Senators Demand Answers on Pricey DMD Drug

Company accused of “gaming” orphan drug program

A few weeks ago, Jeffrey Aronin, CEO of Marathon Pharmaceuticals, announced his decision to charge $89,000 per year for deflazacort––a cheap, generic steroid––for patients with Duchenne muscular dystrophy. Now a group of irate senators wants some answers.

In a letter to Aronin, seven Democrats and one Independent asked him to explain his company’s pricing scheme for deflazacort (Emflaza), noting that the drug has been used internationally to treat patients with DMD for decades. Moreover, U.S. patients with DMD have been importing deflazacort from Canada and Europe for as little as $1,000 per year.

Further, the letter states, Marathon executives have defended the price of the drug, arguing that other treatments for orphan diseases cost more than $300,000. Marathon’s chief financial officer has said that Emflaza is “modestly priced for an orphan drug.”

“While this is true,” the senators point out, “orphan drugs for rare diseases generally are not old compounds that have been used to treat such rare disease for decades.”

The FDA approved Emflaza as a new drug, making Marathon eligible for benefits under the Orphan Drug Act. These benefits include a seven-year monopoly on the DMD indication for deflazacort, even though the drug has long been available as a generic in other countries. Moreover, Marathon received a rare disease priority review voucher, which allows companies that gain approval for pediatric orphan drugs to demand faster approval for another drug. Marathon can either use the voucher for itself or sell it to another company. The vouchers have been sold for up to $350 million.

“We are concerned that Marathon’s pricing unfairly exploits the DMD population and the FDA’s orphan drug incentives,” the senators wrote. They requested answers to several questions by March 13. These questions included:

·         How much did Marathon pay to obtain the deflazacort clinical trial data?

·         How much did Marathon spend to analyze the clinical trial data and to collect additional preclinical and clinical trial data before FDA approval?

·         How much does Marathon anticipate earning from the seven-year monopoly on the DMD indication for deflazacort?

·         How much does Marathon anticipate spending on post-market studies of deflazacort?

As critics began to paint him as the new poster boy for price gouging, Aronin suddenly postponed the launch of Emflaza, saying it was time to reflect on the price and to consult with stakeholders. Since then, he has refused to hold interviews or to make public appearances.

Ironically, Aronin, like the infamous Martin Shkreli before him, is a member of the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), which is spending millions of dollars in a public relations campaign to distance its members from recent drug-pricing scandals.

Sources: Senate Letter; March 3, 2017; and Endpoint News; March 6, 2017.