FDA Grants Orphan Drug Status to Resolaris as Treatment for Limb Girdle MD
ATyr Pharma’s Resolaris has received orphan drug status in the United States for the treatment of limb girdle muscular dystrophy (LGMD).
The Food and Drug Administration designation gives companies financial and other incentives to develop therapies for diagnosing or treating rare diseases. In the United States that means fewer than 200,000 people.
Resolaris is a first-in-class intravenous protein for treating rare myopathies that have an immune component. ATyr developed it from a naturally occurring protein released by skeletal muscle cells. It could benefit those with LGMD and LGMD2B, the company said.1
“Receiving Orphan Drug Designation for the treatment of all types of LGMD patients with Resolaris is an important step in our overall process of bringing our first Physiocrine-based product candidate to the market to treat patients,” John Mendlein, chief executive officer of aTyr Pharma, said in a press release.
The FDA granted fast track designation to Resolaris in February of this year.
The trial showed the drug was safe and did not suppress circulating immune cells. It also displayed effectiveness, improving muscle function in 78 percent of treated patients — 7 of 9 people — at 14 weeks. Their scores in a manual muscle test improved by a mean of 6.2 percent compared to baseline, or scores recorded prior to treatment, the company reported at the time.
Source : MD News