- Amylyx Pharmaceuticals’ experimental ALS drug would “far exceed” common cost-effectiveness measures if the company prices it on par with another currently available treatment for the neurodegenerative disease, the Institute for Clinical and Economic Review suggested in a draft report released Tuesday.
- The drug, known as Albrioza or AMX0035, on Monday received a conditional approval in Canada, although it won’t be available there for several more weeks and Amylyx has not yet disclosed its price. It’s also currently under review by the Food and Drug Administration, which expects to make a decision by Sept. 29.
- ICER, a nonprofit watchdog group, has become influential in the drug pricing debate, although it’s been criticized by pharmaceutical companies and others for how it conducts its analysis. The group is now accepting comments on the report, which it plans to finalize in early August with more definitive assessments on pricing and cost-effectiveness.
Amylyx’s drug is closely watched, given the limited options available for ALS and the disease’s devastating effects. Testing showed treatment delivered modest benefits on a rating scale used to measure function, as well as improved survival by about five months.
But the trial was small, enrolling 140 participants, and had a number of design flaws that FDA scientists flagged in documents prepared for a meeting held in March. Then, advisers to the agency narrowly voted against approval for AMX0035. The FDA doesn’t have to follow their recommendations, however.
While it’s unclear how the FDA might act, Amylyx already has its first approval with Canadian regulators’ decision earlier this week.
ICER, which regularly reviews the evidence and cost of new or under review drugs, began assessing ALS treatments back in February. Its report focuses on AMX0035, as well as a medicine from Mitsubishi Tanabe Pharma called Radicava that’s been on the market since 2017, but was recently cleared in a new oral formulation.
ICER flagged a range of uncertainties with the evidence supporting both drugs, although it concluded each has “minimal” harms. It also found the data underlying AMX0035 more convincing than that collected for oral Radicava.
Yet the group added that its rating was “tempered” by the small size of Amylyx’s trial, lower efficacy on stricter analyses and apparent lack of survival benefit over the first six months of treatment.
“The evidence for AMX0035 comes from a single small [trial] and its extension study,” ICER wrote in its report, which could change when finalized later this summer. “Clinical experts are divided on whether AMX0035 is effective. Nearly all, whether they favored FDA approval or not, felt that only an additional [trial] would answer whether AMX0035 actually affects disease progression and survival in ALS.”
In assessing cost-effectiveness, ICER used a placeholder price for AMX0035, assuming it might cost as much as the older, infused version of Radicava, which has a list price of $171,000 a year. At that price, however, AMX0035 would surpass ICER’s commonly used thresholds for assessing cost effectiveness.
The group did not formally set a price benchmark for the drug in its draft report and cautioned that it might differ from its threshold analyses.
Still, ICER suggested a lower price for AMX0035, if approved, until Amylyx obtains further evidence supporting its effectiveness in a larger, confirmatory study that’s currently underway.
In a statement, ICER’s Chief Medical Officer David Rind also noted how the group used additional measures to better assess the potential value of treatment.
“Throughout this review process, we have heard concerns from patient groups that cost-effectiveness analyses can underestimate the value that people with ALS place on therapies that extend life,” Rind said. ICER’s additional assessment “does not undervalue therapies for ALS,” he added.
Amylyx said in a statement that, if U.S. approval is granted, the company would “work closely with payers on coverage, reimbursement, and access to AMX0035 for people living with ALS in the U.S.”
Editor’s note: This story has been updated to include comment from Amylyx provided to BioPharma Dive after publication.