Recently, the FDA announced the approval of a new drug, vamorolone, as a treatment for Duchenne muscular dystrophy (DMD), a genetic muscle disease that usually affects young boys. Understandably, this news hasn’t made a big splash in myositis circles, but it should.
Vamorolone, marketed under the name Agamree, is a corticosteroid drug with a twist. It was created by a group of scientists, Drs. Eric Hoffman, John McCall, and former TMA Medical Advisory Board member Kanneboyina Nagaraju, as an alternative to steroid drugs like prednisone. Corticosteroids cause severe side effects when used long-term and at high doses, so clinicians always try to limit how long the patient must use them.
The exciting thing is that vamorolone could revolutionize the treatment of so many inflammatory diseases that depend on long-term steroid use. Dr. Nagaraju and his colleague’s invention changes the prednisone molecule to remove the part of the compound that causes the side effects but keeps the part that improves symptoms caused by inflammation.
That means people who rely on long-term, high-dose prednisone—like those who live with myositis—may potentially get relief from muscle weakness, skin rashes, and other debilitating symptoms without worrying about getting diabetes, glaucoma, or other life-altering side effects.
Vamorolone is not without some side effects, however. The anti-inflammatory part of the steroid compound can still cause mild to moderate symptoms like moon face, weight gain, acne, vomiting, vitamin D deficiency, and others.
While it’s too early to celebrate vamorolone as a treatment for myositis, Dr. Nagaraju, who serves as dean of the School of Pharmacy and Pharmaceutical Sciences at SUNY-Binghamton University, agrees it could be a game changer for those who live with this disabling disease of the muscles.
“Vamorolone would be a great drug for myositis,” he says. “I will strongly advocate myositis as the next indication to focus on.”
TMA will keep tabs on this process and will let the community know when clinical trails for vamorolone are available for those with myositis. If you want to stay informed about this and other developments in the myositis world, be sure to join TMA. It’s FREE.