Milo Biotechnology has secured Orphan Drug designation for its AAV1-FS344 from the Federal Drug Administration’s (FDA) Office of Orphan Products Development for treatment of inclusion body myositis.
A gene therapy-delivered follistatin protein, AAV1-FS344 increases muscle strength and function.
At present, AAV1-FS344 is undergoing a Phase I/II trial at Nationwide Children's Hospital in adult patients suffering from sporadic inclusion body myositis.
In May, initial data from the trial indicated safety and improvement in function in six patients.
The trial is funded by the Parent Project Muscular Dystrophy and The Myositis Association.
Milo Biotechnology chief executive officer Al Hawkins said: “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.”
Inclusion body myositis causes severe and progressive muscle weakness in adults. Several patients are wheelchair bound within ten years of diagnosis.
The AAV1-FS344 programme is currently also being studied in two more patient populations, Becker muscular dystrophy and Duchenne muscular dystrophy. For this, Milo secured Orphan Designation in 2012.
The Orphan Drug Act (ODA) grants special status for products to treat rare diseases.
Orphan designation grants a product sponsor to be eligible for a tax credit and seven years of marketing exclusivity via the ODA.
Furthermore, 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 that the drug is intended for must affect fewer than 200,000 people in the US.