CEO defends $300,000-per-year cost for a drug for a rare form of muscular dystrophy

  • Sarepta's Doug Ingram responds to a question about the high cost for a drug for Duchenne muscular dystrophy.
  • "We have already invested $1 billion fighting" this rare disease, he says. "And we're not done yet."
  • Shares of Sarepta soar after the company reported positive results from a clinical trial of another experimental DMD medicine.
Doug Ingram, CEO, Sarepta
Source: CNBC
Doug Ingram, CEO, Sarepta

Sarepta Therapeutics CEO Doug Ingram told CNBC on Wednesday that accessing reimbursement from insurance companies for expensive, rare disease treatments is "always challenging."

But responding to a question about the $300,000-per-year cost for a Sarepta drug for Duchenne muscular dystrophy, Ingram defended the price tag.

"Sarepta is a small company. We have already invested $1 billion fighting Duchenne muscular dystrophy. And we're not done yet," he said on "Squawk Box."

DMD occurs in 1 in every 3,500 to 5,000 males worldwide. Symptoms generally start in early childhood, usually between ages 3 and 5. It rarely affects girls.

Exondys 51 was approved for DMD on a conditional basis by the Food and Drug Administration last year, pending more testing to confirm results. It treats a mutation affecting about 13 percent of sufferers. The cost per-year is about $300,000 for a 55-pound patient. The price goes up for heavier recipients.

"This a societal question more than a Sarepta question," Ingram said. "I think it's the right decision for society to invest in finding solutions for these rare disorders."

Shares of Sarepta, which had a stock market value of $2.6 billion as of Tuesday's close on Wall Street, soared 9 percent Wednesday morning. It had risen 17 percent in premarket trading after the biopharma firm reported positive results from a clinical trial of another experimental DMD medicine. The treatment, golodirsen, targets a genetic mutation affecting about 8 percent of patients.

"Our goal is to treat 100 percent" of DMD suffers, said Ingram, who was appointed CEO of Sarepta in July. "The data that we have this morning shows we're on the right path."

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