Here are seven things to know.
1. PTC Therapeutics purchased the muscular dystrophy treatment from Marathon Pharmaceuticals in March 2017, a month after the FDA approved Emflaza as the second available treatment for the rare condition.
2. For decades — before the FDA approved the drug for sale in the U.S. — patients would import the drug, generically known as deflazacort, from other countries for as little as $1,000 per year.
3. Controversy surrounding the drug arose after Marathon announced the treatment would cost $89,000 per year in the U.S. Marathon delayed the launch of Emflaza in the U.S. following public scrutiny of the drug’s pricetag.
4. PTC launched Emflaza in May 2017 at a list price of about $59,700 annually, although the cost changes based on the weight of the affected individual.
5. PTC spokeswoman Jane Baj told the Wall Street Journal the price hike of nine percent will help finance research and development and the costs of providing free medications to patients in need. PTC ensures patients pay little money out of pocket for the drug by offering discounts on the list price, Ms. Baj added.
6. As of November, 1,500 U.S. patients are using the drug.
7. The drugmaker expects sales of Emflaza to reach between $90 million to $100 million in 2018.
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