Three Things You Missed From the AGAMREE (Vamorolone) Webinar

AGAMREE (vamorolone), an alternative corticosteroid treatment for patients with Duchenne muscular dystrophy 2 and older, received FDA approval on Oct. 26. And on Nov. 29, representatives from Catalyst Pharmaceuticals joined a CureDuchenne webinar to inform the community how the corticosteroid works and the pathway to access. Interested patients can now fill out Catalyst’s enrollment form with the help of their primary care provider.

The webinar covered a multitude of related topics, including the history and development of Catalyst, outlined by Amy Grover, senior director of patient advocacy, the overview of the science behind AGAMREE, and the Catalyst Pathway Program, which will help patients access corticosteroid. Watch the CureDuchenne Webinar with Catalyst now or check out three takeaways from the webinar that you may have missed below.

1. AGAMREE is available to everyone age 2 and older

Luis F. Lay, Jr, MD, medical science liaison for Catalyst, covered the basics. AGAMREE is available to anyone with Duchenne aged 2 and older, regardless of mutation. It’s expected to be available for patients in the first quarter of 2024. AGAMREE works in the traditional anti-inflammatory pathway by inhibiting the inflammation-causing protein NF-kB.

The medication is an orange-flavored oral suspension that will be administered once per day preferably with a meal. It was approved at 6 mg per kg, with a maximum dose of 300 mg. It’s also recommended to update all vaccines 4-6 weeks prior to starting the new corticosteroid. Full prescribing information can be found here.

2. It has fewer side effects than other corticosteroids

AGAMREE departs from other corticosteroids in its side effect profile. All these side effects were compared with the placebo group in the VISION-DMD study and against a different prednisone and deflazacort study.

Biomarkers related to bone breakdown for patients in the VISION-DMD study were not affected by AGAMREE. There were fewer cases of behavior issues, such as irritability, mood swings, temper tantrums, and aggressive behavior. Dose dependent weight gain occurred but leveled off after the initial dose.

Additionally, researchers found AGAMREE may have some cardioprotective effects in mouse studies but have no data yet on this affect in humans. That’s why Catalyst suggests that all patients enter the registry so that any effects can be tracked long-term. The primary study was only done for 4–7-year-olds, so they are hoping to track more data on older patients through this registry.

It’s also important to note that the switch from another steroid to AGAMREE can be made right away if you or your doctor decide to start AGAMREE.

3. How you can get access to AGAMREE

Maria Pandolfo spoke to access considerations. The free patient support program Catalyst Pathways ensures that patients will always have treatment available. A bridge program will provide medication for free if there are any delays in insurance authorizations. A care coordinator will reach out as a point of contact and to help schedule shipments and a patient access liaison will help with navigating the Duchenne diagnosis and answer any related AGAMREE questions. Spanish-speaking members are also available to support families as needed.

“We certainly know that there is a burden on rare disease patients, and we try to lift some of that burden by making sure that they have access to their medication when they need it,” Pandolfo said.

After filling out the enrollment form, Catalyst will work with the insurance company to get it approved. And once it is made available through the specialty pharmacy AnovoRX, a care coordinator will reach out to schedule the shipment, which will occur when the treatment comes to market in the first quarter of 2024.

As of now AGAMREE is only approved in the US, though Catalyst is working through the regulatory process in Canada, and Santhera (the company Catalyst licensed AGAMREE from) is doing the same in the EU.

Please join our next online event on Dec. 12, “Ask Me Anything: Endocrinology Issues in Duchenne.” This is an opportunity for families to ask questions of Nat Nasomyont, MD, a pediatric endocrinologist at Cincinnati Children’s Hospital Medical Center.