FDA Approves Radicava, First New ALS Therapy in 22 Years

FDA Approves Radicava, First New ALS Therapy in 22 Years

The first treatment for amyotrophic lateral sclerosis (ALS) in more than two decades, Radicava (edaravone), has been approved by the  U.S. Food and Drug Administration and will soon be available to patients.

The May 5 FDA decision, hailed by physicians and ALS advocates, brings hope to the ALS community that treatment development, after a long slumber, is again on the move.

“We hope today’s announcement signals the beginning of a new chapter in the fight against this terrible disease,” Barbara Newhouse, president and CEO of the ALS Association, said in a press release.

The approval is based on data from a Phase 3 clinical trial (NCT01492686) in Japan, which demonstrated Radicava’s ability to slow decline in the daily functioning of ALS patients. The trial led to the treatment’s approval in Japan and South Korea in 2015 — an act that caught the FDA’s attention, bringing the agency to contact with the drug developer’s American subsidiary,  Mitsubishi Tanabe Pharma America (referred to simply as MT Pharma).

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” Eric Bastings, MD, deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in an FDA press release.

“This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option,” Bastings said.

Similar reactions were heard from neurologists and patient advocates, cited in an MT Pharma press release.

“For people with ALS and their families, having a new therapy which slows the decline of physical ability is incredibly significant,” said Jonathan S. Katz, MD, ALS clinic director of the Forbes Norris MDA/ALS Research and Treatment Center at California Pacific Medical Center. “This is an uplifting milestone for the ALS community especially since it’s been so long since we had anything new.”

“This is an important time for people living with ALS,” Newhouse added.

According to the ALS Association, the treatment’s list price is $1,000 per infusion, or about $146,000 annually, and it is expected to be available for use by August.

Clinical trial results

Results from the six-month Japanese clinical trial — in which 137 patients were randomized to receive either Radicava or placebo — showed that Radicava reduced the decline in physical ability by 33 percent compared to placebo, the company said.

Researchers measured physical function using the ALS Functional Rating Scale-Revised (ALSFRS-R). The difference in decline seen between the groups equaled 2.49 ALSFRS-R points.

“We believe Radicava offers new hope for people with ALS and exemplifies MT Pharma America’s commitment to innovative therapies for patients in the United States battling life-threatening diseases,” said Atsushi Fujimoto, president of MT Pharma America.

“The approval of Radicava brings us into a new era of treatment by evolving how we manage this complex disease,” added Katz.

How Radicava works

Radicava helps to control excessive oxidative stress in the body. Research shows that in ALS, damaging oxidative processes occur at a higher than normal rate.

The drug is given by intravenous infusion, in a relatively intense schedule of 28-day cycles. Treatment starts with daily infusions for 14 days, followed by 14 days of rest. During later treatment cycles, patients receive Radicava for 10 of 14 days, again followed by a 14-day treatment-free period. Each infusion lasts for about 60 minutes, during which patients receive 60 mg of the drug.

The most common side effects — seen in more than 10 percent of patients — were bruising, gait problems, and headache.

But both the FDA and MT Pharma underscore that although Radicava was seen to help many patients, its use can also cause serious — even life-threatening — side effects. Particularly, severe allergic reactions, known as an anaphylactic shock, can occur both during and after an infusion.

Radicava contains sodium bisulfite, and in addition to general allergic reactions to Radicava, some patients are allergic to the compound. Sulfite allergy can make itself known as both mild reactions and a life-threatening anaphylactic shock. People with asthma are particularly prone to a sulfite allergy.

If patients experience hives; swelling of the lips, tongue, or face; fainting; breathing problems; wheezing; trouble swallowing; dizziness; itching; or an asthma attack (in asthmatics), they should immediately contact their doctor or seek emergency care.

Since these reactions occurred in people once the drug was approved and were reported spontaneously, MT Pharma cannot provide numbers of how common such reactions are.

Patient support programs

MT Pharma has launched a support program for patients prescribed Radicava to help them with its $1,000 per infusion list price. The Searchlight Support assigns a personal case manager to each patient, who assists in ensuring coverage by health insurance providers.

Case managers also help with other aspects of access, including transportation to infusion clinics, navigating co-pay support programs, and patient assistance programs for the uninsured. Support can be reached through the company’s website or by phone at 1-844-SRCHLGT (722-4548)

“We recognize how important this therapy may be to people with ALS and are committed to helping provide access to this important treatment option, with the goal of keeping out-of-pocket costs at a minimum for eligible patients,” said Atsushi Fujimoto, president of MT Pharma.

Radicava’s way to the U.S.

MT Pharma underscored that Radicava has gone through several Phase 3 trials, with a total time in clinical development of 13 years. No studies were performed at U.S. institutions.

However, the company presented trial data at scientific conferences in North America, including the American Academy of Neurology (AAN) Annual Meeting in Canada in 2016, and further updates at the AAN 2017 Annual Meeting in Boston in April.

The drug was originally developed by Japanese Mitsubishi Tanabe Pharma Corporation, and the clinical program there led to its approval in Japan and South Korea in 2015. In that same year, Radicava was granted orphan drug designation by the FDA, following the decision of its EU counterpart, the European Medicines Agency.

The company filed a New Drug Application (NDA) in June with the FDA, which accepted it for review on Aug. 30. Radicava’s approval on May 5 came more than a month before the FDA’s announced action date, which was set for June 16, 2017.

“We applaud the work MT Pharma America and the FDA are doing as they have taken unprecedented steps to get this treatment into the hands of patients as quickly as possible,” said Newhouse, who is familiar with the Japanese company.

MT Pharma became the ALS Association’s national corporate sponsor of the Walk to Defeat ALS, a nationwide campaign aiming to support care service programs, research, and advocacy efforts, in 2016.

To Newhouse, FDA approval signifies much more than a long-overdue new therapy option — it brings renewed hope that treatment development is again on the move.

“The approval of Radicava gives great promise for what we hope will be the first of many new treatments,”  she said in the MT Pharma release. “There are several drugs to treat ALS currently in clinical trials and we are hopeful that people living with ALS have even more therapies available to them sooner rather than later.”

The only other treatment specifically indicated for ALS, the oral tablet riluzole (Rilutek), was approved by the FDA in 1995.

32 comments

  1. Sang Riel says:

    Since these reactions occurred in people once the drug was approved….RED FLAG. No one in trials reacted ?

  2. Minha esposa, diagnosticada em Fevereiro de 2014, não anda, não fala, traqueostomizada e se alimenta via sonda.
    Que efeito o Radicut poderá fazer sobre ela?

    My wife, diagnosed in February 2014, can’t walk, can’t talk, traqueostomizada and feeds via probe.
    What effect the Radicut can do about it?

  3. US Consumer says:

    $146,000 a year, 4 times the price it was launched at in Japan. It’s great there is a new treatment, it’s a shame we have to financially bankrupt anyone that needs it.

    • Echo Jordan says:

      This is my thought as well. Why are drugs marked up sometimes several thousand percent in the US. They come up with something that could possibly help but put it out of reach for most people. There is no way we could ever afford it. This is so sad.

  4. Tina says:

    The cost should be the same as in Japan!!! What if our insurance won’t cover it!!!! We want hope to see our children and grandchildren grow up, not see us die!

      • Magdalena Kegel says:

        Hi Michael,
        Some people have a familial type of ALS, but sporadic ALS—in which people have no family history of ALS—accounts for 90 percent of more of all ALS cases. For reasons not entirely clear, veterans have a higher risk of developing ALS.

  5. Dr Vincent Acorlor says:

    I’m a surgeon in Ghana and have patient diagnosed with ALS Dec. 2016, we are willing to stard her on Radicava as soon as possible. Can someone indicate the duration of the treatment ?

  6. pAL in need of real solution says:

    The price is high because to start the med is coming from Japan as the med is not being made in the US. MT Pharma does not have factories in the US as of now creating this med. I guess they were waiting for FDA approval before making the investment. So we pay the price:( When will “good” news really be “good”news?! I also cannot find eligibility requirements to receive the med. For example, do u have to have ALS less than 2 years? Are some folks considered too far gone? Or will it be eligible for anyone who can afford it? If u have found these requirements please post them on this website so I can look them up. Many thanks.

  7. Robert Cebaus says:

    Sounds great however the cost is something most can not afford. Who would need this more some one just starting a life with ALS or someone well into ALS Not sure I know.

  8. Shi says:

    F1000Res. 2017 Apr 3;6:410. doi: 10.12688/f1000research.10534.1. eCollection 2017.
    Treatment with penicillin G and hydrocortisone reduces ALS-associated symptoms: a case series of three patients.
    Tuk B1, Jousma H1, Gaillard PJ2.
    Abstract
    Three male Caucasian patients with ALS were admitted to the hospital due to progressive dysphagia and dysarthria. During two 21-day courses of penicillin G and hydrocortisone, these patients’ dysphagia and dysarthria resolved. The patient’s other ALS-associated symptoms also improved, including respiratory function, coordination, walking, and muscle strength. This is the first report of a treatment with a protocol for treating dysphagia, dysarthria, respiratory depression and other ALS-related symptoms. Furthermore, the observations are consistent with the recent hypothesis that the successful treatment of ALS symptoms with this treatment course in six patients with syphilitic ALS was not directly due to the treatment of syphilis; but that the administered penicillin G and/or hydrocortisone treated these patients’ ALS symptoms due the off-target pharmacological activity of penicillin G and/or hydrocortisone. This report therefore underscores the need to evaluate the efficacy of this treatment course in a clinical trial.

    https://f1000research.com/articles/6-410/v1

    • August Haller says:

      Immediately, after I read that report, I sent an e-mail to the author Dr. Bert Tuk. I asked for the Hospital where the patients where cured (in Holland) and if access to the therapy for foreigners would be possible. I got the answer that he is not able to answer the many requests personally. Since then I am waiting and I watch the author’s home page http://rypharma.com/ and different forums to find other people interested in that type of cure. Up to now, I could not find more reactions. I started a request to Swiss a German medical doctors and I am waiting for there reaction. (I am a relative of an ALS-Patient).
      I do not understand why ALS News Today ist not overflowed with requests to that promising cure.

      • Shi says:

        If you are in the early stage,as noted in the article, you can recommend this to your doctor and ask him/her to try this method.It’s cheap and effective. Good luck.

      • Ruth McVicar says:

        Hi I would love to know how your progress with researching this treatment, etc is going??My eldest son was diagnosed two years ago, now presenting with all the symptoms associated with ALS. I’d love to hear what you’ve discovered please?

        • Magdalena Kegel says:

          Hi Ruth,
          I’m really sorry to hear about your son. We at ALS News Today are, however, not involved in research. We report on publicly available news and research advances. We will publish information on any potential progress as it becomes available to us. If you wish to stay on top of what we report on, please sign up for our newsletter.

        • August Haller says:

          Hi Ruth McVicar

          Sorry for the late answer. My latest information is from the 06/07/2017. Quote: “We currently are preparing an update of the F1000 paper that includes the current status of the patients. Briefly, P1 and P3 have been improved / stable for 4-5 months, where P2 still is at an improved level, as of today already for 7.5 months.” And further: “To our knowledge at SEVBI, next to the 3 patients published, only 3 additional patients recently started with the medication. This number is low because Dutch neurologist is very conservative when it comes to off-label prescribing. SEVBI tries to follow all patients taking the medication and will publish all data that is gathered, so patients and prescribers will have access to all information available.”
          My sister’s son at this time is not willing to get this treatment; even it seems to me the most promising.

  9. don ng says:

    US146000 is a crazy price for this kind of drugs sorry sir we cant afford so we have to wait for FDA To approve Those pills form FDA pls help us Tirasemtiv and Masitinib or NP001 or AT1501 tks FDA pls help the cost is killing us

  10. Nguyễn Văn Bích says:

    Patients (ALS) name: Nguyễn Thị Hồng, from Vietnam
    My wife (name: Nguyễn Thị Hồng, Hanoi, Vietnam) is 50 years old and has been diagnosed with ALS (Charcot) since February 2013. She has been completely paralyzed now, unable to turn herself back in sleep. At present, the ability to eat and swallow food is also very poor. Please tell me that: can she take Radicava injection or not? Dosage and duration of treatment?
    Thanks!

    • Magdalena Kegel says:

      Hi Nguyễn,
      I am sorry to hear about your wife.
      We at ALS News Today are not physicians and are not able to answer specific medical questions or give advice. Please contact her physician with these questions.

    • Magdalena Kegel says:

      Hi Craig,
      We at ALS News Today are not Medical Doctors, and so, we are not able to advise you on specific medical issues. The prescription label, however, simply says: “RADICAVA is indicated for the treatment of amyotrophic lateral sclerosis (ALS),” and does not hold further information about disease stages.

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