On Tuesday, the FDA granted accelerated approval to the drug, tofersen, meaning others with his type of mutation will be able to get the treatment, too — if insurers agree to pay.
The news was cheered by people with the deadly disease, even though most of them have forms of the disease for which the drug will not work. Most cases have no known genetic cause. About 10% of cases are caused by a genetic mutation, and tofersen is designed to treat a fraction of those. And even for those who do qualify for the treatment, there is debate over how effective it is.
“That’s the difference between him being alive today and not,” Quinn said. “Which is so scary to think about. I actually try not to think about it.”In patients with Legg’s form of ALS, the genetic mutation causes them to make an abnormal, toxic version of a protein called SOD1. As the protein accumulates in the central nervous system, it causes deterioration in the nerves that control muscles, which start to wither away as a result. Patients — currently numbering several hundred in the U.S.
Legg, a high-school math teacher who came to Penn on Tuesday for his 36th injection, likes to repeat an analogy that an acquaintance came up with: “I was 47 years old,” he told the panel, speaking via teleconference. “I wasn’t sure if I was going to make it to 50. And here I am.”FDA’s process for approving certain drugs without conclusive evidence has sparked controversy.
He cited the fact that after the first six months of the trial, participants who had been taking the placebo switched to getting the real drug. And in the two years since then, their condition appears to have stabilized somewhat.
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