Original Source
” FDA approves a new steroidal-type anti-inflammatory drug for Duchenne muscular dystrophy”
The Food and Drug Administration (FDA) on Thursday approved vamorolone, a new steroidal-type anti-inflammatory drug for boys with Duchenne muscular dystrophy (DMD), based on research performed at Children’s National Hospital. This approval provides boys with DMD with a clinically proven new treatment option.
Vamorolone, a new drug developed by ReveraGen BioPharma Inc., is structurally similar to traditional corticosteroids, but it has been chemically modified to reduce unwanted side effects such as brittle bones and reduced stature. Nearly two decades ago, Eric Hoffman, Ph.D., and Kanneboyina Nagaraju, D.V.M., Ph.D., began research on vamorolone while they were leading the Center for Genetic Medicine Research at Children’s National. They worked with Mark Batshaw, M.D., to develop vamorolone into a new clinical option for boys with Duchenne muscular dystrophy (DMD).
Duchenne muscular dystrophy (DMD) is a severe, life-threatening disease that affects only boys. ReveraGen BioPharma Inc. developed vamorolone, a new drug to treat DMD, in the hope of providing a new, FDA-approved treatment option for these children. In clinical trials, vamorolone was shown to improve muscle strength and stature with results comparable to prednisolone, but without some of the most impactful side effects of steroids, such as stunted growth and weakened bones.
The work of Drs. Eric Hoffman and Kanneboyina Nagaraju on vamorolone paved the way for entrepreneurship at Children’s National Hospital. They were the first faculty members to launch a spin-off company based on their research. Since then, more than 130 faculty members have been named as inventors on 132 patents. Children’s National is now home to Innovation Ventures, the hospital’s intellectual property development and commercialization arm, which provides guidance and resources to academic entrepreneurs who introduce a concept for pediatric medical products.
“We cannot wait to see the tremendous effort behind vamorolone in the hands of patients and clinicians treating Duchenne muscular dystrophy,” Eskandanian said. “Today’s FDA approval for ReveraGen shows the importance of supporting clinicians and researchers who are developing solutions to advance healthcare for children.”
Hoffman said in an interview that the drug has been through a series of clinical trials and proved to have advantages over other treatment options. In 2024, Catalyst Pharma will market vamorolone under the trade name Agamree in the United States.
For days now I’ve been glued to this gem of a site. The owner works tirelessly to engage fans with quality content. I’m mega impressed and can’t wait to see what they wow me with next!