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US price group to include PTC Therapeutics' Emflaza in Duchenne drug review

U.S. drug pricing watchdog Institute for Clinical and Economic Review will include PTC Therapeutics Inc.'s Emflaza in its ongoing price review of several Duchenne muscular dystrophy drugs.

Duchenne muscular dystrophy, or DMD, is a genetic disorder characterized by progressive muscle degeneration and weakness. The disease is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.

The institute, also known as ICER, said December 2018 that it will analyze the cost of Sarepta Therapeutics Inc.'s DMD therapy Exondys 51 and its upcoming follow-up golodirsen. The nonprofit institution uses a measure called quality-adjusted life years to compare a drug's cost to its effectiveness in improving patients' lives.

The institute said its report on the medicines will be the subject of a July meeting of the New England Comparative Effectiveness Public Advisory Council. ICER will accept public comments on the draft scoping document until Feb. 1.

In February 2017, Marathon Pharmaceuticals LLC received U.S. marketing approval for Emflaza, or deflazacort, but delayed the launch of Emflaza after lawmakers criticized the drug's $89,000 list price. The drug was available in Canada and certain European nations for about $1,000 annually.

Marathon sold the drug to PTC Therapeutics in a deal which closed April 2017. In May that year, South Plainfield, N.J.-based PTC lowered Emflaza's list price to $35,000 annually.

In January 2018, The Wall Street Journal reported that PTC Therapeutics raised the list price of Emflaza to more than $65,000 annually.