FDA nod for muscular dystrophy drug comes at a cost to U.S. consumers
Recent approval of the corticosteroid deflazacort is good news/bad news for patients and their doctors. How much the predicted discounts will hope those in need remains to be seen.
To great applause from members of the medical community, the FDA has approved a steroid
used in treating a rare form of muscular dystrophy:
The news was heralded by the Muscular Dystrophy Association of America
(MDA) and others for the treatment of the debilitating disease:
However, for U.S. patients with Duchenne muscular dystrophy who have been
paying about $1,600 a year for the drug from an U.K.-based online pharmacy,
the announcement comes with sticker shock. Now that the drug will be
available in the U.S., its cost is set to increase dramatically and
international imports of the drug will end.
The Wall Street Journal
reported that Northbrook, Illinois-based Marathon Pharmaceuticals will
begin selling the drug in the U.S. market in March under the Emflaza name
with an annual price tag of $89,000. Patients might not shoulder the full
Though the corticosteroid is not a cure for Duchene muscular dystrophy, it
has been successful in improving muscle strength for those with access to
it. In the U.S., some 12,000 males suffer from this form of the disease;
most have a life expectancy into their 20s or 30s.
offers more information on its site for patients who will be making the
transition from deflazacort to Emflaza: