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This drug could be life-saving, but it'll cost you $2.125 million

Posted at 4:03 PM, Oct 31, 2019
and last updated 2019-10-31 19:34:54-04

BOISE — On Wednesday, the FDA halted enrollment in a trial testing of a drug for patients with spinal muscular atrophy older than the age of two, delaying plans to expand the treatment option to affected individuals.

The drug is currently approved for patients younger than two years of age and may be the closest thing to a cure.

“It is really exciting; it is a potential cure for Spinal Muscular Atrophy," said Dr. Jackie Whitesell, a neurologist at Saint Alphonsus.

The drug Zolgensma is a potentially long-term fix to the motor neuron problems people have when diagnosed with SMA. What’s not exciting is the price tag. At wholesale it is $2.12 million dollars, or $425,000 per year for five years.

“It is probably the most expensive drug on the market at this point, so there is a lot of unknowns when it comes to prescribing it," said Whitesell.

That multi-million dollar price tag is steep, but Whitesell predicts the insurance approval process will be similar to the alternative drug treatment they’re currently using—Spinraza—which was no walk in the park.

“When Spinraza first came to the market, we had difficulty getting it approved through people’s insurance and had a lot of denials, so we had to go through the appeal process and resubmit," said Whitsell.

"We’ve actually developed this monitoring program here where we’re following patients with physical therapy evaluations every four months to show their improvement, and that’s helped them get the medication approved."

Whitesell only sees a handful of patients with SMA, but she has seen results with the similar drug.

“Patients are noticing that they have gained motor function. We have one patient who now is able to stand and walk a little bit, and previously he was just in a wheelchair," said Whitesell.

Zolgensma would be a more long-term solution by comparison. However, the drug company has a few red flags to overcome before insurance even comes into the equation. FDA recently halted a recent trial phase, which would be vital for expanding it to patients over the age of two. If FDA approval comes for adults, then the focus shifts back.