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“Our Son’s Fate”: Parents Fighting for Kids’ Spine Drug Eye New Data

Xconomy Boston — 

Melissa Marotta pulled no punches to get her three-year-old son C.J., who has the genetic disease spinal muscular atrophy, access to nusinersen (Spinraza), the first approved drug for his condition. For months, she couldn’t schedule a treatment date at Columbia University Medical Center/New York-Presbyterian Morgan Stanley Children’s Hospital, and for much of that time, she didn’t know why.

She wrote every day to the hospital board until a breakthrough came. C.J. (pictured) received his first dose in early April.

“I reminded them that our son’s fate was in their hands and that I held them fully responsible for the decline in his health,” she says. “While I’m thrilled that they figured it out, it took way too long.”

C.J. has Type 3 SMA. He could walk normally until about 14 months old, when he started to fall frequently. He used to stand up by himself, using his hands to push off his thighs, but now he has to grab something to stay up. In the photo above—taken to show his fight against the disease—he could only stand for a few seconds without falling, according to his mother. “He’s a little bit weaker,” Marotta says.

C.J. was easily cleared by insurer UnitedHealthCare months ago but didn’t receive treatment until April 7. Columbia’s eventual explanation, said Marotta, was that stocking the drug was complicated by its high price.

Xconomy made several requests to Columbia but no one would comment by press time. New York-Presbyterian didn’t respond to requests for comment.

Marotta is one of many parents who have been at wit’s end about the delays and difficulties accessing nusinersen, from Cambridge, MA-based Biogen (NASDAQ: BIIB), which the FDA approved in December. For a small child with SMA, getting nusinersen—which may slow the disease’s progression—could mean more time being able to sit up, stand, or walk. The disease robs people of their ability to function independently, and, in the most severe types, usually kills the children who have it before the age of two. “The sooner you get [nusinersen] the better,” says Jahannaz Dastgir, a pediatric neurologist at Goryeb Children’s Hospital in Morristown, NJ.

That’s why the SMA community of patients, their parents, other caregivers, and doctors—not to mention insurers—will be watching closely next week when Biogen presents new clinical data for nusinersen at the American Academy of Neurology’s annual meeting in Boston.

The data, from a study called CHERISH, could help make nusinersen more widely available. Though the FDA approved nusinersen for all children and adults with SMA, broad approval hasn’t equaled broad access. That’s because when the FDA made its decision, the most extensive, published data available on nusinersen was from trials of Type 1 SMA patients. Full data from CHERISH, a study of patients with Type 2 SMA, wasn’t available yet. Without such data, insurers have had wiggle room to write policies restricting access. Some insurers, such as Anthem, have indicated they could update their policies when they see more data. Biogen will present at least some of that data—the full results of the CHERISH study—at AAN.

Much of the difficulty stems from the high price. Nusinersen is a chronic therapy with a list price of $750,000 for the first year, and $375,000 each year after that, and insurers have balked. But it’s more complicated than that.

“There are still so many people struggling to get this drug.” says Marotta. “We had to claw our way to get there.”

SMA affects anywhere from 10,000 to 25,000 people in the U.S., according to the nonprofit SMA Foundation. It varies in severity among the five forms, Type 0 through 4.

Type 0 patients show disease symptoms in utero, and like Type 1 patients, diagnosed as infants, often die at an early age. Type 2 progresses more slowly, though patients might never be able to walk. Type 3 patients can walk initially before losing strength later in life. Many SMA patients of all types have trouble breathing, sucking, and swallowing because of muscle weakness; the more severe cases are vulnerable to dangerous respiratory complications, like lung infections.

Some insurance policies favor one SMA type over another or require annual or bi-annual checkups to prove nusinersen is benefiting patients, which means patients have to re-apply for authorization every year. “Each patient is still an individual case,” says Stanford University neuromuscular neurologist Charles Cho.

Some insurers haven’t issued a policy at all; patients with that coverage must simply wait. Other insurers have initially denied coverage, and clinicians must battle through one appeal after another before getting clearance. Two similar patients with different insurers might have vastly different experiences.

“It’s been really hard to navigate,” says Dastgir, an investigator in several nusinersen trials.

It’s not clear at this point how many patients across the country have access to nusinersen. In March, Leerink Partners analyst Geoffrey Porges found “very few” SMA clinics that had actually treated patients. He warned Biogen investors to expect just $5 million to $10 million in nusinersen revenues this quarter. Biogen CEO Michel Vounatsos said in January that with reimbursement challenges and capacity constraints at treatment centers, the company expected a “gradual uptake” of the drug in 2017. Biogen will provide more details next week when it discloses quarterly results, according to spokesman Matt Fearer.

In recent interviews with Xconomy, two major treatment centers and one smaller clinic say … Next Page »

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