Agamree for Duchenne muscular dystrophy available by prescription in US

Agamree oral suspension 40 mg/mL is now commercially available in the United States for the treatment of Duchenne muscular dystrophy in patients aged 2 years and older, according to a press release.

Agamree (vamorolone, Santhera Pharmaceuticals) will be available by prescription and dispensed throughout the country through a specialty pharmacy network.

FDA approval for the novel corticosteroid was announced in October 2023, at which time Santhera announced that its partner Catalyst Pharmaceuticals would spearhead U.S.-based marketing.

Approval was based on data from the phase 2b VISION-DMD study, supplemented with safety information collected from three open-label trials, per the release.

In these studies, Agamree was administered at doses ranging from 2 mg/kg to 6 mg/kg per day, up to 48 months. Compared with current standard-of-care corticosteroids, Agamree demonstrated comparable efficacy, including data which suggested reductions in adverse events, particularly those related to bone health, growth trajectory and improved behavior.

“Agamree represents a transformative breakthrough in Duchenne muscular dystrophy treatment, offering hope and improved quality of life for patients,” Catalyst Pharmaceuticals CEO Richard J. Daly, told Healio in an email. “We are committed to helping ensure patient access through our Catalyst Pathways program. Together, we’re reshaping the DMD treatment landscape and advancing care for those in need."

The most common form of muscular dystrophy, Duchenne is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure and death.

According to statistics cited in the release, between 11,000 and 13,000 individuals in the U.S. are affected by Duchenne muscular dystrophy, with approximately 70% of those affected currently receiving corticosteroid treatment.

“Following the first global market introduction of this novel product in Germany in January, this launch represents an important next step in making Agamree available to as many patients with DMD as soon as possible,” Santhera CEO Dario Eklund, said in the release.

Reference:

Santhera’s Partner Catalyst Pharmaceuticals Launches AGAMREE® (Vamorolone) in the United States. https://www.santhera.com/assets/files/press-releases/2024-03-14_CPRXlaunchUSA_e_final.pdf. Published March 14, 2024. Accessed March 15, 2024.