Biotech Values

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SRPT: An article from my local paper; the Center for Gene Therapy at Nationwide Children’s Hospital is here in Columbus.

Drug showing promise with MD
Company might get an expedited approval by FDA

http://www.dispatch.com/content/stories/local/2012/10/05/drug-showing-promise-with-md.html

By Misti Crane,The Columbus Dispatch
Friday October 5, 2012 6:26 AM

Parents of boys with Duchenne muscular dystrophy now have even more reason for hope.

The company that makes an experimental drug designed to treat about 13 percent of the approximately 12,000 U.S. boys with the disease has released new results from a small but important study that shows significant benefit from the drug eteplirsen.

Dr. Jerry Mendell, director of the Center for Gene Therapy at Nationwide Children’s Hospital’s research institute, is the lead researcher on the study of 12 boys with the debilitating, and ultimately deadly, disease.

He and the company, Sarepta Therapeutics, previously shared optimistic news after observing the boys’ health about nine months into treatment with a weekly infusion of the drug.

Now, they say they have seen significant benefit after almost a year. Sarepta Therapeutics said this week that the drug increases dystrophin, a vital protein missing in boys with the genetic disorder. It also slowed progression of the disease, as measured by how far the boys could walk in six minutes.

The four boys who had the strongest dose for 48 weeks were able to walk an average of about 23 yards farther than when they began taking the drug. Those who received a placebo for 24 weeks followed by 24 weeks of treatment lost about 74 yards.

The 97-yard difference is profound in a disease that forces most boys into wheelchairs before they reach their teens.

What remains unclear is how the drug performs in the long term and whether there are side effects.

Mendell said he’s cautious about extrapolating more from the data than what is reasonable, but he suggested that signs are pointing toward increased benefit the longer patients are on the drug and more benefit when it’s started at a younger age.

And he’s grateful to be seeing something positive after decades of trying to help boys with Duchenne muscular dystrophy.

“Whoever created this disease was not very gentle with these boys ... and made every obstacle possible to make it difficult to get a clinical effect (with a treatment),” he said.

On a broader level, Mendell and company executives have said that success with eteplirsen bodes well for similar drugs designed to treat boys with other genetic variations of Duchenne muscular dystrophy.

“Any treatment that shows benefit in this condition is wonderful and amazing,” said Dr. Kenneth Silver, a pediatric neurologist with the University of Chicago and Comer Children’s Hospital. “If something like this can really reverse some of the weakness, it’s quite dramatic.”

The company plans to meet soon with the U.S. Food and Drug Administration to discuss the future of the drug. Speedy approval of eteplirsen before conducting a larger, multicenter trial is a possibility.

It’s an exception sometimes made in cases where the benefit is judged to exceed any potential risks and other unknowns that have yet to be brought to light.

Many parents have argued that this case is one in which fast approval is warranted considering that, in a matter of months, their sons could lose the ability to walk and, for now, face a certain early death.

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