Experimental Gene Therapy for MPS I Gets Fast Track Designation

According to a story from PR Newswire, the biotechnology company REGENXBIO recently announced that the U.S. Food and Drug Administration (FDA) has granted its experimental gene therapy product RGX-111 Fast Track designation. REGENXBIO has developed a unique, proprietary NAV technology that is intended as a gene therapy delivery system that could hopefully have curative potential. RGX-111 utilizes this new approach and is in development for the treatment of mucopolysaccharidosis I (MPS I).
MPS I causes a buildup of glycosaminoglycans which occurs because of a deficiency in the enzyme alpha-L iduronidase, which normally breaks down these substances in the lysosome, a cellular organelle. It is a disorder caused by a genetic mutation. MPS I has several subtypes that vary by severity. Symptoms include developmental delays, coarsening of facial features, major skeletal deformities, joint stiffness, and carpal tunnel. Vision problems are also common, and even the milder forms can cause some learning impairments. Surgery and enzyme replacement therapy are the most common treatments. To learn more about MPS I, click here.

Fast Track designation is meant to facilitate a more rapid development and approval process for drugs that treat severe, life threatening diseases. Drugs that fulfill a severe unmet medical need or demonstrate major advantages in effectiveness or safety over current treatments may qualify. The FDA will work more closely with a company to aid in the drug’s development and a treatment with Fast Track designation may qualify for Priority Review.

RGX-111 would be one of the only treatments that could effectively address the symptoms of MPS I that affect the nervous system. The therapy is intended as a one-time treatment that will be delivered directly to the nervous system. Conventional enzyme replacement is unable to address the decline in mental function that can occur in patients with MPS I.

Hopefully RGX-111 will perform well in the clinical trials to come and be approved as an innovative new option for treatment that addresses symptoms that current treatments cannot manage.

Share this post

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