F.D.A. Approves First Drug Designed to Prevent Migraines
Image
By Gina Kolata
The
first medicine designed to prevent migraines was approved by the Food
and Drug Administration on Thursday, ushering in what many experts
believe will be a new era in treatment for people who suffer the most
severe form of these headaches.
The
drug, Aimovig, made by Amgen and Novartis, is a monthly injection with a
device similar to an insulin pen. The list price will be $6,900 a year,
and Amgen said the drug will be available to patients within a week.
Aimovig
blocks a protein fragment, CGRP, that instigates and perpetuates
migraines. Three other companies — Lilly, Teva and Alder — have similar
medicines in the final stages of study or awaiting F.D.A. approval.
“The
drugs will have a huge impact,” said Dr. Amaal Starling, a neurologist
and migraine specialist at the Mayo Clinic in Phoenix. “This is really
an amazing time for my patient population and for general neurologists
treating patients with migraine.”
Advertisement
Millions
of people experience severe migraines so often that they are disabled
and in despair. These drugs do not prevent all migraine attacks, but can
make them less severe and can reduce their frequency by 50 percent or
more.
As a recent editorial in the journal JAMA put it, they are “progress, but not a panacea.”
Until
now, drugs used to prevent migraines were designed to treat other
diseases, like high blood pressure. They are not very effective, may
work only temporarily, and often are laden with intolerable side
effects.
In
clinical trials, people taking the new drugs reported no more side
effects than those taking a placebo. The side effects over the long term
and among people with chronic diseases remain to be determined.
“For
now, they look fantastic,” Dr. Stewart J. Tepper, a professor of
neurology at Dartmouth College, said of the new drugs. “They shake the
ground under our feet. They will change the way we treat migraine.”
Dr.
Tepper and Dr. Starling, like most leading migraine specialists, have
consulted with the drug companies and enrolled patients in their
clinical trials.
Advertisement
One
in seven people worldwide experience migraines, among them 37 million
Americans — as many as 20 percent of women, and 10 percent of men.
About
2 percent of the global population copes with chronic migraines. By
some estimates, migraine is the third most common disease in the world,
and it ranks among the top ten causes of disability.
It
is not just a headache: A migraine often is accompanied by disabling
symptoms like nausea and vomiting, difficulty speaking, and an aversion
to light and noise. The headache can be throbbing and last for hours or
days.
The new medicines are intended
for the estimated 2.8 million Americans who have a migraine many times
each month. Treating these people has been challenging, doctors say.
Patients
often try the currently available treatments one after another, in
varying combinations. Drug side effects are frequent and include mental
fogginess, sedation, weight gain, sexual dysfunction and dry mouth
leading to cavities.
Some patients
find the side effects worse than the migraines. Eighty-five percent of
migraine patients stop taking the drugs within a year.
For a draft review of migraine drugs,
the nonprofit Institute for Clinical and Economic Review surveyed
patients with frequent migraines. Many said they did not make plans or
commitments — even staying out of the work force — because they never
knew when they would get a migraine that could disable them for hours or
days.
Advertisement
They
were “frustrated, depressed, defeated, isolated,” the report said, or
felt they were a burden to society. They complained of being stigmatized
— their migraines were not taken seriously. They often tried a long
list of treatments to little or no avail.
“It’s a pretty miserable situation,” Dr. Tepper said.
Even
small children get migraines, said Dr. Andrew D. Hershey, chairman of
neurology and director of the headache center at Cincinnati Children’s
Hospital Medical Center. Recently, he saw a two-year-old with migraines.
Dr.
Hershey is involved in a clinical trial testing one of the new drugs in
12- to 17-year-olds and plans eventually to enroll children as young as
age 6.
But monoclonal antibodies
like the new drugs are grown in living cells and expensive to produce.
The high price of the Amgen drug raises questions about whether insurers
will pay and whether patients with high co-payments can afford the
medicine.
In a preliminary analysis of cost-effectiveness,
I.C.E.R. concluded that if the Amgen drug cost $8,500 a year, the price
would be reasonable for the expected improvement in quality of life for
patients with a migraine at least 15 times a month and no other
options, said Dr. David Rind, the institute’s chief medical officer.
The group will publish a final analysis in two weeks, incorporating public input and the actual price of Amgen’s drug.
The
idea behind the new drugs dates back to the 1980s, when researchers
noticed that the protein fragment CGRP seemed to play a role in
migraines. It transmits signals between nerves and also dilates blood
vessels.
Advertisement
Over
the years, researchers continued to gather evidence. “Information came
in dribs and drabs over time,” said Dr. Sean E. Harper, executive vice
president for research and development at Amgen.
Eventually, a fuller picture emerged: People who get migraines seem to make too much CGRP.
When
investigators infused CGRP into people prone to migraines, they got
headaches. When they gave the molecule to people who do not get
migraines, it usually did not instigate one.
That
was a pivotal finding, said Dr. Robert Conley, global development
leader for migraine therapeutics at Lilly. It meant that blocking some
but not all CGRP might prevent headaches without interfering with its
normal functions in the body.
Companies
began developing small molecules to block CGRP, but they turned out to
be too toxic to use as drugs. So researchers turned to antibodies, which
work differently. Aimovig, the newly approved drug, blocks the molecule
on the cell surface that CGRP must attach to in order to work.
Antibodies
persist in the body, Dr. Harper noted, which is why patients can be
treated just once a month. (Unlike the other companies, Alder is
developing a medicine to be infused intravenously in a doctor’s office
every three months.)
Dr. Laura Greer,
38, a pediatrician in Etna, N.H., has about eight migraine days a month
despite using eight treatments, including a device that transmits a
magnetic pulse to her head. Without them, her monthly migraine days
numbered 14.
She tried over 40
treatments over a period of years to find a handful that helped. And she
puts up with side effects like a dry mouth and forgetting words.
No comments:
Post a Comment