Excitement to Cautious Optimism — Reactions to Approval of Ocrevus

The historic approval of Ocrevus (ocrelizumab), the first-ever treatment for primary progressive multiple sclerosis (MS), set off ripples in the relatively calm waters of MS news reporting. The drug, which was also approved Tuesday as an unusually effective and safe treatment for relapsing MS, is viewed as a game-changer by many experts in the field.

“The FDA’s approval of Ocrevus is the beginning of a new era for the MS community and represents a significant scientific advance with this first-in-class B-cell targeted therapy,” Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech, said in a press release.

“We believe Ocrevus, given every six months, has the potential to change the disease course for people with MS, and we are committed to helping those who can benefit gain access to our medicine,” she added.

Genentech’s approval statement did not hold any surprises, with Ocrevus being approved for both relapsing and primary progressive forms of MS, as studied in three Phase 3 clinical trials.

“Multiple sclerosis can have a profound impact on a person’s life,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the FDA’s official release. “This therapy not only provides another treatment option for those with relapsing MS, but for the first time provides an approved therapy for those with primary progressive MS.”

The drug, with an annual list price of $65,000 a year (about 25 percent less than Rebif, the active control in the RMS trials), will be available to patients in the U.S. within two weeks.

Although the safety of Ocrevus was found to be similar to the comparator Rebif (interferon beta-1a), with mainly mild or moderate airway infections and infusion-related reactions, the drug does hold a risk for more serious complications, including cancer.

In its statement, Genentech emphasized that patients need only stick to routine screening — based on age and medical history — for cancer and other side effects.

“This is an exciting day for everyone touched by MS, a disease that strikes in the prime of a person’s life when she or he may be starting a career or family,” said June Halper, CEO at the Consortium for MS Centers.

“We have eagerly awaited the FDA approval of Ocrevus because it not only offers a new, highly efficacious treatment option for people with relapsing multiple sclerosis, but it is also the first disease-modifying therapy indicated for primary progressive multiple sclerosis, a highly disabling type of this chronic disease,” Halper said. “For many people living with MS, this FDA approval is a source of hope.”

As the word about Ocrevus’ approval spreads throughout the world, Multiple Sclerosis News Today watched the reactions of patients, patient organizations, physicians, and researchers to see how the news was received.

MS Society: Ambitious hopes for future

The National MS Society is an untiring force in the work to form a better present and a better future for MS patients. As the news broke late last night, its news statement of the approval simply held a single quote: “This is a real game changer.”

It was Cyndi Zagieboylo, the MS Society’s president and CEO, who put words to the thoughts of many.

Our hope is that “this is just the beginning of the development of the next generation of treatments for MS,” Zagieboylo told Multiple Sclerosis News Today. And, she noted, her nonprofit group is “the largest private funder of MS research in the world.”

But although the MS Society is hoping for more, and it’s chief advocacy, services and research officer Tim Coetzee called the drug’s effects in primary progressive patients “modest” in an earlier interview with this news website, he conceded that “it is a huge step forward compared to no available treatment at all.”

The same opinion was voiced by the National MS Society’s executive vice president of research, Bruce Bebo, in an article in STAT — a publication that focuses on health and medical research.

Physicians’ voices

STAT’s main subject of interest was, Dr. Stephen Hauser — the researchers who is likely the reason the world is discussing the approval of Ocrevus today.

It was Hauser’s research team that discovered that B-cells are likely involved in MS, persuading Genentech to initiate a clinical trial of a drug no one believed would work.

“It’s personally incredibly rewarding,” Hauser told STAT’s Ron Winslow. “This is a big deal for people with MS.”

Hauser sounds more optimistic in his assessment of the drug than representatives of the National MS Society. According to STAT, he believes that people who get an MS diagnosis today will be able to “look forward to a full life without significant disability.”

One of Ocrevus’ strengths is that, in addition to effectively preventing relapses and slowing disease progression, it has not shown any signs of the serious safety concerns such as other effective treatments like Tysabri (natalizumab). This drug can, in rare cases, cause PML (progressive multifocal leukoencephalopathy) — a virus-triggered condition that can devastate the brain much quicker than MS itself.

While no cases of PML have been observed with Ocrevus treatment, it is no guarantee they will not appear as more patients get treated.

“Anytime a new drug comes out you want to get some experience with it. But over time [Ocrevus] could be accepted and used in a lot of patients,” Dr. Harold L. Weiner, director of the Partners MS Center at Brigham and Women’s Hospital in Boston, told STAT. Weiner was not involved in the clinical trials of the drug.

Dr. Steven L. Galetta, chairman of the department of neurology at NYU Langone Medical Center and an MS expert, aired a similar opinion in the New York Times’ coverage of the news, in which Genentech’s Dr. Peter Chin also stated that the drug’s safety profile makes it a candidate for early use.

“I think if the safety holds up, it will become the leading MS therapy,” said Galetta, who, like Weiner, was not involved in the Ocrevus trials. But Galetta pointed out that the medical community will be keeping an eye on Ocrevus to see how it performs once it becomes available.

What about patients?

Because the drug was only approved Tuesday, the vast majority of MS patients have no experience with the treatment.

But the New York Times spoke to one who does. In its coverage of Ocrevus’ approval, they spoke to Jerrie Gullick, a 51-year-old primary progressive MS patient who participated in one of the clinical trials.

In the past seven years since her diagnosis, Gullick’s condition had deteriorated rapidly. Like many other MS patients, she saw her life shrink as she got worse. She had to quit her job, could walk only short distances, and started losing her ability to think.

Gullick told the New York Times’ Katie Thomas that when she noticed that her disease slowed down, she had hoped that she was receiving the placebo. “From a patient perspective, what you want is to get back what you’ve lost,” Gullick said. “What this drug seems to do is to stop what was happening.”

But she grew to realize that she was, in fact, thankful for the time that the treatment has given her. “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,” she told the Times reporter.

But some seem to have been more lucky than just seeing their deterioration slow down. Christina Reyes — another patient who had been ill since 1990 when she was diagnosed at the age of 15 — told “NBC News” a story of improvement.

Even in her younger days, she needed walking aids, and at times, even a wheelchair. “There were times when I could not bathe myself. I had to ask my mother to wash my hair. I couldn’t write. I felt drained all the time,” she told NBC.

But after the Ocrevus trials, Reyes, now 42, has no need for leg braces or wheelchairs. Instead, she asks her husband if he wants to go dancing. “This is exactly what I wished and hoped and prayed for. It’s my miracle drug,” she said.

The price — expensive, yet cheaper than some

As tends to be the case with newly approved drugs, there is the issue of the price tag. Genentech has set the annual price tag at $65,000. This, the company pointed out, is 25 percent lower than the less effective comparator Rebif, which has a yearly cost of $86,000.

According to the STAT, a recent study found no MS drug priced under $50,000 when examining nine of the common drugs on the market. The National MS Society has also noted that MS drugs cost nearly four times more than 12 years ago.

“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,” Genentech said, according to the New York Times.

With Ocrevus being made available in two weeks, the number of patients and physicians with first-hand experience of the drug will grow, allowing more voices to be heard. And while the trials have provided a scientific basis for the drug’s evaluation, its assessment in real life is only now beginning.

Of note, Genentech will be offering services to help minimize barriers to access and reimbursement for patients prescribed Ocrevus. For more information, call 1-844-OCREVUS (844-627-3887). Genentech also plans to offer patient assistance programs through Genentech Access Solutions to eligible patients; more information is available at 866-4ACCESS (866-422-2377).