The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.
Pharmacy Times® originally published this article. This version has been lightly edited.
The FDA has approved the new drug application for vamorolone (Agamree; Santhera Pharmaceuticals) oral suspension 40 mg/mL, a novel corticosteroid treatment indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years and older. At the 24-week follow-up of the phase 2b VISION-DMD study (NCT03439670), vamorolone met the primary end point of time to stand velocity compared with placebo (P = .002); it also demonstrated a favorable safety and tolerability profile, which is not the case for current standard-of-care corticosteroids.1-3
“We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with DMD and potentially other chronic inflammatory diseases,” said Patrick J. McEnany, founder, chairman, and CEO of Catalyst Pharmaceuticals, in a press release.1
Catalyst is the US-license holder for vamorolone, and plans a commercial launch in Q1 of 2024. The company also noted it will provide a financial assistance program to reduce co-pays and deductibles, promoting the accessibility and affordability of the product.1
DMD is the most common form of muscular dystrophy, affecting 11,000 to 13,000 people in the United States. The disease is characterized by the regression of muscle function. Patients with the rare, fatal disorder will slowly lose ambulation and suffer from respiratory failure, with the disease eventually leading to death. The current standard of care is corticosteroids (despite their severe adverse event [AE] profile), and approximately 70% of patients are on concomitant corticosteroid treatment.1,2
The disease is characterized by the regression of muscle function, and patients with the rare, fatal disorder will slowly lose ambulation and suffer from respiratory failure | Image Credit: Dr_Microbe - stock.adobe.com
In 2021, the drug was evaluated in the VISION-DMD study, a randomized, double-blind, parallel group, placebo and active-controlled study that looked at the efficacy, safety, pharmacodynamics, and pharmacokinetics of daily oral vamorolone in a population of 120 boys with ambulant disease who were older than 4 years and younger than 7 years. Secondary study outcomes comprised safety measures, including organ system class-related AEs and biomarkers, such as fasting insulin.2,3
Vamorolone is different than other corticosteroids because its mode of action is founded on its differential effects on glucocorticoid and mineralocorticoid receptors. The treatment can modify the downstream activity of the receptors to create an effective treatment that also has a more tolerable AE profile. Common AEs associated with vamorolone are mild to moderate cushingoid features, vomiting, and vitamin D deficiency.1
On October 26, vamorolone also received orphan drug and pediatric rare disease designations for the treatment of DMD, earning it 7 years of orphan drug exclusivity.1
“The approval of vamorolone underscores the potential of reshaping the DMD treatment paradigm for this life-threatening rare disease,” McEnany concluded in the press release. “We look forward to successfully commercializing this product with a continued commitment to serving our patient communities."1
References
1. Catalyst Pharmaceuticals reports FDA approval of Agamree (vamorolone) for Duchenne muscular dystrophy granted to Santhera Pharmaceuticals. News release. Catalyst Pharmaceuticals. October 26, 2023. Accessed October 26, 2023. https://www.biospace.com/article/releases/catalyst-pharmaceuticals-reports-fda-approval-of-agamree-vamorolone-for-duchenne-muscular-dystrophy-granted-to-santhera-pharmaceuticals/
2. Vamorolone phase 2b (VISION-DMD): a study to assess the efficacy and safety of vamorolone in boys with Duchenne muscular dystrophy (DMD). DMD HUB. Accessed October 26, 2023. https://dmdhub.org/trials/vamorolone-phase-2b/
3. A study to assess the efficacy and safety of vamorolone in boys with Duchenne muscular dystrophy (DMD). ClinicalTrials.gov. Updated March 9, 2023. Accessed November 1, 2023. https://clinicaltrials.gov/study/NCT03439670
Higher Life’s Essential 8 Scores Associated With Reduced COPD Risk
November 21st 2024Higher Life’s Essential 8 (LE8) scores, especially those reflecting lower nicotine exposure and better sleep health, are inversely associated with chronic obstructive pulmonary disease (COPD) risk, emphasizing the importance of cardiovascular health (CVH) in disease prevention.
Read More
New Study Finds Risk Groups, Outpatient Care Barriers in Chronic Liver Disease
November 20th 2024Patients with chronic liver disease who were unable to establish care were 85% more likely to require recurrent hospitalizations. This group included a disproportionate number of women and individuals with physical limitations affecting their health.
Read More
Insurance Insights: Dr Jason Shafrin Estimates DMD Insurance Value
July 18th 2024On this episode of Managed Care Cast, we're talking with the author of a study published in the July 2024 issue of The American Journal of Managed Care® that estimates the insurance value of novel Duchenne muscular dystrophy (DMD) treatment.
Listen
Why Right Heart Catheterization Confirming PAH Diagnosis May Be Underperformed
November 20th 2024Professional guidelines say that when pulmonary arterial hypertension (PAH) is diagnosed, right heart catheterization should be performed, but a quarter of the time, it isn’t—so investigators set out to discover why.
Read More
OS Better With Belantamab Mafodotin Triplet vs Daratumumab in R/R MM
November 19th 2024The key secondary end point of overall survival (OS) was met in the DREAMM-7 trial of belantamab mafodotin (Blenrep; GSK) for the treatment of patients with relapsed/refractory multiple myeloma (R/R MM).
Read More
