Rare Pediatric Disease Designation Awarded to a Drug for MPS I

The United States Food and Drug Administration has awarded Rare Pediatric Disease Designation to a potential drug for Mucopolysaccharidosis type I. For more detailed information about this news, you can view the source press release at Business Wire by clicking here.

About Mucopolysaccharidosis Type 1

Mucopolysaccharidoses are a group of rare genetic disorders that develop because the body isn’t able to properly break down a type of carbohydrate called mucopolysaccharides, and, as a result, this substance can collect and cause damage to cells. There are several sub-types of mucopolysaccharidoses, of which mucopolysaccharidosis type I (MPS I) is one. MPS I varies a lot in severity between patients. For more information about MPS I, click here.

The Drug

A potential new drug to treat MPS1 is being developed by Immusoft Corporation. The drug is Iduronicrin genleukocel-T, which may support alpha-L-iduronidase (IDUA) to be delivered and expressed. IDUA is implicated in MPS1.

FDA Designations

This drug has previously been awarded Orphan Drug status by the FDA for the treatment of MPS I. More recently, Immusoft’s drug has been granted Rare Pediatric Disease Designation (RPDD). This status may be given to conditions that mainly affect children, and are believed to have less than 200,000 patients in the United States. Companies that receive this designation may receive a priority review voucher. This voucher may be used to reduce the FDA review period by half; from about one year to six months. The voucher can be sold or transferred, and, previously, they have been priced at more than $100 million.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email

What are your thoughts on being a rare disease advocate? Share your stories, thoughts, and hopes with the Patient Worthy community!

Close Menu