FDA approval

FDA has asked Sarepta Therapeutics for additional information about dystrophin production and safety before it will consider accepting an application for accelerated approval of its exon-skipping drug

posted on April 15, 2013 - 6:27pm
The U.S. Food and Drug Administration (FDA) has said it will consider accepting an application for accelerated approval for eteplirsen, an experimental drug for Duchenne muscular dystrophy (DMD), after it reviews additional data from clinical trials of the drug.

How the FDA tries to balance urgent needs against real risks when evaluating new drugs

posted on April 1, 2013 - 9:25am
Quest Vol. 20, No. 2
Update (April 15, 2014): This story has been updated to reflect that there is now a new FDA office, the Office of Health and Constituent Affairs, whose job it is to serve as a liaison between the FDA and various outside stakeholders, including patient advocates and consumers. See Resources, below. ===================================================================================== Original...

A drug approved in Europe to treat Lambert-Eaton myasthenic syndrome is now being tested in the United States; clinical trial participants sought

posted on November 18, 2011 - 6:00am
BioMarin Pharmaceutical of Novato, Calif., is conducting a multicenter study of 3,4-diaminopyridine phosphate (3,4-DAP), also known as amifampridine phosphate, in adults with Lambert-Eaton myasthenic syndrome (LEMS). There are eight U.S. trial sites, with additional sites planned for France, Germany, Italy, Poland and Spain.

"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval

posted on June 21, 2010 - 4:25pm
Update (Aug. 4, 2014): The U.S. Food and Drug Administration (FDA) has expanded the approval of Lumizyme so that it can now be used to treat Pompe disease patients of all ages, including children younger than age 8. See FDA Expands Approval of Drug to Treat Pompe Disease to Patients of All Ages; Removes Risk Mitigation Strategy Requirements, Aug. 1, 2014.

The Food and Drug Administration has OK'd  the first U.S. treatment specifically for late-onset Pompe disease

posted on May 25, 2010 - 12:34pm
The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.
posted on May 1, 2006 - 11:59am
QUEST Vol. 13, No. 3
Genzyme seeks FDA ok for Pompe treatment