FDA grants orphan drug status to Milo’s Duchenne and Becker muscular dystrophy candidate

AAV1-FS344 is myostatin inhibitor delivered through gene therapy to increase muscle strength in patients.

At present the company is conducting a Phase I/II trial in patients with Becker muscular dystrophy and inclusion body myositis at Nationwide Children’s Hospital.

Milo Biotechnology CEO Al Hawkins said the orphan drug designation will make the company eligible for incentives that support the continued development of the therapy in rare muscular dystrophies.

"We hope that our potent muscle strengthening approach will someday augment emerging genetic strategies to transform care for these intractable diseases," Hawkins added.

With the receipt of orphan drug designation, the company is eligible for tax credit and seven years of marketing exclusivity of the product.