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US drug pricing watchdog will review Sarepta's Duchenne muscular dystrophy drugs

The U.S. drug pricing watchdog Institute for Clinical and Economic Review will publish a report analyzing the cost of Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy drug Exondys 51 and its upcoming follow-up golodirsen.

Duchenne muscular dystrophy is a genetic disorder, caused by the absence of a protein called dystrophin, characterized by progressive muscle degeneration and weakness.

The institute, known as ICER, uses a measure called quality-adjusted life years to compare a drug's cost to its effectiveness in improving patients' lives.

The institute's report will be the subject of a July 2019 meeting of the New England Comparative Effectiveness Public Advisory Council. ICER will release a draft scoping document Jan. 11, 2019, with more details of the planned analysis and is accepting public comment until Jan. 8.

Sarepta's Exondys 51 had sales of $78.5 million in the third quarter of 2018 and has been controversial for its price tag, prompting some payers to refuse coverage. But when it was approved in 2016, it was the first drug to ever treat Duchenne muscular dystrophy.

Golodirsen is Sarepta's follow-up to Exondys 51, treating the same disease. It is undergoing a rolling FDA submission with approval expected in 2019, CEO Douglas Ingram said on the company's Oct. 24 earnings call.