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The Food and Drug Administration approved the first drug to treat patients with the most common childhood form of muscular dystrophy, a vivid example of the growing power that patients and their advocates wield over the federal government’s evaluation of drugs. The agency’s approval went against the recommendation of its experts. The main clinical trial of the drug was small, involving only 12 boys with the disease known as Duchenne muscular dystrophy, and did not have an adequate control group of boys who had the disease but did not take the drug. A group of independent experts convened by the agency this spring said there was not enough evidence that it was effective. (Tavernise, 9/19)

The circumstances of the approval at the agency were highly unusual, and included sharp internal protests that were ultimately resolved by FDA Commissioner Dr. Robert M. Califf. An advisory committee to the FDA in April voted 7-3, with 3 abstentions, that the data for the drug weren't enough for agency approval. ... Sarepta officials said Monday that the drug’s cost would be based on the patient’s weight. For the average-size child, the company said, the annual cost would be $300,000. (Burton, 9/19)

Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients. It is designed for the 13 percent of patients with a particular genetic mutation that prevents them from making dystrophin, a key protein that keeps muscles intact. Without that protein, muscles weaken so that children are unable to walk and must use wheelchairs by the time they’re teens. Eventually, the disease can fatally weaken the heart and muscles needed to breathe. Patients often die in their 20s or 30s. (Szabo, 9/19)

Approval of the medicine, which will be sold under the brand name Exondys 51 starting as early as this fall, came after hundreds of patients and their families lobbied for a new approach, packing an emotionally charged FDA advisory committee meeting in April to clamor for Sarepta’s experimental treatment. “These kids have hope now,” said Christine McSherry of Pembroke, whose 20-year-old son Jett benefitted from Sarepta’s treatment, called eteplirsen, in the clinical trial. “It’s wonderful for the next generation of kids with this terrible disease. It opens the door for more research and more treatments for Duchenne and other genetic diseases.” (Weisman, 9/19)

“It sets a precedent for patient advocate involvement and potential power; it sets a precedent for the degree of flexibility that FDA can show if they want to,” said Ritu Baral, an analyst at Cowen & Co. The decision makes rare disease drug development more unpredictable, she said. (Cortez and Edney, 9/19)

The newly approved Sarepta Therapeutics drug for Duchenne muscular dystrophy will cost about $300,000 a year for the average patient in the United States, which is less than what some Wall Street analysts had expected. Sarepta CEO Dr. Edward Kaye said the pricing was “in the middle of the range” for rare disease drugs. “And given the sensitivity to pricing, we tried to be reasonable when looking at all the costs,” he said on a phone call Monday with securities analysts. (Silverman, 9/19)

Max and Austin Leclaire have lived a lot of hard days together. The brothers have Duchenne muscular dystrophy, a rare degenerative disease that wastes their muscles and will likely take their lives at an early age. Over the last few years, they found glimmer of hope in an experimental drug, called eteplirsen. Max was able to get on the drug in 2011. Austin started on it three years later in another clinical trial. Both boys believe it has helped slow their physical decline. And Max has remained able to walk, although not easily, beyond the age when boys with Duchenne usually lose the use of their legs. (Delviscio, 9/20)

The run-up to Monday’s approval of a Sarepta Therapeutics drug to treat Duchenne muscular dystrophy was marked by unusual bickering inside the Food and Drug Administration, where debate over a key scientific question morphed into a formal dispute, and the head of the drug review division was accused of being too intensely involved in the process for evaluating the medicine.Ultimately, the decision to greenlight the drug fell to the FDA Commissioner, Dr. Robert Califf. (Silverman, 9/19)

The experimental drug that federal regulators approved Monday will only be used by a few thousand patients.But the approval may have set a precedent that could rocket through the health care system, opening the door for drug makers to get more medicines to market — even with scant evidence that they work. The Food and Drug Administration’s decision elated families struggling with Duchenne muscular dystrophy, a rare and deadly disease. It sent the stock of the drug maker, Sarepta Therapeutics, soaring. (Garde, 9/19)

She’s been attacked by parents of dying children, vilified by consumer groups, and grilled by Congress, but over the past 30 years, Dr. Janet Woodcock has risen to become the top drug cop at the Food and Drug Administration — and managed to stay there. “She sticks to her guns,” said Marc Boutin, chief executive officer of the National Health Council, a coalition of patient advocates, pharmaceutical companies, and others. “She’s tough as nails.” That may be an understatement. (Kaplan, 9/20)