This year’s top content in the Duchenne muscular dystrophy (DMD) space focused on a novel gene therapy approval, the high cost of care for this genetic disorder, and steps forward in other treatments.
The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.
Less than two weeks after getting a thumbs-up from the EMA’s Committee for Medicinal Products for Human Use (CHMP), Santhera Pharmaceutical AG’s vamorolone secured U.S. FDA approval for use in patients, 2 and older, with Duchenne muscular dystrophy (DMD). A first-in-class drug, vamorolone, branded Agamree, is expected to offer a safer alternative to the steroid therapy, which the company has said will remain a foundational treatment of DMD, even with the introduction of gene therapies.
Swiss drug maker Santhera Pharmaceuticals Holding AG (SPHDF.OB) announced Friday that European Commission's Committee for Medicinal Products for Human Use or CHMP adopted a positive opinion in favor of approval of AGAMREE (vamorolone) to treat Duchenne muscular dystrophy or DMD patients aged 4 years and older.