Lawmakers question pricing of drug for genetic disease

Lawmakers question pricing of drug for genetic disease

Lawmakers question pricing of drug for genetic disease

Deflazacort has been available outside the United States for decades. The third major proof point in the Muscular Dystrophy Association's research program, Emflaza joins two other drugs approved for treating muscular dystrophy: Exondys 51 and Spinraza. 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.

The regulator gave a green light to Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with the rare genetic disorder, which causes progressive muscle deterioration and weakness in children, nearly always boys.

Duchenne Muscular Dystrophy is caused by an absence of a protein that helps keep muscle cells intact. DMD occurs in about one of every 3,600 male infants worldwide.

People who have DMD eventually lose the ability to carry out activities on their own and are wheelchair bound by the time they reach their early teen years. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Marathon anticipates a smooth transition for patients ready getting deflazacort from overseas pharmacies.

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The drug wasn't sold in the US mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval. In its response last week to the FDA's approval, the Illinois-based company said it was committed to ensuring that US patients with prescriptions have access to the drug and that the company would provide the most robust patient support programs "allowed by law to qualifying patients".

Marathon Pharmaceuticals is a U.S. biopharmaceutical company that develops treatments for rare diseases, with a focus on patients who now have no treatment options. 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. Sarepta's drug treats a subset of DMD patients representing about 13 percent of the total.

How was the company able to get away with such an outrageous list price in America?

The two lawmakers say that "exorbitantly" pricing potentially life-saving medications hurts patient access and drives up prices for the entire health care sector. According to scientists, at week 12 the patients who were under deflazacort showed improvements in their muscle strength compared to those who were taking a placebo. 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). Another study showed patients who took deflazacort lost the ability to walk later than patients who took placebo.

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