The Food and Drug Administration approved on Tuesday the first drug to treat a severe form of multiple sclerosis, offering hope to patients who previously had no other options to combat a relentless disease that leads to paralysis and cognitive decline.
The federal agency also cleared the drug to treat people with the more common, relapsing form of the disease.
“I
think that this is a very big deal,” said Dr. Stephen Hauser, the
chairman of the neurology department at the University of California,
San Francisco, and leader of the steering committee that oversaw the
late-stage clinical trials of the drug, ocrelizumab. “The magnitude of
the benefits that we’ve seen with ocrelizumab in all forms of M.S. are
really quite stunning.”
The drug, which will be sold under the brand name Ocrevus by Genentech,
showed the most notable results in patients with relapsing multiple
sclerosis, appearing to halt progression of the disease with few serious
side effects. In patients with the more severe form, primary
progressive multiple sclerosis, the drug only modestly slowed patients’
decline, but medical experts described it as an important first step.
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“This
sort of opens the door for us,” said Dr. Fred D. Lublin, who was a
crucial investigator for the clinical trial and is director of the
Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai
Hospital in New York. “Once we open that door, then we do better and
better and better. It’s a very encouraging result.”
Genentech,
which is owned by the Swiss pharmaceutical giant Roche, said Tuesday
that it would charge a list price of $65,000 a year, which — though
expensive — is 25 percent less than an existing drug, Rebif, that was
shown to be clinically inferior to Ocrevus in the two clinical trials
that led to Ocrevus’s approval.
Rebif,
sold in the United States by the German company EMD Serono, carries a
list price of about $86,000. In a statement, Genentech noted that the
price of drugs to treat multiple sclerosis had risen sharply in recent
years.
“We
feel that the industry needs to start to reverse this trend, and
believe that pricing Ocrevus 25 percent less than the comparator in our
trials is an important first step,” the company said.
Although
people with the relapsing form of the disease have more than a dozen
treatment options, many of the most effective drugs also come with
significant side effects, which means that doctors often wait to
prescribe them until a patient’s disease has advanced significantly.
Ocrevus is viewed as relatively safe: Side effects included reactions at
the injection site (the drug is infused every six months), and more
upper respiratory infections and cold sores.
As
a result, “it represents a new treatment option that has the potential
to be used earlier,” said Dr. Peter Chin, the group medical director of
neuroscience at Genentech, who was closely involved in developing the
drug.
An estimated 400,000 people have multiple sclerosis in the United States, and about 15 percent have the primary progressive form of the disease.
In the trials that studied
the relapsing form of the disease, which involved 1,656 patients, those
taking Ocrevus saw a 47 percent reduction in their rate of relapses
compared with patients who were taking an existing treatment, Rebif. In
the clinical trial for people with primary progressive multiple
sclerosis, which involved 732 patients, those on the drug had 24 percent less risk of their disability progressing compared with patients who were taking a placebo.
Ocrevus
works by depleting a specific type of a patient’s B cells, which
circulate in the blood and are part of the immune system. While they
normally help the body fight off infections, they are believed to
malfunction and contribute to central nervous system damage in people
with multiple sclerosis.
“I
think if the safety holds up, it will become the leading M.S. therapy,”
said Dr. Steven L. Galetta, the chairman of the department of neurology
at NYU Langone Medical Center, who is an expert in multiple sclerosis
and who was not involved in the clinical trials. But, Dr. Galetta said,
the medical community will be watching to see how the drug performs once
it is widely available. The clinical trial showed a slightly heightened
rate of tumors in patients with primary progressive multiple sclerosis,
which he said needed to be monitored closely. “There can be side
effects, but you just didn’t have enough patients initially to confirm
that signal,” he said.
Jerrie
Gullick, one of the patients who received Ocrevus in the clinical
trial, said the drug had significantly slowed the progression of her
primary progressive multiple sclerosis since she began taking it about
three and a half years ago.
Ms. Gullick,
who is 51, had been declining steadily since she learned she had the
disease in 2010. At the time she learned she had multiple sclerosis, Ms.
Gullick was an active 45-year-old who walked six miles a day between
her home in Park Slope and her office in Downtown Brooklyn, where she
worked as the chief financial officer of a technology start-up.
But
her condition quickly deteriorated, and the drugs she was given
appeared to do little to help her. She lost the ability to walk more
than two to three blocks at a time, and she had to move out of her
third-floor walk-up to an elevator building. Fatigue led her to leave
her job, and she began to notice cognitive decline.
Ms.
Gullick said that at first she hoped she was on the placebo because
while she noticed her disease slowing, her condition did not improve.
“From a patient perspective, what you want is to get back what you’ve
lost,” Ms. Gullick said. “What this drug seems to do is to stop what was
happening.”
Now,
she said, she is thankful that Ocrevus appears to have bought her more
time. “I was figuring another five years, and I was going to be
bed-bound, and one day I realized I might have 20 or 30 more years of
this,” she said. “It’s like I was on a bullet train, and I was
transferred to a local.”
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