Milo Biotechnology Announces FDA Orphan Drug Designation For Treatment Of Inclusion Body Myositis

CLEVELAND, OH and COLUMBUS, OH--(Marketwired - October 04, 2016) - Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of inclusion body myositis. AAV1-FS344 is a gene therapy-delivered follistatin protein that increases muscle strength and function.

The program is currently in a Phase I/II trial at Nationwide Children's Hospital in adult patients with sporadic inclusion body myositis, clinical work which was funded by the foundations Parent Project Muscular Dystrophy and The Myositis Association. In May 2016, initial data from the trial was presented, demonstrating initial safety and improvement in function in 6 patients. "We're very encouraged by the results to date in inclusion body myositis, which form the foundation for larger pivotal studies in this intractable disease," said Milo CEO Al Hawkins.

Inclusion body myositis is an adult onset myopathy that causes severe and progressive muscle weakness; many patients are wheelchair bound within 10 years of diagnosis. Milo's AAV1-FS344 program is currently also being studied in 2 other patient populations, Becker muscular dystrophy and Duchenne muscular dystrophy, for which Milo received Orphan Designation in 2012.

The Orphan Drug Act (ODA) grants special status for products to treat rare diseases. Orphan designation qualifies the sponsor of the product for a tax credit and seven years of marketing exclusivity via the ODA. A marketing application for a prescription drug product that has been designated as a drug for a rare disease or condition is not subject to a prescription drug user fee. The disease or condition for which the drug is intended must affect fewer than 200,000 people in the United States.

MILO BIOTECHNOLOGY is a clinical stage company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases. For more information, visit

Contact Information:
Al Hawkins
Milo Biotechnology

Back to news