The US Food and Drug Administration (FDA) has approved the
corticosteroid deflazacort (Emflaza, Marathon Pharmaceuticals) to treat
patients age 5 years and older with Duchenne muscular dystrophy (DMD), the
agency said today.
"This is the first FDA approval of any corticosteroid
to treat DMD and the first approval of deflazacort for any use in the United
States," according to an FDA news release. Deflazacort will be available
in tablets and oral suspension.
"This is the first treatment approved for a wide range
of patients with Duchenne muscular dystrophy," Billy Dunn, MD, director,
Division of Neurology Products, FDA Center for Drug Evaluation and Research,
said in the release. "We hope that this treatment option will benefit many
patients with DMD."
DMD is a rare genetic disorder that causes progressive
muscle deterioration and weakness. The first symptoms typically appear between
age 3 and 5 years and worsen over time. The disease primarily affects boys.
Estimates are that DMD occurs in about 1 of every 3600 male infants worldwide.
The effectiveness of deflazacort was demonstrated in a
clinical study of 196 boys with DMD aged 5 to 15 years.
At week 12, patients receiving deflazacort had improvements
in muscle strength compared with those taking a placebo, which was maintained
at week 52.
In a separate trial of 29 male patients lasting 104 weeks,
deflazacort demonstrated a numeric advantage over placebo on an assessment of
average muscle strength. Patients receiving deflazacort also appeared to lose
the ability to walk later than those treated with placebo.
Adverse effects related to deflazacort mirror those seen
with other corticosteroids, the FDA said. The most common side effects include
facial puffiness (cushingoid appearance), weight gain, increased appetite,
upper respiratory tract infection, cough, pollakiuria, hirsutism, and central
obesity.
Less common side effects include problems with endocrine
function, increased susceptibility to infection, elevation in blood pressure,
risk for gastrointestinal perforation, serious skin rashes, behavioral and mood
changes, decrease in the density of the bones, and vision problems (such as
cataracts).
Deflazacort was given fast-track designation and priority
review by the FDA. The drug also received orphan drug designation, which
provides incentives to assist and encourage the development of drugs for rare
diseases.
http://www.medscape.com/viewarticle/875608
A drug to treat muscular dystrophy will hit the U.S. market with a price tag of $89,000 a year despite being available for decades in Europe at a fraction of that cost.
ReplyDeleteMarathon Pharmaceuticals LLC’s pricing of the drug, which has been available in Europe, is the latest example of a business model that has drawn ire from doctors, patients and legislators in recent years: cheaply acquiring older drugs and then drastically raising their prices.
The practice has prompted congressional investigations and hearings into companies including Valeant Pharmaceuticals International Inc. and Turing Pharmaceuticals LLC, the firm formerly run by onetime hedge-fund manager Martin Shkreli.
The U.S. Food and Drug Administration on Thursday approved Marathon’s drug, a corticosteroid called deflazacort, to treat a rare type of muscular dystrophy that affects some 12,000 boys in the U.S., most of whom die in their 20s and 30s. The drug isn’t a cure, but it has been shown to improve muscle strength, the FDA said in a statement announcing the approval.
http://www.foxnews.com/health/2017/02/11/marathon-pharmaceuticals-to-charge-89000-for-muscular-dystrophy-drug-after-70-fold-increase.html