop oral medications that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE) technology platform.
  • Such forward-looking statements reflect the Companys current beliefs and assumptions and are based on information currently available to management.

Global Lipodystrophy Syndrome (LS) Pipeline Overview 2020 - Therapeutic Assessment, Product Profiles, Unmet Needs, Key Companies

Retrieved on: 
Tuesday, February 18, 2020

Lipodystrophy Syndrome (LS) Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Lipodystrophy Syndrome (LS) market.

Key Points: 
  • Lipodystrophy Syndrome (LS) Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Lipodystrophy Syndrome (LS) market.
  • A detailed picture of the Lipodystrophy Syndrome (LS) pipeline landscape is provided, which includes the disease overview and Lipodystrophy Syndrome (LS) treatment guidelines.
  • The assessment part of the report embraces in-depth Lipodystrophy Syndrome (LS) commercial assessment and clinical assessment of the Lipodystrophy Syndrome (LS) pipeline products from the pre-clinical developmental phase to the marketed phase.
  • The Lipodystrophy Syndrome (LS) report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Lipodystrophy Syndrome (LS) across the complete product development cycle, including all clinical and nonclinical stages.