Specialty Pharma Journal Specialty, Precision & Technology
CRANBURY, N.J., Aug. 10, 2018 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Galafold™ (migalastat) 123 mg capsules. Galafold is an oral, precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha …
Tag Archives: Fabry disease
August, 2018
September, 2017
-
22 September
Avrobio Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease
CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program …
February, 2017
-
6 February
FDA Permits Marketing of First Newborn Screening System for Detection of Four, Rare Metabolic Disorders
The U.S. Food and Drug Administration today permitted marketing of the Seeker System for the screening of four, rare Lysosomal Storage Disorders (LSDs) in newborns. The Seeker system is designed to detect Mucopolysaccharidosis Type I(MPS I), Pompe, Gaucher and Fabry. It is the first newborn screening test permitted to be …
April, 2015
-
29 April
Genzyme’s Fabry Disease Drug Granted FDA Fast Track Status
US health regulators have granted a speedy review for Genzyme’s investigational drug for treatment of a rare lysosomal storage disorder. Genzyme, a Sanofi company, announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for the development of GZ/SAR402671, a new investigational oral substrate reduction therapy …
March, 2015
-
20 March
Amicus to Seek Accelerated US and European Approval of its Investigational Fabry Disease Drug
Amicus Therapeutics announced its plans to file for accelerated approval of its investigational drug for a rare, inherited disease, following meetings with regulators in the US and Europe. The company said that it has recently met with regulatory authorities in Europe and the US to discuss the approval pathways for …