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WASHINGTON (AP) — Federal health regulators remain unconvinced about the benefits of a closely watched experimental drug for the debilitating illness known as Lou Gehrig’s disease, even as they prepare to give its drugmaker a rare second opportunity to make a public case for the treatment.
Amylyx
Pharmaceuticals’ experimental drug has become a rallying cause for patients
with the deadly neurodegenerative disease, their families and members of
Congress who are pushing the FDA to approve the drug.
But
regulators said Friday that the drugmaker’s new analyses are not “sufficiently
independent or persuasive” to establish effectiveness. The agency posted its
review ahead of a Wednesday meeting of its outside advisers, who will vote on
whether to recommend approval.
In March,
the same panel of neurological experts voted 6-4 that the company’s data failed
to show a convincing benefit for ALS, or amyotrophic lateral sclerosis. It’s
extremely rare for the FDA to call a second review meeting after its advisers
have already voted.
The FDA will
ask the panel to review several new statistical analyses, which the company
says strengthen the case that its drug prolongs life and delays hospitalization
and other severe complications. The FDA says the experts can take into account
“the unmet need in ALS,” the disease’s seriousness and other factors specific
to the terminal diseases.
Elsewhere in
its review the FDA detailed the flexibility it can apply to drug approval
decisions, particularly for deadly diseases, which suggests “there is a chance
that the FDA is still looking for a way to approve the product,” SVB analyst
Marc Goodman wrote in a note to investors. He gives Amylyx a 50% chance of
approval.
ALS destroys
nerve cells needed to walk, talk, swallow and — eventually — breathe. There is
no cure and most people die within three to five years.
The FDA’s
review reflects some of the biggest questions facing the agency, including: How
strict should it be in enforcing approval standards for drugs against rare,
fatal diseases? And how much weight, if any, should be given to outside appeals
from patients, advocates and their political allies?
Typically,
FDA approval requires two large studies or one study with a “very persuasive”
effect on survival.
Amylyx’s
data comes from one small, mid-stage trial that showed some benefit in slowing
the disease, but which was marred by missing data, implementation errors and
other problems, according to FDA reviewers.
But FDA said
Friday the new approach “suffers from the same interpretability challenges” as
Amylyx’s initial study and that the new analysis “is not independent data.”
The FDA does
not publicly explain its rationale for holding meetings. But some outside
analysts believe the agency is hoping that more external input will strengthen
its hand when it renders its final decision, expected by the end of the month.
Amylyx’s
drug is a combination of two older drug ingredients: a prescription medication
for liver disorders and a dietary supplement associated with traditional
Chinese medicine. The Cambridge, Massachusetts, company has patented the
combination and says the chemicals work together to shield cells from premature
death. Its co-founders first hit upon the combination as Brown University
students.
Some ALS patients
already take both pills. FDA approval would likely compel insurers to cover the
treatment.
The FDA will
hear again from patients and advocacy groups, such as I AM ALS, which has
lobbied the FDA and Congress for more than two years to make the drug available.
The group’s founder, Brian Wallach, said ALS patients, physicians and
researchers believe that the company’s data warrants approval.
“Patients do
their homework— we know this isn’t going to cure us,” said Wallach, who was
diagnosed with ALS in 2017 and spoke through an interpreter. “But we also know
it might keep us here until the next drug comes along and that one might be a
cure.”
Wallach
currently takes the part of Amylyx’s treatment that is available as a dietary
supplement.
Despite the
negative FDA review, there are several outside developments that could tip the
FDA toward approval.
In June,
Canadian regulators approved the drug for ALS patients, the first country to do
so. That decision puts FDA regulators in a “precarious position,” says bioethicist
Holly Fernandez-Lynch.
“They
typically like to be out ahead when making approval decisions,” said
Fernandez-Lynch, who teaches at the University of Pennsylvania. “They like to
make the argument that they are not a barrier to patients accessing things that
might help them.”
Shares of
Amylyx fell more than 23% to close at $18 in trading Friday.
Follow
Matthew Perrone on Twitter: @AP_FDAwriter
The
Associated Press Health and Science Department receives support from the Howard
Hughes Medical Institute’s Department of Science Education. The AP is solely
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