Agamree Now Commercially Available to Treat Duchenne Muscular Dystrophy

Catalyst Pharmaceuticals announced the commercial availability of vamorolone (Agamree) in the US for the treatment of Duchenne muscular dystrophy (DMD).¹

Duchenne | Image credit: hafakot – stock.adobe.com

Approved by the FDA in fall 2023, vamorolone is a first-in-class corticosteroid to treat DMD in patients aged 2 years and older.² It is available by prescription as a once-daily, orange-flavored liquid oral suspension of 40 mg/mL.

DMD is a rare and severe neuromuscular disorder affecting thousands of patients in the US. It is estimated that approximately 11,000 to 13,000 Americans are affected by DMD. Among them, roughly 70% of patients are currently receiving treatment with corticosteroids, which often come with notable side effects.

Vamorolone represents a groundbreaking advancement in the treatment of DMD as a less toxic alternative to conventional corticosteroids, with an emphasis on improving quality of life and reducing adverse events (AEs). One notable concern with long-term corticosteroid use is its adverse effects on bone health, leading to increased brittleness and susceptibility to fractures over time.

However, in clinical trials, vamorolone has demonstrated its ability to prolong ambulation in patients with DMD while minimizing the occurrence of AEs commonly associated with traditional corticosteroid therapies. This less toxic profile is highly important for patients and their families, offering hope for improved outcomes without compromising safety or tolerability. By offering a safer alternative, Agamree aims to enhance the overall treatment experience for individuals living with DMD, empowering them to lead fuller, more active lives.

"With the potential to extend ambulation and mobility for patients, AGAMREE marks an important therapeutic advancement with the prospect of reshaping the treatment paradigm for this life-threatening condition,” Richard J. Daly, CEO of Catalyst, said in a news release.¹ “We are firmly positioned to leverage our well-established commercial capabilities for a successful U.S. launch and remain resolute in our mission to help ensure that all patients have access to this novel therapy. Our Catalyst Pathways, a personalized program, is readily available to assist Duchenne Muscular Dystrophy patients and their families with the one‐on‐one education and financial support they need, thereby enhancing the access and affordability of AGAMREE for every patient.”

Daly was appointed as CEO only a few days before vamorolone was approved, replacing Patrick J. McEnany, cofounder and chairman, as CEO.³ In the same month, the European Medicines Agency's Committee for Medicinal Products for Human Use expressed a positive opinion on vamorolone's potential to treat DMD in children and adults aged four years and older.⁴ If endorsed by the European Commission, vamorolone will mark a milestone as the first drug to receive full approval in Europe for DMD treatment.

In the US, the FDA’s approval of vamorolone was based on comprehensive data from the phase 2b VISION-DMD study and additional safety information from open-label trials.

Agamree stands out due to its unique mechanism of action, which involves targeting glucocorticoid and mineralocorticoid receptors with a differential effect and modifying downstream activity, potentially offering a more tolerable treatment option for patients with DMD. With orphan drug exclusivity and pending patents, vamorolone represents a significant advancement in addressing the unmet needs of individuals living with DMD.

References

1. Catalyst Pharmaceuticals announces AGAMREE® now commercially available in the U.S. for the treatment of Duchenne muscular dystrophy (DMD). News release. Catalyst Pharmaceuticals. March 13, 2024. Accessed March 19, 2024. https://ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-announces-agamreer-now-commercially
2. Hunter E. FDA approves vamorolone to treat Duchenne muscular dystrophy. AJMC. November 2, 2023. Accessed March 19, 2024. https://www.ajmc.com/view/fda-approves-vamorolone-to-treat-duchenne-muscular-dystrophy
3. Catalyst Pharmaceuticals appoints Richard J. Daly as chief executive officer. News release. Catalyst Pharmaceuticals. October 19, 2023. Accessed March 19, 2024. https://ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-appoints-richard-j-daly-chief-executive
4. Clinical Trials Arena. Agamree for the treatment of Duchenne muscular dystrophy, US. November 22, 2023. Accessed March 19, 2024. https://www.clinicaltrialsarena.com/projects/agamree-treatment-dmd-us/?cf-view