Radicava and Its Swift FDA Approval Are Reasons for Hope, ALS Association Official Says

Radicava and Its Swift FDA Approval Are Reasons for Hope, ALS Association Official Says

Radicava (edaravone), the first approved treatment for amyotrophic lateral sclerosis in 22 years, is being welcomed with “a great deal of excitement” in the ALS community, an ALS Association executive said.

The excitement stems both from Radicava’s potential to treat the disease and the hope that its approval will lead to future treatment authorizations, said Calaneet Balas, the association’s executive vice president of strategy. She spoke with ALS News Today in a telephone interview. (A transcript of the interview can be found here.)

In addition, the community is pleased by a U.S. Food and Drug Administration decision to approve Radicava without U.S. clinical trials, on the basis of trial results in Japan.

Not surprisingly, there are “a lot of questions and maybe a little bit of anxiety,” surrounding Radicava’s introduction next month, but also “a great amount of excitement. I think, if anything, this approval has brought true hope to this community.”

The anticipation is largely a reflection of Radicava’s potential to benefit people with few treatment options.

But an important part of the Radicava story is the FDA doing “something they don’t do,” Balas said, in deciding “to move this drug forward [quickly] because of such a grave, unmet need in a really terrible disease.”

New treatment, new hopes

Radicava’s journey started in Japan, which approved it to treat ALS under the brand name Radicut in 2015 before the FDA action opened the door to it in the United States.

Initially developed by Mitsubishi Tanabe Pharma Corporation as a stroke treatment, edaravone demonstrated in three Phase 3 clinical studies in Japan that it could preserve function in some ALS patients and slow the rate of the disease’s progression.

The results came in a pivotal Phase 3 trial, MCI186-19. Edaravone led to a 33 percent reduction in the rate of patients’ decline in physical function in that study. In an extension of the trial, it significantly slowed disease progression.

Radicava “doesn’t necessarily stop the disease or increase overall survival, but I think it might be very useful for some people in order to preserve function,” Balas said. “And function like being able to speak, or swallow, or move — breathe — are very important aspects of anybody’s life.”

The therapy belongs to a class of free-radical scavenger drugs known for protecting cells and tissue from the damaging effects of free oxygen reactive elements. Repeated studies have shown those elements to be an important feature of ALS.

The trial results in Japan prompted MT Pharma America to apply for FDA approval. In a surprise to many, the agency accepted the application in August 2016 without requesting that the results be confirmed in a U.S. trial. On May 5, it announced Radicava’s approval.

Importantly, the FDA designated it as a treatment for all ALS patients, not just those in earlier phases of the disease. That decision honored a request from the ALS community.

“This is only the second [approved] therapy and neither are curative by any means,” Balas said. “The FDA showed that it is anxious to move forward with therapies that may have impact for an unbelievably unmet need.”

Prior to May, the only FDA-approved ALS treatment was the oral tablet riluzole (Rilutek), which arrived on the market in 1995.

FDA action is the real story

Radicava’s entire clinical trial development occurred in Japan. Even the FDA, in its announcement, recognized how extraordinary it was for it to approve a therapy without U.S. trials.

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer,” Dr. Eric Bastings, a deputy director in the FDA’s Center for Drug Evaluation and Research, said in the May 5 announcement. “We are pleased that people with ALS will now have an additional option.”

Balas agrees. Approval brings a necessary and immediate treatment option to ALS patients in the United States, she emphasized. And it may open the door to the approval of other therapies in testing stages.

“I think it shows their [the FDA’s] willingness to start thinking in different directions, and really trying to expedite drugs when they think they have something that might have some efficacy and value to the patients,” she said. “To me, that’s one of the really big highlights here.”

After more than two decades of drought, a number of potential therapies are in clinical trials that could “ring out in the very near future,” Balas said. “My hope is that this is the first of many treatments.”

Of particular interest to the ALS Association is an oral therapy called tirasemtiv, developed by Cytokinetics. The company is concluding a Phase 3 clinical trial (NCT02496767) assessing tirasemtiv’s ability to improve respiratory function in more than 700 ALS patients.

The association is also closely watching work on various cocktail, or combination, therapies that could be used to reduce ALS symptoms or slow the disease’s progression.

By giving Radicava a quick and relatively painless approval, the FDA stepped onto a path that “I think is going to drive some more research and development,” Balas said. “I certainly hope so.”

Insurance policies, price, and a look ahead

When Radicava arrives in August, it will come with a list price of nearly $1,100 per infusion, meaning a year’s worth of treatment will be roughly $146,000. Many patients and their families will likely be unable to pay that price with their own resources.

But Balas said the association has yet to become overly concerned about the price. It is neither out of line with other new therapies nor likely to be borne by an individual, she said.

As an approved drug, Radicava is eligible for private insurance coverage. In addition, government aid exists, and MT Pharma is working with insurance carriers and pharmacies to come up with a final price. The company is also running a patient assistance program, Searchlight Support, that includes medical helplines, infusion center locations, and ways of addressing cost.

“While on the face value the drug price is high, or it might be high, I’m not sure that’s really the whole story,” Balas said. “List prices here in the United States are very different from the negotiated prices.”

Ensuring access to Radicava is the more important issue, she said. “If the drug is not accessible because of pricing, then that’s another conversation,” she added.

Access includes getting to centers that offer the infusion treatment, which is administered in intensive 28-day cycles, or getting to the specialty pharmacies where Radicava may be distributed. Transportation can be a problem for people with a debilitating disease.

What insurance companies will and will not cover is still a question.

So far, only United Healthcare has provided detailed information on its Radicava coverage policy plan. While that plan may be adjusted, as it stands it limits coverage to earlier-stage ALS patients with good respiratory function — the clinical trial population. The FDA approval covers all patients.

That coverage policy “sets very bad precedent not only for this drug, but for any future therapy that might come out,” Balas said. “Trials are designed for a variety of reasons, and for United Healthcare to make a decision based only on the clinical trial design, as opposed to what the FDA has asked — in a population that has pretty much no therapies — is gravely concerning to me.”

The coverage is also worrisome, she said, because of its potential impact on investments in ALS research and drug development. Another concern is a possible requirement that patients be reassessed every six treatment cycles as to their eligibility for continued coverage.

“If someone’s FVC — forced vital capacity — drops below that 80 percent, then they lose the ability for coverage? ” Balas asked. “I don’t know what their plan is there, but I think it puts people at risk.”

Radicava’s use — like any medication, especially for a chronic and progressive disease — is a decision best left between a patient and a doctor, she said.

United Healthcare was asked in emails over the course of a week to explain its policy’s stipulations and restrictions. But as of ALS News Today‘s publication time on June 30, the company had yet to provide a response, attributing that inability to key personnel being in meetings and on vacation.

The association is not aware of restrictions that other carriers might be planning, but many have yet to make their coverage details public. Conversations about coverage with private carriers and government agencies continue, Balas said.

Until more is known, the association is moving ahead with what needs to be done to prepare the community for Radicava’s arrival, especially as “this is a space that hasn’t had new therapy for a long time, so there will be a lot of questions, a long learning curve,” Balas said.

Among its plans are webinars for healthcare professionals, clinical care conferences, and question-and-answer sessions and discussions open to all in the ALS community.

“My hope is this is the first of many treatments, and then we’re talking to our constituents about a variety of educational materials on a variety of treatments,” Balas said.

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    • Karl says:

      If diagnosed with ALS and the doctor won’t write a prescription for Radicava go to an ASL doctor who will.

      • Charlie says:

        A very specific sub-group of ALS patients will benefit from Radicava, giving an extra 12 months, on average. Those who fall outside of this group have been proven unaided by it. That may be the reason this doctor is ‘not open’ to it.
        However, should the patient fall in to the sub-group that would not benefit from Radicava then the doctor who prescribes it is giving false hope and acting unnecessarily, and even unethically. Some doctors will prescribe it and the only persons to benefit will be the shareholders of MT Pharma.

  1. Charlie says:

    “Importantly, the FDA designated it as a treatment for all ALS patients, not just those in earlier phases of the disease. That decision honored a request from the ALS community.”

    The FDA is controlled by Big Pharma and so ‘designating’ as wide a market as possible is high on their agenda for bottom-line profits.
    Of course the ALS community asked for it. The ALS community utterly is desperate for absolutely anything that gives hope. However, ALS specialist neurologists are still saying that Radicava is only going to be useful for an ALS patient in the very early stages of the progression.

    You have to carefully follow the dollar when it comes to Big Pharma. Big Pharma is seeking a profitable treatment. Researchers are seeking a cure.

  2. Charlie says:

    ‘…cryo-electron microscopy to see how the Hsp104 yeast enzyme breaks up clumps of the misfolded proteins that are hallmarks of neurodegenerative diseases like ALS.”
    This is where research should focus effort, because it shows enormous promise. A yeast enzyme is likely to be a fraction of Radicava’s price and far more effective if the promise is fulfilled.

    Radicava will be very useful to those newly-diagnosed next month.
    Existing patients of a year’s duration with failing functionality and lung weakness won’t medically qualify for Radicava Rxs, and the research shows that.

    I suggest that researchers focus on the yeast enzyme thing. They haven’t sought my opinion on this,yet……..

  3. Charlie says:

    “In amyotrophic lateral sclerosis, Alzheimer’s, and other neurodegenerative disorders, the main disease-triggering event is the formation of misfolded proteins.”

    That’s a pretty definitive statement, isn’t it?
    let’s urge them to develop this line of research as quickly as possible.

  4. Charlie says:

    My personal opinion is this :MT Pharma most likely see the ‘unfolding proteins’ work as a real threat to Radicava’s sales if research gets a speed going forward. It will therefore, as a business, and not surprisingly, seek to cover its costs on development costs for Radicava.

    There are far too many scatter-gun research projects. The research community should either fess up to not having a good idea how this disease is caused or have some courage to focus on the most promising areas and tell us why.

    • Charlie says:

      Bill, I do a lot of research a lot on this and I also have experience of business tactics and objectives (not in the pharma sector though).

  5. Iraj Farhad says:

    costing 146,000 USD is better than not approving the Radicava, because 80% of the patients will be able to pay for it. As a human being please reconsider the price.

  6. Adnan says:

    We are fighting to get this medication but the doctor will not write a script for it. The search light support requires to get the enrollment forms signed from the neurologist. The patient have Kaiser Permanente insurance. The medication is available is US and the patient cannot get it. If someone’s FVC — forced vital capacity — drops below that 80 percent, then they lose the ability for coverage? Please advise

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