FDA grants orphan drug designation to AAV1-FS344 for Inclusion Body Myositis

Release Date: 21-Nov-2016

US Food and Drug Association (FDA) announces orphan drug designation for Milo Biotechnology’s AAV1-FS344 for treatment of inclusion body myositis. AAV1-FS344 is a gene therapy delivered follistatin protein that increases muscle strength and function.

Currently, AAV1-FS344 is in its phase I/II trial in the Nationwide Children's Hospital in adult patients with sporadic inclusion body myositis which is funded by the foundations Parent Project Muscular Dystrophy and The Myositis Association.

Inclusion body myositis (IBM) is an adult onset myopathy causing severe and progressive muscle weakness. Most of the patients suffering from IBM are wheelchair bound within 10 years of diagnosis. Milo's AAV1-FS344 program is also being studied for other two rare diseases namely, Becker muscular dystrophy and Duchenne muscular dystrophy, for which the company received FDA approved orphan drug status in 2012.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com