FDA Approves ALS Drug on Phase 2 Data

On 28 September, the U.S. FDA approved Relyvrio, a drug for amyotrophic lateral sclerosis that by its sponsor's own admission may or may not work. Amylyx Pharmaceuticals' AMX0035 is a fixed-dose combination of the supplement taurursodiol and of phenylbutyrate, a generic drug for urea cycle disorders. In a Phase 2 trial, it seemed to slow disease progression (September 2020 news).

The trial data failed to persuade an independent advisory panel, which last March voted 6 to 4 not to approve the drug without further testing. In an unusual move, the FDA reconvened the advisory committee for another look at essentially the same data. This time, the committee voted 7 to 2 for approval after extracting a pledge from the company's CEO that he would remove the drug from the market if an ongoing Phase 3 trial turns out negative (Endpoint News).

In a summary brief, Emily Freilich, Teresa Buracchio, and Billy Dunn from the FDA note “a degree of residual uncertainty about the evidence of effectiveness that exceeds that which might typically remain following a conclusion that substantial evidence of effectiveness has been demonstrated.” In short, they seem unconvinced that the drug works.

The FDA had initially asked Amylyx to run a Phase 3 trial, but that met with a torrent of complaints from patients and advocacy groups who are desperate for a treatment. ALS progresses rapidly, leading to paralysis and death usually in two to five years of symptom onset.

The agency's decision is mired in controversy, stemming from the lack of Phase 3 data and doubt over the company’s willingness to voluntarily withdraw the drug should the ongoing Phase 3 trial show no efficacy. Yesterday, Amylyx announced a list price of $158,000 per year. ICER, the Institute for Clinical and Economic Review, recently calculated that a fair value would be between $9,100 and $30,700 per year.

All this has raised the question of just how much power advocacy groups hold over the FDA (see reports from The New York Times and Endpoints News).

Canadian regulators conditionally approved the drug last June.—Tom Fagan

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References

News Citations

  1. Micro Nudge? Positive Phase 2 Results for ALS Combination Drug

External Citations

  1. Endpoint News
  2. fair value
  3. The New York Times
  4. Endpoints News

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