Santhera receives FDA nod for Agamree

Santhera Pharmaceuticals has received the Food and Drug Administration’s blessing for Agamree (vamorolone) oral suspension 40 mg/ml for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older.

“We are delighted to secure FDA approval which comes just weeks after the positive opinion from the CHMP of the European Medicines Agency. This is a hugely important moment for DMD patients who need an efficacious and well-tolerated therapy for this debilitating condition,” said Dario Eklund, CEO of Santhera. “Today is a very satisfying day for the Santhera team and we are grateful to the patients, their families and physicians who participated in the vamorolone program. We look forward to working closely with our partner Catalyst Pharmaceuticals as it prepares for U.S. commercialization of the product.”

“This new drug approval gives Duchenne patients and their families one more reason for hope,” said Pat Furlong, founding president and CEO of the Parent Project Muscular Dystrophy. “Steroids are considered standard of care for Duchenne, due to their valuable role in slowing disease progression. However, they often come with considerable side effects. Vamorolone has the potential to be an alternative steroid with a better tolerability profile addressing an important unmet medical need for patients.”

[Read more: Walgreens, Cariloop support patients with Multiple Sclerosis]

“I am excited for the Duchenne community who have been waiting a long time for an alternative to the current standard of care. Today’s news represents the culmination of many years of research to bring vamorolone to patients,” said Eric Hoffman, president and CEO of ReveraGen BioPharma. “We are pleased to have worked alongside Santhera, the DMD patient community, researchers and healthcare practitioners to reach this important milestone.”

Santhera will now transfer the U.S. marketing authorization for Aga,ree to its partner Catalyst Pharmaceuticals who holds an exclusive license for AGAMREE in North America and plans to launch the product in the United States in the first quarter of 2024.

[Read more: Retailers build out their specialty pharmacy offerings]