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Oral Drug for Fabry Disease Approved

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Oral Drug for Fabry Disease Approved

FDA gives nod to migalastat (Galafold)

MedpageToday

  • by Managing Editor, MedPage Today

WASHINGTON — The first oral treatment for Fabry disease in adults, migalastat (Galafold), was approved Friday by the FDA.

“Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research, in a press release. “Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme.”

To be eligible for migalastat, made by Amicus Therapeutics, patients must have a mutation in the alpha-galactosidase (GLA) gene determined to be responsive to the drug. Amicus, in its own announcement, said 348 GLA variants were approved as amenable to migalastat therapy.

Evidence of efficacy came from a 6-month, placebo-controlled trial involving 45 Fabry patients, which demonstrated that the drug reduced accumulation of globotriaosylceramide (GL-3) in renal blood vessels.

Because migalastat was approved in a fast-track review using this non-clinical efficacy endpoint, the FDA said Amicus must conduct another trial to confirm a clinical benefit.

In Fabry disease, patients lack the alpha-galactosidase enzyme, resulting in buildup of GL-3 in various tissues. Clinical manifestations include progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke, and early mortality, the FDA said.

Adverse effects of migalastat seen in the clinical program, involving a total of 139 patients, included headache, nasopharyngitis, urinary tract infection, nausea, and fever.

2018-10-08T00:00:00-0400

last updated

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Medpage Today

Oral Drug for Fabry Disease Approved

FDA gives nod to migalastat (Galafold)

MedpageToday

  • by Managing Editor, MedPage Today

WASHINGTON -- The first oral treatment for Fabry disease in adults, migalastat (Galafold), was approved Friday by the FDA.

"Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease," said Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research, in a press release. "Galafold differs from enzyme replacement in that it increases the activity of the body's deficient enzyme."

To be eligible for migalastat, made by Amicus Therapeutics, patients must have a mutation in the alpha-galactosidase (GLA) gene determined to be responsive to the drug. Amicus, in its own announcement, said 348 GLA variants were approved as amenable to migalastat therapy.

Evidence of efficacy came from a 6-month, placebo-controlled trial involving 45 Fabry patients, which demonstrated that the drug reduced accumulation of globotriaosylceramide (GL-3) in renal blood vessels.

Because migalastat was approved in a fast-track review using this non-clinical efficacy endpoint, the FDA said Amicus must conduct another trial to confirm a clinical benefit.

In Fabry disease, patients lack the alpha-galactosidase enzyme, resulting in buildup of GL-3 in various tissues. Clinical manifestations include progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke, and early mortality, the FDA said.

Adverse effects of migalastat seen in the clinical program, involving a total of 139 patients, included headache, nasopharyngitis, urinary tract infection, nausea, and fever.

2018-10-08T00:00:00-0400

last updated

Comments

Accessibility Statement

At MedPage Today, we are committed to ensuring that individuals with disabilities can access all of the content offered by MedPage Today through our website and other properties. If you are having trouble accessing www.medpagetoday.com, MedPageToday's mobile apps, please email legal@ziffdavis.com for assistance. Please put "ADA Inquiry" in the subject line of your email.



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Dr Irfanullah Khan Born: 15th July,1994 in Khagram,Dir Upper KPK Pakistan. Others names:Doctor Irfo,Peshoo Education:Pharm-D Scholar Graduated from Abasyn University Peshawar. Occupation:Clinical Pharmacist,Doctor,Entrepreneur. Home Town:Dir Upper Height: 6 feet. Website:Iukmedonline.com

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