Lawmakers question pricing of drug for genetic disease

The steroid drug, deflazacort, which treats Duchenne muscular dystrophy, has been approved overseas for years and is sold as a generic.

The two congressmen, who have previously eyed Valeant, Turing, and Mylan, among other pharma firms, on Monday wrote a letter to Marathon Pharmaceuticals seeking answers about its newly approved $89,000 treatment for Duchenne muscular dystrophy. The FDA approved Marathon's version of the drug for the US on Thursday.

Corticosteroids are commonly used to treat DMD across the world.

The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. However, U.S. patients have been importing the drug from foreign countries since the 1990s, after clinical studies demonstrated its potential for reducing inflammation with fewer adverse effects than another steroid.

"Marathon will have a monopoly on deflazacort for years to come, preventing less expensive generic competitors from entering the market, despite the fact that this drug is already available in generic form in other countries", the lawmakers wrote. The drug will be available in both oral suspension and tablets, and can be administered to patients 5 years of age and older.

The Wall Street Journal reports the now-FDA-approved deflazacort will be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the U.S.; Marathon CFO Babar Ghias tells the Washington Post the net price will be $54,000 after rebates and discounts.

A physical therapist treats a patient with DMD. With the orphan designation, the company is getting seven years of exclusive rights to sell the drug in the U.S.

The sponsor is receiving a rare pediatric disease priority review voucher under a program meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. The drug isn't a cure, but it has been shown to improve muscle strength, the FDA said in a statement announcing the approval. Patients who showed improvement at 12 weeks maintained their muscle strength through the end of the 52-week study. Patients lack a protein called dystrophin that's essential for healthy muscle, and eventually lose the ability to walk, stand and breathe. The most common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism) and excessive fat around the stomach (central obesity).

  • Anthony Vega