FDA approves treatment for disease affecting fewer than 150 worldwide

[Webdog]

http://www.raredr.com/news/fda-approves-mepsevii

FDA Approves MPS VII Treatment
NOVEMBER 15, 2017
Mathew Shanley

Today, the U.S. Food and Drug Administration (FDA) approved Mepsevii (vestronidase alfa-vjbk) for use in pediatric and adult patients with mucopolysaccharidosis type VII (MPS VII), often referred to as Sly syndrome.

MPS VII is a hereditary metabolic disorder resulting from the deficiency of an enzyme called beta-glucuronidase, which is required for the breakdown of glycosaminoglycans (GAGs) including: dermatan sulfate, chondroitin sulfate, and heparin sulfate. Mepsevii is an enzyme replacement therapy that operates by replacing the missing enzyme.

The condition affects less than 150 patients worldwide and is characterized by an array of skeletal abnormalities, including short stature.

“This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER) in the official FDA press release. “Prior to today’s approval, patients with this rare, inherited condition had no approved treatment options.”

"'This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,' said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER)"

It's great to see the FDA focussing on exceedingly rare disorders, but where is the commitment from the FDA to approve treatments for common diseases as well?

Fewer than 150 patients worldwide (and perhaps fewer than 7 patients in the United States) might benefit from the FDA approval of Mepsevii.

What about the 20,000,000 worldwide ME/CFS sufferers still with no approved treatment? Where is the FDA commitment to them (us)?

Priorities seem out of balance at the FDA.

 

[ivorin]

Amen

 

[MErmaid]

Wow, I am guessing their advocacy group and scientific advisory board must be stellar in order to gain funding and get Pharma onboard.

 

[Alvin]

The FDA did not invent this drug, they just allowed it for human use in the US

 

[MErmaid]

The FDA did not invent this drug, they just allowed it for human use in the US

Yes, exactly! Ultragenyx Pharmaceutical Inc invented the new drug, and the FDA approved its use for US consumption.

http://www.ultragenyx.com/

 

[Sly Saint]

"Sly syndrome"

 

[Webdog]

The FDA did not invent this drug, they just allowed it for human use in the US

The FDA also affirmed they have a "commitment to making treatments available to patients with rare diseases". (ETA: Which is a noble goal, as many rare disease have no treatment yet.)

What about an FDA commitment for non-rare diseases that don't yet have a treatment?

 

[Alvin]

"Sly syndrome"

What coincidence

The FDA also affirmed they have a "commitment to making treatments available to patients with rare diseases". (ETA: Which is a noble goal, as many rare disease have no treatment yet.)

What about an FDA commitment for non-rare diseases that don't yet have a treatment?

You'll get no argument from me, but they are going to need a drug to approve and the documentation they require. The FDA is not an evil beast, in fact they respond quite well to industry malfeasance in getting drugs approved as long as its subtle which is not actually a good thing...