(Adds UnitedHealth, Aetna)
By Natalie Grover
Oct 7 Health insurer Anthem Inc said it
will not cover Sarepta Therapeutics Inc's drug to treat
the rare condition of Duchenne muscular dystrophy (DMD), calling
it "investigational and not medically necessary."
The U.S. Food & Drug Administration approved the drug last
month under pressure from patient advocates, even though an
outside panel of experts and the agency's own reviewers had
questioned its efficacy.
Sarepta has priced the drug, called Exondys 51, at about
$300,000 per patient per year.
The Cambridge, Massachusetts-based company's stock was down
about 7 percent on Friday.
UnitedHealth Group Inc., the largest U.S. health
insurer, does plan to cover the drug, a spokesman said on
Friday. Aetna Inc plans to conduct a full clinical
review to determine its coverage policy, spokesman T.J. Crawford
said on Friday.
Anthem, the second-largest U.S. health insurer, said on
Thursday on its website that clinical benefit, including
improved motor function, had not been demonstrated by Exondys
"Exondys 51 failed to show it improves health outcomes, and
therefore it is not a covered benefit for our members," Anthem
spokeswoman Leslie Porras said in an emailed statement on
DMD, a rare genetic muscle-wasting condition, typically
emerges in childhood and mostly affects boys. It causes weakness
in the arms and legs and eventually affects the lungs and heart.
Patients frequently die in their 20s or 30s, according to the
National Institutes of Health.
The drug is designed to treat about 13 percent of all DMD
patients, or some 1,300 to 1,900 people in the United States.
The FDA gave it accelerated approval based on data believed
to predict a clinical benefit. Sarepta has to prove that benefit
in a subsequent clinical trial, the outcome of which is expected
to take at least a few years.
To keep health insurance affordable, companies need to
ensure that they are paying for safe and effective treatments,
said Diana Zuckerman, president of non-profit organization
National Center for Health Research.
"When FDA fails to ensure those standards, then 'FDA
approval' is no longer a gold standard that insurance companies
can rely on," she said.
Shares of Sarepta, which has announced DMD collaborations
with Catabasis Pharmaceuticals Inc and Summit
Therapeutics Plc, have more than doubled since the FDA
announced its approval on Sept. 19.
(Reporting by Natalie Grover in Bengaluru and Caroline Humer in
New York; Editing by Shounak Dasgupta and Steve Orlofsky)