The FDA on Feb. 9, 2017, approved deflazacort (brand name Emflaza) for the treatment of Duchenne muscular dystrophy (DMD). In April, South Plainfield, N.J.-based PTC Therapeutics acquired all rights to Emflaza, and on May 5 the company announced plans to launch the drug “in the coming weeks” at a cost of $35,000 per year.

There is a lot of interest in the drug and what the latest news means, and we wanted to share some answers to the most common questions we have received.

As always, you can direct any additional questions to the MDA National Resource Center at 800-572-1717 or resourcecenter@mdausa.org, and be sure to check mda.org for updates.

Emflaza

What is Emflaza?

Emflaza is the first drug in the United States approved for use by people who are 5 years or older with DMD, regardless of genetic mutation. Prior to gaining approval in the U.S., a version of the drug already had been approved in Europe.

Emflaza is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects. In clinical studies, those treated with Emflaza:

• Showed improved muscle strength
• Showed slower decline in functional ability

What are corticosteroids?

Corticosteroids (also known as glucocorticoids) are medications that suppress the immune system and decrease inflammation. They commonly are used to treat some neuromuscular disorders, including DMD.

Can Emflaza be used to treat all forms of DMD?

Emflaza potentially may treat all forms of DMD, regardless of genetic mutation. Individual circumstances and experiences may vary, however. It’s important to talk with your physician about whether treatment with Emflaza is right for you or your child.

Is there a specific age range for which Emflaza will work?

Emflaza is approved for use by patients who are 5 years or older with DMD.

Will Emflaza cure DMD?

Treatment with Emflaza will not cure Duchenne.

Will Emflaza help me or my child?

• Treatment with Emflaza has been shown to slow the loss of muscle strength and function, preserve cardiac and respiratory function, and reduce the incidence of scoliosis (curvature of the spine) in people with DMD.
• Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with Emflaza as compared to other steroids.
• To learn more, please contact your or your child’s physician.

Is Emflaza significantly better/different from other corticosteroids?

Yes. In clinical studies, patients treated with Emflaza experienced similar benefits to those who were treated with prednisone. However, those who received Emflaza experienced fewer severe side effects such as weight gain, glucose intolerance (diabetes), loss of bone mass and behavioral issues.

What’s the difference between deflazacort available abroad and PTC Therapeutics’ deflazacort product, Emflaza?

Both versions of the drug produce similar blood levels of the active metabolite.

Can Emflaza be taken by those on Exondys 51? How do the two treatments compare?

As always, patients should speak to their healthcare provider regarding their medical care and treatment options. The labeling for Emflaza contains no limitation of use regarding the concurrent use of Emflaza and Exondys 51 in people living with Duchenne.

What work was involved in getting Emflaza approved by the FDA? Why was this necessary?

Marathon Pharmaceuticals conducted the activities necessary to gain FDA approval in the United States — including studies to demonstrate safety and efficacy. FDA approval makes it possible for insurance companies to cover the cost.

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 DMD and other diseases. For some diseases, including DMD, comprehensive treatment may involve multiple drugs that target different disease mechanisms.

Cost

What will Emflaza cost?

PTC Therapeutics announced on May 5 that treatment with Emflaza is expected to cost, on average, $35,000 per year.

Families can reach PTC’s support programs via a website and toll-free number. For questions about Emflaza, EMFLAZACares and accessing the treatment, visit www.EMFLAZA.com or call 1-844-EMFLAZA (1-844-363-5292) to learn more.

What will be the amount billed to my insurance for Emflaza per year?

The price insurance companies pay will vary according to discounts and rebates that will be negotiated directly with each insurer.

What will be the anticipated out-of-pocket cost of Emflaza to most patients?

PTC has developed a patient support services program to help ensure U.S. patients who are prescribed Emflaza have access to it.

Will patients still be able to get deflazacort from abroad?

PTC Therapeutics has no control over deflazacort availability from overseas pharmacies and will be focused on making Emflaza available to patients in the U.S.

A website and toll-free number are available to those with questions about Emflaza, EMFLAZACares and accessing the treatment. Visit www.EMFLAZA.com or call 1-844-EMFLAZA (1-844-363-5292) to learn more.

Do you think the $35,000 price tag is justified?

MDA does not engage in price setting for drugs. We are, however, concerned with ensuring that the appropriate therapies are available to those who most need them and are committed to helping connect patients and families with information and guidance in seeking support through PTC’s access programs.

Access

How soon will Emflaza be available?

PTC Therapeutics on May 5 reported that it plans to make Emflaza available “in the coming weeks.”

How can I get a prescription for Emflaza?

PTC urges anyone who may be interested in getting a prescription for Emflaza to begin the process now. As soon as the forms are started, work can begin on obtaining prior authorization and insurance coverage.

Here are the appropriate steps to take to get a prescription for Emflaza:

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

2. The healthcare provider will initiate an Emflaza prescription start form, which is available on www.EMFLAZA.com and will include patient information, insurance information and consent forms.

3. The healthcare provider will submit the completed prescription start form to EMFLAZACares, PTC Therapeutics’ patient support services program.

4. A patient support specialist from EMFLAZACares will be in touch to review current insurance coverage, provide information about the qualifications for and benefits from patient assistance programs, and make arrangements for Emflaza delivery.

5. You are welcome to call EMFLAZACares at 1-844-EMFLAZA (1-844-363-5292) to learn more about the patient support services and speak to a nurse case manager.

What if I already received deflazacort through Marathon Pharmaceuticals’ Expanded Access Program?

The AccessDMD Expanded Access Program will continue until Emflaza is in full production. Patients who currently are getting the drug for free through this program will continue to receive it until the drug is ready through the specialty pharmacy.

You will need a new prescription for FDA-approved Emflaza. Be sure to speak with your health care professional.

Will I be able to continue importing deflazacort from overseas?

Masters Pharmaceutical will fill a 90-day supply prior to product launch in the United States. Once Emflaza becomes available in the U.S., the drug will no longer be able to be shipped across the border.

What happens next for those who previously signed up for information on www.deflazacort.us?

If a patient and/or caregiver signed up with Marathon’s Deflazacort Transition Program at www.deflazacort.us, they should already have received communications by email (if email address was shared) and a call from an EMFLAZACares patient support specialist with information on next steps. Patients and/or their caregivers should contact their health care professional to discuss Emflaza. All patients will need a new prescription even if they are currently receiving Emflaza or deflazacort from the Expanded Access Program or an overseas pharmacy.

Where can I learn more about Emflaza and PTC Therapeutics’ patient support service and assistance program, EMFLAZACares?

A website and toll-free number are available to those with questions about Emflaza, EMFLAZACares and accessing the treatment. Visit www.EMFLAZA.com or call 1-844-EMFLAZA (1-844-363-5292) to learn more.

What are some key benefits of PTC Therapeutics’ patient support services?

The benefits of EMFLAZACares services are as follows:

• Potentially low or no out-of-pocket costs
• Bridge program that provides treatment at no cost to patients with delays awaiting insurance verification for most patients
• Copay assistance program for those who qualify
• Help with transitioning from an overseas pharmacy or Expanded Access Program
• Free drugs program for those who qualify

Will insurance companies require the use of a generic corticosteroid before agreeing to cover Emflaza?

Yes. In order for insurance plans to cover Emflaza, a prior steroid treatment regimen is required. This means the patient will need to have been treated with prednisone for 3-6 months with no apparent benefit.

Was this medication already being used in the United States by patients?

Some individuals and families in the United States have been purchasing deflazacort from overseas suppliers and paying for it out-of-pocket.

Can I get this drug for my son who is under the age of 5? Or who has Becker muscular dystrophy?

This would be considered off-label use and would be at the discretion of the prescribing physician. Be sure to speak with your health care professional.

If people already were able to access the drug, why was it necessary to get it approved by the FDA?

Marathon Pharmaceuticals conducted the activities necessary to gain FDA approval in the United States — including studies to demonstrate safety and efficacy. FDA approval makes it possible for insurance companies to cover the cost.

DMD and MDA

What causes DMD?

DMD is caused by a mutation (flaw) in the gene that codes for dystrophin, a protein that helps keep muscle cells intact.

How many people in the United States have DMD?

While the incidence and prevalence of DMD is not known with absolute certainty, the incidence of the disease in the U.S. population is estimated to be between 1:3,500 to 1:6,000 male births and prevalence estimates range from approximately 8,000 to 17,950.

What everyday freedoms and abilities does DMD take away?

• Symptoms vary, but it is expected that a boy diagnosed with DMD will lose the ability to walk by the time he is 13 years old, making a wheelchair necessary to preserve mobility.
• By age 18, boys who have DMD will most likely have lost the ability to feed themselves, stripping them of one of the most basic functions of independence.
• The ability to breathe without assisted ventilation typically is lost by the age of 19.

What is the life expectancy for someone with DMD?

In the last 50 years, improved standards of care have increased life expectancy for people diagnosed with DMD, from 14 years in the 1960s to 25 years in the 1990s and beyond.

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

• MDA is proud of our decades-long approach to providing coordinated expert care through our nationwide network of more than 150 MDA Care Centers.
• We have facilitated direct patient care to tens of thousands of individuals living with DMD over more than half a century.
• MDA is committed to continuing to increase quality and standards of care, and we are proud of our 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 DMD 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 MDA Summer Camp for kids with DMD and related neuromuscular diseases, an equipment assistance program, support groups and educational materials.

How has MDA been involved in DMD research?

• MDA has invested more than $1 billion in neuromuscular disease research grants since 1950, with more than $200 million dedicated to research for DMD.
• MDA facilitated the identification of the gene that is flawed in DMD and the protein that it specifies. These breakthroughs paved the way for precision medicine therapies that specifically target the underlying genetic defects of DMD.

How has MDA been involved in development of corticosteroids to treat DMD?

• MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including Emflaza, in DMD, with studies to determine the drugs’ mechanism of action, drug effects, side effects and best dosing regimen.