Urgent pleas from parents greet biotech's new drug

By Rami Grunbaum

Seattle Times deputy business editor


There's a hard, urgent logic to Jenn Mcnary's plea: Her 13-year-old son, Austin, is wasting away from an incurable genetic disease, while her 10-year-old with the same disorder has been helped by testing a cutting-edge drug that's not yet approved for sale.

"I would love to get him on this medication to give him as good a chance as Max has gotten," the Vermont mom says. "At this stage anything that can stop the progression is a lifesaver."

There's an equally compelling logic to the strategy of Sarepta Therapeutics CEO Chris Garabedian, who recently reported clinical-trial results that suggest the drug may be the first effective treatment for slowing Duchenne muscular dystrophy.

"We could spend all our time answering phone calls from parents who would like our drug, and putting in the paperwork to the FDA" for individual "compassionate use" cases in which Sarepta can't charge money, he says. "That is the surest path to insolvency."

Sarepta -- until recently called AVI BioPharma and based in Bothell but now headquartered outside Boston -- may be one of those biotech fairy tales that sustains belief in the entire industry: A little company that brings a novel lifesaving drug to market.

But its steps toward that goal can't come fast enough for parents whose sons are losing the ability to move their own feet. It makes for a complicated relationship.

The degenerative Duchenne disorder, which affects an estimated 13,000 U.S. boys with a faulty X chromosome, usually leads to loss of mobility in the teens, then difficulty in breathing and eventually heart failure, usually by age 30.

The current version of Sarepta's treatment targets a subset of about 15 percent of that population, making it an "ultra-orphan" drug.

Sarepta in late July unveiled the results from a 36-week Phase 2 clinical trial of the drug, Eteplirsen, in 12 boys aged 7 to 13. The principal investigator on the trial, at Nationwide Children's Hospital in Columbus, Ohio, called the drug's effect "unprecedented."

That set off a surge in shares of Sarepta, which shot from $3.46 to a high above $15 over six weeks.

Duchenne's usual progress is so rapid that the clinical trial's key measure of success was how much patients' ability to walk dwindled.

For boys treated with the drug over 36 weeks, the median decline in a "6-minute walk test" was only 8.7 meters; those who received a placebo or delayed treatment with the drug declined by 78 meters.

Mcnary reports that the drug did more than delay deterioration in Max, a fourth-grader "about half the size" of his classmates. He's gained energy as well as strength and coordination in his upper body, she says.

Previously unable to climb into the family van, now "he's opening up the side door and jumping in himself," she says. "He's jumpy and bubbly."

Max had been using a wheelchair to move between classrooms at school, but now he's walking on his own -- "and running," she says.

That only makes it harder for Mcnary to think about when her older son may get access to the drug.

"I think he possibly needs it even more than Max," she says. "I don't have time to wait even for accelerated approval."

Accelerated approval is a fast-track process that Congress and patient groups have encouraged the FDA to use for drugs that show some evidence of effectiveness in diseases where there is no alternative and nothing to lose.

It may be faster than the usual years-long rounds of ever-larger clinical trials and slow, deliberate regulatory consideration, but it's hardly speedy.
More data
If another round of data to be revealed by researchers next month is as positive as the July results, Garabedian says he will seek a meeting with the FDA about qualifying for fast-track approval. Then it would still take months to prepare the application, and months more to hear about approval.

Meanwhile, he's gearing up contracting arrangements to manufacture more of the drug -- aided by a $40 million financing the company announced this past week.

But Garabedian says there simply won't be enough of the costly and complex product to do more than treat the patients in the clinical trials. He can't afford to make it and give it away to needy cases.

"Every drug that we divert to doing something that doesn't support FDA approval is not money well spent," he says.

He understands the parents' sense of urgency, says Garabedian, but the company needs to put all its resources into gaining the FDA's OK "so we can get reimbursement and produce the drug for every family or every boy who can benefit, rather than the select few who have the loudest voices or the most impassioned story."

Parents and patients will be crucial to making that case to the FDA, he says: "Here are parents who are willing to sign waivers that say, 'I don't care that this drug hasn't been tested fully ... because I know the alternative."

Advocacy groups are looking for other avenues to speed the drug's progress, too.

Christine McSherry, a Boston mother who founded the Duchenne Alliance, says the umbrella organization is talking to Sarepta about ways to support additional testing that would bring the drug to more advanced cases, such as her son.

"I don't see money being the obstacle in funding a clinical trial," she says. "I think you'll have plenty of parents who will come forward with dollars."



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