The U.S. Food and Drug Administration (FDA) on May 5, 2017, approved edaravone (brand name Radicava) for the treatment of ALS (amyotrophic lateral sclerosis). Radicava is the first ALS treatment to be approved in the United States in more than 20 years, and is only the second drug ever approved to treat this devastating disease.

Although MDA was not directly involved in the development of Radicava, we are excited for it to become an available treatment option for our ALS families, and with 44 active ALS research projects under way — with a total funding commitment of $11.4 million — we are continuing our work to free people from the harmful effects of ALS.

In connection with the FDA’s decision to grant approval to edaravone (brand name Radicava), under development by MT Pharma America to treat ALS (amyotrophic lateral sclerosis), MDA has prepared the following Q&A, which includes material provided by MT Pharma America, for its spokespeople and leaders.

What is Radicava?

Radicava, developed by MT Pharma America (MTPA), is a prescription medicine approved by the FDA to treat people with ALS.

In clinical trials, people given Radicava showed significantly less decline in physical function compared to those who were given a placebo, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in people with ALS.

What will Radicava do for people with ALS? How meaningful a treatment is it?

Although Radicava is not a cure, it has been designed to help people live with the disease. While people with ALS experience varying rates of progression, the loss of physical abilities can be fast. Slowing the decline of that loss of function in ALS is incredibly important. Depending on the level of function when someone begins treatment, the impact Radicava demonstrated in clinical trials could translate into potentially helping people preserve function longer, assisting their ability to get out of bed or feed themselves.

What is the recommended dose of Radicava?

According to the prescribing information, Radicava is administered by intravenous infusion. It takes 60 minutes to receive each 60 mg dose.

For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period.

Your insurance coverage may determine whether it is administered at an outpatient center, in your home or someplace else. MTPA will ship Radicava to ALS Centers, and to specialty pharmacies that can deliver it to patients who will receive treatments in their homes.

What data formed the basis for the FDA approval?

Study MCI186-19 was the pivotal phase 3 study that evaluated the efficacy and safety of Radicava compared with placebo in 137 people with ALS. In the six-month study, after a 12-week pre-observation period, eligible patients were randomized into groups. Those in one group were treated with 60 mg of Radicava, administered intravenously for 60 minutes; those in the other group received a placebo. The primary endpoint for the study was change in the ALSFRS-R score from baseline to six months.

What were the safety results?

 The most common adverse reactions that occurred in greater than 10 percent of patients and greater than placebo were bruising (contusion), problems walking (gait disturbance) and headache.

Hypersensitivity (allergic) reactions:

• Hypersensitivity reactions have happened in people receiving Radicava and can happen after an infusion is finished. Tell your healthcare provider right away or go to the emergency room if you have any of the following symptoms: hives, breathing problems, itching, dizziness, wheezing, fainting or swelling of the lips, tongue or face.
• Your health care provider will monitor you during treatment to watch for signs and symptoms of all the serious side effects.

How does Radicava work?

Radicava is thought to work by relieving the effects of oxidative stress, which has been suspected to play a role in the death of nerve cells called motor neurons in people with ALS. (Oxidative stress is an imbalance between the production of free radicals and the ability of the body to counteract or detoxify their harmful effects with antioxidants.) Targeting this pathway could potentially preserve motor neuron health, which could in turn, keep muscles functional for a longer period of time.

Will Radicava cure ALS?

Treatment with Radicava will not cure ALS. However, while people with ALS experience varying rates of progression, the loss of physical abilities can be fast. Slowing the decline of that loss of function in ALS is incredibly important. Depending on the level of function when someone begins treatment, the impact Radicava demonstrated in clinical trials could translate into potentially helping people preserve function longer, assisting their ability to get out of bed or feed themselves.

Can Radicava be taken by those on riluzole?

As always, patients should speak to their healthcare provider regarding their medical care. The labeling for Radicava contains no limitation of use regarding the concurrent use of Radicava and riluzole in people living with ALS.

There’s an ongoing debate about whether these incremental drugs are really helping to advance medicine. Do you think more resources should be pushed towards novel therapies?

With every incremental success, researchers, biotechs, pharmaceutical companies and other industry stakeholders gain more confidence and rationale to support the development of more ambitious technologies and approaches to treatments for ALS and other diseases. For some diseases, including ALS, comprehensive treatment may involve multiple drugs that target different disease mechanisms.

What will Radicava cost?

MT Pharma America has indicated that Radicava will cost $1,086 per infusion. Following the dosing and administration as per the label, annual cost for 12 months or 13 cycles will cost $145,524.

Your affordability questions can be answered by contacting a specialist at Searchlight Support, 844-SRCHLGT (844-772-4548). Patients prescribed Radicava should check eligibility requirements for each aspect of MTPA’s patient assistance program as restrictions apply.

What do I do if I don’t have insurance?

MTPA has created a program that provides Radicava at no charge if you do not have insurance and meet income and certain other requirements. The program is available to eligible patients without insurance.

What happens while I’m waiting for Searchlight Support to confirm my insurance?

MTPA has a program in which eligible patients may receive Radicava at no charge for up to two months while insurance is making a coverage determination.

How do I get Radicava?

MT Pharma America anticipates Radicava will be available in the United States by August 2017. However, there are several steps you can take now to be able to access Radicava when it becomes available:

• Like other FDA-approved medications, Radicava requires a prescription from your health care professional. Talk to him/her to determine if Radicava is right for you. If so, they will provide you a prescription.
• Go to www.RADICAVA.com to provide your email address. This will enable you to receive updates on availability.
• As soon as you receive a prescription, contact Searchlight Support by calling 844-SRCHLGT (844-772-4548). Searchlight Support can assist you with personal case management, reimbursement support and, once you start treatment, 24/7 product support. Some people with ALS who have received prescriptions for Radicava may be eligible to receive additional assistance from MTPA.

How can I get a prescription for Radicava?

Speak with your healthcare provider, who will initiate treatment (if appropriate).

Why is Radicava not available immediately after FDA approval?

Although MTPA is taking multiple steps to accelerate getting Radicava to the United States, there are certain portions of the complex supply chain that could not be done prior to approval. For example, FDA approval of the manufacturing facility and certain required customs clearances.

MTPA is working to get the medicine into the U.S. as quickly as possible. In addition to manufacturing hundreds of thousands of IV bags ahead of FDA approval and taking steps to prepare logistics ahead of time, the company will be air-shipping Radicava to further accelerate supply of the medicine from Japan where it is manufactured to the U.S.

How does the Searchlight Support program work?

Searchlight Support is a program designed to help people with ALS prescribed RADICAVA access the medicine and to support people once they are taking the medicine. As soon as a patient receives a RADICAVA prescription and then opts in to participate in the program, Searchlight Support will initiate:

• Case Manager: Every person who opts in will have access to a dedicated, personal case manager who will help them investigate and secure coverage through their insurance. The case manager will assist the person with ALS through the process.
• Bridge Program: MTPA created a program in which eligible patients may receive Radicava at no charge for up to two months while their insurance is making a coverage determination.
• Clinical Educator Support: The challenges of living with ALS or caring for a loved one with the disease are immense. Under Searchlight Support, people who sign up can ask a clinical educator questions about ALS, Radicava, or our support programs. These clinical educators are available to answer product or insurance-related questions day or night.

For more information, call 844-SRCHLGT or 844-772-4548. Patients prescribed RADICAVA should check eligibility requirements for each aspect of our patient assistance program as restrictions apply.

Can Searchlight Support help with my transportation?

When you are first prescribed Radicava and opt in to Searchlight Support, your case manager can help you identify transportation options in your area.

Where can I learn more about Radicava and MT Pharma America’s patient support service and assistance program, Searchlight Support?

Call 844-SRCHLGT or 844-772-4548. People prescribed Radicava should check eligibility requirements for each aspect of our patient assistance program as restrictions apply.

Can I get Radicava before August?  

There is currently no Radicava available in the United States. As soon as it arrives in the U.S. and is delivered to treatment centers and/or health care provider offices, patients will have immediate access. MTPA expects Radicava to be available in August.

Do I need to travel to an ALS Center? 

Radicava is given to patients through an IV, and your insurance coverage may determine whether it is administered at an outpatient center, in your home or someplace else. MTPA will ship Radicava to ALS Centers, and to specialty pharmacies that can deliver it to patients who will receive treatments in their homes.

What if I have clinical questions about Radicava?  

The challenges of living with ALS or caring for a loved one with the disease can be immense. Under Searchlight Support, patients who sign up can ask a clinical educator questions about ALS, Radicava, or MTPA support programs. These clinical educators are available to answer product or insurance-related questions day or night.

 

ALS and MDA

What causes ALS?

The causes of the vast majority of ALS cases are still unknown. Although the majority of ALS cases are sporadic, meaning there is no family history of the disease, about 5 to 10 percent of cases are familial, meaning the disease runs in the family.

In addition to the familial cases, investigators theorize that some individuals may be genetically predisposed to developing the disease, but only do so after coming in contact with an environmental trigger. The interaction of genetics and environment may hold clues as to why some individuals develop ALS. One thing doctors and scientists do know is that ALS cannot be “caught,” or transmitted from one person to another.

How many people in the United States have ALS?

An estimated 5,000-6,000 Americans are diagnosed each year with ALS, an incurable disease that affects the nerve cells in the brain and spinal cord. Initial symptoms can be subtle at first, and it can take 12 to 14 months to be accurately diagnosed with ALS.

What everyday freedoms and abilities does ALS take away?

ALS results in muscles that are weak and soft, or stiff, tight and spastic. Muscle twitches and cramps are common. Symptoms may be limited to a single body region, or mild symptoms may affect more than one region. When ALS begins in the bulbar motor neurons, the muscles for swallowing and speaking are affected first. As ALS progresses, symptoms become more widespread, and some muscles become paralyzed while others are weakened or unaffected. In late-stage ALS, most voluntary muscles are paralyzed.

What is the life expectancy for someone with ALS?

Each person’s disease course is unique. Standard longevity statistics cite an average survival time of three to five years after diagnosis.

What has MDA done to support people living with ALS today?

• Since inception, MDA has dedicated more than $363 million to ALS research and support services.
• MDA is proud of our decades-long approach to providing coordinated expert care through our nationwide network of more than 150 MDA Care Centers and nearly 50 designated MDA ALS Care Centers.
• We have facilitated direct patient care to tens of thousands of individuals living with ALS over more than half a century.
• MDA is committed to continuing to increase quality and standards of care, and is proud of its ongoing collaborative efforts with policy makers and stakeholders in the neuromuscular and rare disease community, and beyond, to ensure that every effort possible is made to free individuals — and the families who love them — from the harm of ALS and related neuromuscular diseases.
• MDA convenes annual conferences, where leading scientists, researchers, and medical professionals specializing in neuromuscular disease confer and collaborate with the aim of accelerating scientific discoveries, drug development and clinical care.
• We provide support and services to help improve quality of life and help our families thrive, including an equipment assistance program, support groups and educational materials.

 How has MDA been involved in ALS research?

• MDA has invested over $1 billion in neuromuscular disease research grants since 1950, with more than $156 million dedicated to research for ALS.
• MDA currently has 44 active ALS grants with a total funding commitment of $11.4 million.

 Has MDA been involved in development of Radicava for ALS?

MDA has not been directly involved in development of Radicava for ALS, but we have supported research investigating the role of oxidative stress in ALS and other neuromuscular disorders as well as therapeutic approaches to combat the damage caused by free radicals.