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GSK says it will move "as quickly as possible"

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Regulatory experts say the maneuver bypasses usual,

lengthier

processes

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Autism experts say more trials are needed

By Patrick Wingrove, Maggie Fick and Julie Steenhuysen

Oct 6 (Reuters) - U.S. Health Secretary Robert F Kennedy

Jr. could deliver a policy win for the Trump administration in

just a few months after the Food and Drug Administration

enlisted GSK to help it fast-track approval of a

decades-old drug to treat an autism-related disorder.

The FDA's unusual move will allow it to bypass a lengthy

label update for generic versions of the drug, leucovorin, or

new clinical trials, a tactic academics, lawyers and doctors

questioned.

A GSK spokesperson told Reuters it plans to complete the new

use application for the branded version of leucovorin "as

quickly as possible."

Once the British drugmaker does that work, the FDA would

normally take about four to six months but could process the

request even faster, said Giuseppe Randazzo of the Association

for Accessible Medicines, a generic medicines lobby group.

The accelerated process will give doctors additional

justification to prescribe the drug for cerebral folate

deficiency, a metabolic disorder that can lead to a range of

neurological symptoms including some associated with autism,

delivering on Kennedy's promise to President Donald Trump and

the "Make America Healthy Again" movement with which he is

aligned.

Without robust evidence, the label change represents at most

a hollow bureaucratic victory, said Ameet Sarpatwari, a

pharmaceutical policy researcher at Harvard Medical School.

However, the drug, which is used to mitigate toxic effects

of certain cancer treatments and sells for $34.14 for a bottle

of 30 high-dose pills on Cost Plus Drugs, would more likely be

covered for the condition by insurance plans with the label

change.

An HHS spokesperson said the evidence clearly supports

leucovorin's ability to address the causes of cerebral folate

deficiency and improve patient outcomes.

DEMAND RISES AFTER TRUMP PROMOTES DRUG

Demand for the drug has increased, first after a February

CBS story about its use in a nonverbal five-year-old boy, and

more recently after Trump promoted its use.

"My nurses have been saying the phone is ringing off the

hook," said Dr. Larry Gray, an expert in developmental and

behavioral pediatrics, who sees patients with autism at Lurie

Children's Hospital of Chicago.

Because the treatment is not approved for autism, the

institution's policy has been to only offer it in clinical

trials, which are rare. The drug is FDA-approved, however, so

doctors can prescribe it off-label.

Kennedy has declared the rising rates of autism in the U.S.,

now estimated at 1 in 31 children by age 8, to be an epidemic

and had pledged to find some answers behind its cause as well as

cures by September.

At a White House event on September 22, Kennedy, Trump and

other health officials backed leucovorin as an autism treatment.

They also warned against the use of Tylenol by pregnant women,

saying studies suggested a link to autism. Health experts and

medical groups called that warning dangerous and without sound

scientific basis.

RELYING ON OBSCURE RULE

The FDA was able to speed the process by using an obscure

rule to reinstate GSK's approval application and request a label

update adding cerebral folate deficiency, based on the agency's

own analysis of 40 patient cases found in a review of literature

from 2009 to 2024.

GSK sold the drug as Wellcovorin until 1997. A generic

version, which is also called folinic acid and is a form of

folate or vitamin B9, is now made by U.K.-based Hikma.

Once GSK's application is approved, U.S. law requires

generic drugmakers to match the change.

The more commonly used label update process for generic

drugs, which requires consultation with generic drugmakers,

typically takes up to a year and a half, according to Skadden

lawyer Rachel Turow. It is typically used for cancer drugs after

new uses are proven in clinical trials, she and several other

lawyers said.

Aaron Kesselheim, professor of medicine at Harvard Medical

School, described the process being used as "very atypical," and

said that without the FDA sharing its data or trials, it is hard

to know if the agency is following the normal standard of

evidence.

LIMITED AVAILABLE EVIDENCE

Dr. Andy Shih, chief science officer at the advocacy

organization Autism Speaks, said the evidence for leucovorin's

use was limited and potentially suggestive of benefit for a

small subgroup of autistic children. Larger trials are needed,

he said.

The evidence is based on four studies, each of which

involved 50 to 60 patients, with three of them done by the same

author, said Dr. Karam Radwan, director of the

Neurodevelopmental Disorders Program at the University of

Chicago, who uses the drug in his practice.

"You want to replicate that with a different lab, in a

different setting, to make sure we have enough support" for the

change, he said.

Three mid-stage trials are underway studying a new, liquid

version of leucovorin as an early language impairment treatment

for children with autism, according to the government clinical

trials site. The earliest data is expected around December.

The trials are being led by one autism researcher in

partnership with the National Institutes of Health, the

Department of Defense, and Autism Speaks, and involve up to 80

children each.

Larger, more conclusive trials would take years. The FDA's

approach does not require new trials.

This change should be based on scientific evidence, and so

far, studies supporting its use are not robust, Radwan said.