Agamree, next-gen steroid, now available for DMD patients in US

Treatment expected to cause fewer side effects than other corticosteroids

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

A large bell with the word

Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, in the U.S., where it will be dispensed through a specialty pharmacy network.

The U.S. Food and Drug Administration (FDA) approved the next-generation steroid, expected to be more tolerable than standard DMD corticosteroids, in October. It will be marketed by Catalyst Pharmaceuticals, which acquired North American rights to the therapy from Santhera ahead of its approval.

“Today, we proudly announced the U.S. commercial availability of Agamree, an innovative alternative steroid treatment for Duchenne Muscular Dystrophy,” Richard J. Daly, Catalyst’s CEO, said in a company press release. “This significant milestone offers hope for improved quality of life for individuals living with this devastating rare disease.”

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Duchenne MD steroid also an approved treatment in EU and UK

Agamree also has been approved in the European Union and U.K. for DMD patients ages 4 and older, where it is being marketed by Santhera Pharmaceuticals. It has been available to patients in Germany since January, the company reported.

“Congratulations to our partner Catalyst on the launch of Agamree in the United States,” Dario Eklund, Santhera’s CEO, said in a separate press release. “This launch represents an important next step in making Agamree available to as many patients with DMD as soon as possible.”

Catalyst is offering a support program called Catalyst Pathways, where enrolled patients will be paired with a “personal access liaison” or guide for program resources that include financial help, educational materials, and free in-person and online community events enabling patients to meet others with shared experiences.

According to Daly, Catalyst Pathways extends to patients and their families “the one‐on‐one education and financial support they need, thereby enhancing the access and affordability of Agamree” to them.

“We … remain resolute in our mission to help ensure that all patients have access to this novel therapy,” Daly added.

Corticosteroids have long been the backbone of DMD treatment, with potent anti-inflammatory properties that help to ease muscle damage and slow disease progression. But their long-term use risks serious side effects, including weight gain, fragile bones, high blood pressure, and fluid retention.

Agamree is a dissociative oral steroid, meaning that it is expected to have the same clinical benefits as standard corticosteroids but is designed to avoid activating the signaling pathways that lead to these various side effects. As such, it intends to offer patients with an equally effective, but more tolerable option.

“Agamree marks an important therapeutic advancement with the prospect of reshaping the treatment paradigm for this life-threatening condition,” Daly said.

Agamree showed a better safety profile than prednisone in a Phase 2b trial

Data from four clinical trials support the therapy’s safety and efficacy profile in DMD. The Phase 2b VISION-DMD trial (NCT03439670) was one such study; it compared 48 weeks of treatment Agamree to prednisone, a conventional corticosteroid, in 121 boys with Duchenne MD.

Results showed that Agamree and prednisone were similarly effective at preserving muscle function, but children given Agamree had fewer adverse events, particularly those related to bone health, growth, and behavior.

Catalyst recently announced plans to continue evaluating Agamree via an observational patient registry, involving about 25 U.S. sites, that will monitor its real-world treatment effects in some 250 DMD patients for up to five years.