Rare Genetic Diseases Archives - Page 2 of 2

Orphazyme’s Rare Lipid Disorder Drug Gets FDA Priority Review

Accepted NDA, Clinical Data, Clinical Trials, Fast Track Designation, FDA, FDA/Regulatory, Heat-Shock Proteins (HSPs), Lipid disorders, Niemann-Pick C Disease (NPC), Primary Endpoints, Priority Review Status, R&D, Rare Genetic Diseases, Secondary Endpoints

The U.S. Food and Drug Administration accepted Copenhagen, Denmark-based Orphazyme’s New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C.

September 16, 2020/by Andrew Humphreys

Alnylam’s rare genetic disorder drug priced at $575,000 per year

Acute Hepatic Porphyria (AHP), Analysts, Approvals, Drug Pricing, FDA, FDA/Regulatory, Gene Silencing, Peak sales estimates, Rare Genetic Diseases, RNA Interference (RNAi), Severe Pain

Alnylam Pharmaceuticals Inc. priced the company’s gene silencing drug to treat patients with a rare genetic disorder that can cause severe pain at $575,000 per year after receiving an early U.S. approval.

November 20, 2019/by Reuters Health

FDA Issues Draft Guidance for Fabry Disease Drugmakers

Biologicals, Draft Guidance, Drugs, Fabry disease, FDA, FDA/Regulatory, Rare Genetic Diseases

The U.S. FDA issued a new draft guidance for drugmakers aiming their resources at Fabry disease, a rare genetic disease.

August 9, 2019/by BioSpace

Amicus and Penn Broaden Gene Therapy Collaboration

Adeno-Associated Virus (AAV), Angelman Syndrome, Business, CDKL5 deficiency disorder (CDD), Collaborations, Fabry disease, Gene Therapy, Gene Transfer Technologies, Inherited Lysosomal Storage Disorders, Muscular Dystrophy, Mycopolysaccharidosis Type IIIA (MPS IIIA), Mycopolysaccharidosis Type IIIB (MPS IIIB), Myotonic Dystrophy, Niemann-Pick Type C (NPC), Pompe disease, Rett Syndrome, University of Pennsylvania (Penn)

Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.

May 29, 2019/by BioSpace

AskBio Snags $235 Million Investment to Drive Gene Therapy Development

Adeno-Associated Virus (AAV), Business, Clinical Studies, DNA, Fast Track Designation, FDA, Gene Therapy, Heart Failure, Pompe disease, Rare Genetic Diseases

Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.

April 11, 2019/by BioSpace

Pfizer buys stake in French gene therapy firm Vivet

Acquisitions, Analysts, Brain, Business, Gene Therapy, Hemophilia, Liver, Neurodegenerative Disorders, Organs, Rare Genetic Diseases, Wilson Disease, Wilson disease

Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.

March 20, 2019/by Reuters Health

EU panel approves drug for rare genetic disease

Blood, Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency (EMA), Familial Chylomicronemia Syndrome (FCS), Fat Accumulation, FDA, FDA/Regulatory, Inflammation, Pancreas, Platelet Count, Rare Genetic Diseases, Recommended For Approval

An advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.

March 1, 2019/by Reuters Health