CureDuchenne Comments on U.S. Food and Drug Administration Breakthrough Therapy Designation for GlaxoSmithKline’s/Prosensa

  CureDuchenne Comments on U.S. Food and Drug Administration Breakthrough
  Therapy Designation for GlaxoSmithKline’s/Prosensa Therapeutics' Drisapersen
  for Treatment of Patients with Duchenne Muscular Dystrophy

Business Wire

NEWPORT BEACH, Calif. -- June 27, 2013

CureDuchenne is pleased that the U.S. Food and Drug Administration (FDA)
granted Breakthrough Therapy designation to drisapersen, an exon-51 skipping
compound for the potential treatment of patients with Duchenne Muscular
Dystrophy. Drisapersen is being developed by GlaxoSmithKline plc (GSK) and
licensed from Prosensa Therapeutics.

The Breakthrough Therapy designation is one of several programs created by the
FDA to expedite the development and review of drugs for serious or
life-threatening conditions. It was enacted in 2012 as part of the Food and
Drug Administration Safety and Innovation Act (FDASIA). For more information
on the Breakthrough Therapy designation, click here.

“The Breakthrough Therapy designation means the FDA has reviewed the data for
drisapersen and will provide additional resources,” said Debra Miller, CEO and
Founder, CureDuchenne. “This classification generally is for clinical programs
that demonstrate medical significance. Duchenne is a disease that progresses
very quickly and for which there’s currently no cure, so on behalf of the
Duchenne community, we are grateful to the FDA for recognizing the need and
potential for this drug. We hope to see the drug approved soon.”

CureDuchenne, a leader in raising awareness and funding research to find a
cure for Duchenne, committed significant funds at an early stage to Prosensa’s
exon skipping research that led to the development of drisapersen.
CureDuchenne has worked with the FDA to help expedite approval of Duchenne
drugs based upon the severity of the disease and the risk tolerance of the
patient community.

“These results validate our early efforts to fund novel research and offer
hope for finding an effective and safe treatment for all those living with
Duchenne,” Ms. Miller continued. “We applaud GlaxoSmithKline and Prosensa for
their tireless work in developing drisapersen as a treatment for the disease.”

Currently, there is no treatment for Duchenne, a progressive muscle wasting
disease and the most common and lethal form of muscular dystrophy. Duchenne
impacts one in every 3,500 boys; nearly 20,000 boys are living with the
disease in the United States. Boys with Duchenne are usually diagnosed before
the age of 5 and are in a wheelchair by age 12; most don’t survive their

About CureDuchenne

CureDuchenne is a national nonprofit organization dedicated to finding a cure
for Duchenne, the most common and most lethal form of muscular dystrophy. As
the leading genetic killer of young boys, Duchenne affects more than 300,000
boys worldwide.

CureDuchenne has garnered international attention for its efforts to raise
funds and awareness for Duchenne through venture philanthropy. With the help
of CureDuchenne’s distinguished international panel of Scientific Advisors,
the organization has raised more than $11 million towards promising research
aimed at treating and curing Duchenne. In fact, seven CureDuchenne research
projects have made their way into human clinical trials – a unique
accomplishment as few health-related nonprofits have been successful in being
a catalyst for human clinical trials. For more information on how to help
raise awareness and funds needed for research, visit


For CureDuchenne
Karen Harley, 949-872-2552
Stephanie Blank, 646-805-2019
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