Inherited Lysosomal Storage Disorders Archives

Takeda Drops One Hunter Syndrome Therapeutic, Puts Faith in Another

Business, Central Nervous System Diseases, Clinical Trial Endpoints, Clinical Trials, Collaborations, Hunter Syndrome, Inherited Lysosomal Storage Disorders, Metachromatic Leukodystrophy (MLD), Primary Endpoints, Quarterly results, R&D, Secondary Endpoints, Takeda

In the company’s financial results for 2021 released on May 11, Takeda announced the decision to discontinue the development of TAK-609, a therapeutic for Hunter Syndrome. Additionally, Takeda is collaborating with JCR Pharmaceuticals in another therapeutic attempt for the condition.

May 11, 2022/by BioSpace

Astellas Gene Therapy for Pompe Disease Clears Important Safety Hurdle

Acid alpha-glucosidase (GAA) enzyme, Adeno-Associated Virus (AAV) Vectors, Astellas Pharma, BioSpace, Clinical Trials, Gene Therapy, Interim Data, Metabolic Diseases, Neutralizing Antibodies, Pompe disease, R&D, Safety Data

At WORLDSymposium, Astellas Pharma presented interim safety data from a Phase I/II study assessing the company’s AAV gene therapy for the rare, autosomal metabolic disease Pompe disease.

February 8, 2022/by BioSpace

Amicus Splits into Two as FDA Sets PDUFA for Pompe Disease Treatment

Pompe disease, Prescription Drug User Fee Act (PDUFA)

Amicus Therapeutics is launching a next-generation genetic medicine company called Caritas Therapeutics through a merger deal with ARYA Sciences Acquisition Corp IV, a special purpose acquisition company sponsored by Perceptive Advisors.

September 29, 2021/by Andrew Humphreys

FDA approves Nexviazyme for late-onset Pompe disease

Approvals, Clinical Trials, Enzyme Replacement Therapy (ERT), FDA, Late-onset Pompe disease, Muscle Disorders, Pompe disease, R&D, Sanofi

The U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients 1 year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.

August 6, 2021/by Andrew Humphreys

Phase III Trial Failure for Pompe Disease Drug

Analysts, Clinical Data, Clinical Trials, Double-Combination Therapies, Inherited Lysosomal Storage Disorders, Pompe disease, Pompe disease, Primary Endpoints, R&D, Recombinant human acid alpha-glucosidase (rhGAA) enzym

Amicus Therapeutics announced topline results from the Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease.

February 12, 2021/by Andrew Humphreys

Avrobio Gene Therapy Shows Promise in Lysosomal Diseases

Business, Clinical Data, Clinical Trials, Fabry Disease, Gaucher Disease, Gene Therapy, Inherited Lysosomal Storage Disorders, Kidneys, Nephropathic Cystinosis, R&D, Shares

Shares of Avrobio were up in trading after the company posted positive clinical data from gene therapy trials in three different rare lysosomal diseases: Fabry, Gaucher type 1 and cystinosis.

February 8, 2021/by BioSpace

Data presented at WORLDSymposium reinforces Sanofi’s robust rare disease pipeline

Acid alpha-glucosidase (GAA) enzyme, Business, Clinical Data, Enzyme Replacement Therapy (ERT), Muscle Cells, Pompe disease, Product Pipeline, Rare Diseases

Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.

February 2, 2021/by Andrew Humphreys

Fast Track, Breakthrough and Priority Reviews: Sanofi and ViiV

Acid alpha-glucosidase (GAA) enzyme, Biologics License Application (BLA), Breakthrough Therapy Designation, Bruton's Tyrosine Kinase (BTK) Inhibitors, Chronic Immune Thrombocytopenia (ITP), Clinical Trials, Enzyme Replacement Therapy (ERT), Fast Track Designation, FDA, HIV pre-exposure prophylaxis (PrEP), HIV Prevention, Muscle Disease, Orphan Drug Designation, PDUFA, Pompe disease, Priority Review Status, R&D, Sanofi

The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.

November 18, 2020/by BioSpace