FDA approves vamorolone, now Agamree, for DMD patients 2 and older

Therapy has a more favorable side effect profile than other corticosteroids

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The U.S. Food and Drug Administration (FDA) has approved vamorolone for treating people with Duchenne muscular dystrophy (DMD), ages 2 and older. The therapy will be marketed as Agamree.

“We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with Duchenne muscular dystrophy and potentially other chronic inflammatory diseases. The approval of AGAMREE underscores the potential of reshaping the DMD treatment paradigm for this life-threatening rare disease,” Patrick J. McEnany, Catalyst Pharmaceuticals chairman and CEO, said in a press release.

Catalyst plans to launch the therapy in the U.S. in early 2024. The Catalyst Pathway Program will offer personalized treatment support with a dedicated team to answer questions and coordinate financial assistance. More information is available at www.yourcatalystpathways.com or by calling 1-833-422-8259.

“We expect to launch the product in the first quarter of 2024. At that time, we will introduce a comprehensive financial assistance program aimed at helping ensure accessibility and minimizing patient copays and deductibles, thereby enhancing affordability for all DMD patients. We look forward to successfully commercializing this product with a continued commitment to serving our patient communities,” McEnany said.

The therapy is under review in the European Union, where a committee issued a positive recommendation. A final decision from European regulators is expected by year’s end. Santhera Pharmaceuticals has also applied for regulatory clearance in the U.K.

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A more favorable side effect profile

Corticosteroids are a standard of care in DMD and work to reduce inflammation by mimicking the effects of the hormone cortisol. While they ease muscle damage in DMD, corticosteroids cause serious side effects in the long term, including high blood pressure, weight gain, emotional disturbances, weak bones, and stunted growth.

Originally developed by ReveraGen BioPharma and later acquired by Santhera, Agamree is called a dissociative steroid, meaning it seeks similar anti-inflammatory effects as traditional corticosteroids, but with a more favorable side effect profile. The difference lies in how medications interact with cortisol receptors in the body to exert their effects.

“Our unwavering commitment extends beyond this important milestone as we are resolute in our mission to ensure that DMD patients in the U.S. have access to this treatment option as we believe that AGAMREE may offer the potential of increasing the duration of ambulation and mobility in these patients, thereby significantly improving their overall quality of life,” McEnany said.

Agamree comes as an oral flavored liquid at 40 mg/mL and should be taken at a dose of 6 mg/kg per day, preferably with a meal, up to a maximum of 300 mg daily for patients weighing more than 50 kg (110 lbs). It is contraindicated for patients with hypersensitivity to vamorolone or any of the inactive ingredients.

Effects of Agamree in clinical trials

Santhera and ReveraGen completed a rolling application to the FDA last year, backed by data from the VISION-DMD trial (NCT03439670), which the regulatory agency indicated were sufficient to support a request for approval.

In VISION-DMD, 121 boys with DMD who could walk, ages 4-6, were assigned to receive oral Agamree (2 or 6 mg/kg), the traditional corticosteroid prednisone (0.75 mg/kg), or a placebo once daily for about six months. All the participants were then given Agamree for another six months.

Top-line results after about six months showed Agamree had a similar ability to improve muscle function as prednisone relative to a placebo. Those who switched from prednisone to Agamree didn’t see a loss in treatment efficacy after a year.

The dissociative steroid was associated with fewer side effects, however.

Agamree-treated patients’ growth was similar to boys on a placebo, whereas stunting was generally observed in the prednisone group, with a significant difference between the two types of steroids after six months.

When those on prednisone switched to Agamree, they experienced a 30% reduction in the number of side effects and a 60% reduction in side effects normally associated with corticosteroids. This included a reversal of stunted growth, decreases in behavioral changes, and stabilized body weight.

The most common side effects associated with Agamree include vomiting, vitamin D deficiency, psychiatric disorders, weight increase, and physical changes associated with high cortisol levels (cushingoid features).

“The approval of AGAMREE (vamorolone) provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some negative side effects associated with corticosteroids,” Sharon Hesterlee, PhD, chief research officer at the Muscular Dystrophy Association (MDA), said in a separate press release. “MDA is extremely proud to have been involved with vamorolone since its inception. It was one of the earliest investments to be made by our venture philanthropy arm and we are gratified to see that paying off in the form of a viable new therapy for Duchenne patients and the community.”

Debra Miller, CureDuchenne‘s founder and CEO, said in another press release: “CureDuchenne is proud to have contributed to the early development of vamorolone at ReveraGen, recognizing its potential to overcome some of the challenges related to steroid treatment. Reducing these side effects could have significant impact on the quality of life for patients with Duchenne, a paramount goal for CureDuchenne.”

Catalyst obtained North American marketing rights for Agamree in a deal with Santhera earlier this year. As part of the deal, Santhera agreed to transfer the approved new drug application for Agamree to Catalyst.

The FDA had granted Agamree orphan drug and rare pediatric disease designations for DMD. Orphan drug status means the therapy is eligible for seven years of market exclusivity upon approval date. Pending patents could provide further protection until 2040, according to Catalyst.

Phase 2 trial (NCT05166109) is testing vamorolone in men with Becker muscular dystrophy. Participants, ages 18-64, are still being sought at a single site in Pittsburgh.