Express Potential "first-in-class" DMD new drugs have been submitted to avoid common side effects of steroids

Recently, Santhera Pharmaceuticals and ReveraGen BioPharma jointly announced the completion of a rolling New Drug Application (NDA) submission to the US FDA and seeking priority review status
for its investigational drug vamorolone for the treatment of Duchenne muscular dystrophy (DMD).
Vamorolone is a "first-in-class" therapy
for the potential treatment of DMD.
If approved, Vamorolone will likely be available
in the U.
S.
in the second half of 2023.

DMD is a rare X-chromosome-linked genetic disorder caused by mutations in the gene encoding dystrophin on the X chromosome, so it affects almost exclusively male patients and is one of
the most common neuromuscular diseases in children.
DMD is characterized by an inflammatory response at birth or shortly after birth, leading to muscle fibrosis and clinical muscle atrophy and degeneration
.
Milestones of disease progression include loss of the ability to walk and eat, as well as the need for respiratory assistance and myocardial disease
.
Due to respiratory and/or heart failure, the life expectancy of people with DMD usually does not exceed 40 years
.
Coricosteroids are the standard of care for children and adults with DMD, but although these hormones can delay disease progression, they have many side effects
.

Vamorolone is an investigational drug that, although bound to the same receptors as corticosteroids, is considered a "dissociative" anti-inflammatory drug
because it selectively activates certain signaling pathways of steroids.
That is, while triggering anti-inflammatory effects, it has the potential to avoid ("free") the safety concerns and side effects of traditional steroids, which may replace existing therapies
.
Vamorolone is an orphan drug for the treatment of DMD in the U.
S.
and Europe, as well as Fast Track designation and rare pediatric disease designation
from the U.
S.
FDA.
This drug has the potential to be a "first-in-class" free steroid therapy
.

The submission is based on positive results from the VISION-DMD pivotal Phase 2b trial, including:

• Efficacy and safety
  of vamorolone (2, 6 mg/kg/day) for 24 weeks compared with placebo and prednisone (0.
  75 mg/kg/day).
  

• The 24-week trial examined the maintenance of drug efficacy and continued to collect long-term safety and resistance data
  .
  

• Four open-label trials (including extension trials) in which patients were treated with vamorolone at doses of 2 or 6 mg/kg/day for up to 30 months
  .
  An external control trial
  was also included.
  

  
  

"The completion of vamorolone's new drug application submission is an important step for us to bring this investigational therapy to DMD patients, and it represents an important milestone for Santhera," said Mr.
Dario Eklund, CEO of Santhera.

”

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