Lawmakers Question the FDA's "Unusual" Approval of Marathon Pharma's Controversial Old DMD Drug

Lawmakers Question the FDA's March 16, 2017
By Alex Keown, BioSpace.com Breaking News Staff

WASHINGTON – U.S. Sen. Bernie Sanders has once again set his sights on Northbrook, Ill.-based Marathon Pharmaceuticals. Sanders continues to hammer the company for the price of its Duchenne muscular dystrophy. Now he has raised questions about the federal approval process.

In a letter to the U.S. Food and Drug Administration (FDA), Sanders and U.S. Rep. Elijah Cummings, D-Maryland, expressed concern about the approval process behind Marathon’s treatment for patients with Duchenne muscular dystrophy. In February, Marathon won approval for Emflaza (deflazacort) to treat patients five years of age and older, regardless of their genetic mutation. Emflaza is the first drug approved in the U.S. for those patients. Sanders has been critical of Marathon’s proposed $89,000 price tag for Emflaza, particularly since Sanofi sells the same drug in Europe under the trade name Calcort for about $1,000. The pricing for the drug remains a concern for Sanders “in light of the incredibly lucrative benefits FDA has granted to Marathon and the limited amount of innovative research the company appears to have conducted to develop Emflaza."

The letter questions why Emflaza was given orphan drug status that gives the company “sales exclusivity—a monopoly on the marke—for seven years.” This is a point Sanders has made before. In a February letter to the company, Sanders said Marathon was “abusing” the orphan drug program so it could have “market exclusivity” for a drug that has been “available for decades.”

In the letter, Sanders and Cummings said most of the research the FDA relied upon for Emflaza’s approval was based on research from a 1995 clinical study. They questioned if it was standard practice for the FDA to rely upon efficacy data that was more than 20 years old. If so, the two wanted to know how often that has happened and if not, why was the approval of Emflaza an exception. The lawmakers also questioned why the FDA approved a brand name for deflazacort in the United States, since the drug does not have a patent protection.

Although available elsewhere, Marathon acquired the U.S. rights to deflazacort and conducted their own clinical trials to meet FDA approval standards—which was costly. Jeff Aronin, chairman and chief executive officer of Marathon, said in an open letter to the Duchene community, that the company invested “substantial” resources into developing the drug and does not expect to recoup its investment for several years.

Marathon has declined to comment on Sanders’ latest letter, the Chicago Tribune reported. A spokesperson for the FDA said the agency was reviewing the letter and will respond directly to Sanders and Cummings, the Tribune added.

Marathon may not comment as the company announced this morning that it intends to sell Emflaza to PTC Therapeutics for $140 million plus royalties. The sale followed Marathon’s decision to delay marketing of the drug following criticism of the pricing.

“PTC has deep-rooted experience with Duchenne and will build upon the progress Marathon has made to date in securing FDA approval for EMFLAZA and bringing it to the point of commercialization. PTC provides a broader platform for patient access and is committed to ongoing research to advance the knowledge, diagnosis and treatment of this rare and fatal genetic disorder,” Marathon said in a statement this morning.

Duchene is a fatal genetic disease. Most patients are diagnosed by the time they reach five years of age and live until their mid-20s. Duchene is a rare disease, so it qualifies for orphan status under the FDA. Most drugs developed to treat orphan diseases come with a high price tag.

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