The FDA announced today that it has approved deflazacort (brand name Emflaza), under development by Marathon Pharmaceuticals, to treat Duchenne muscular dystrophy (DMD).
There are a lot of questions about the newly approved drug — from what is it, to how much will it cost, to how can it be accessed. Read below to learn the answers and find out about important safety information, and be sure to check back for updates at mda.org.
What is Emflaza?
Emflaza, developed by Marathon Pharmaceuticals, is the first drug in the United States approved for use by patients who are 5 years or older with DMD, regardless of genetic mutation.
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
Marathon has made Emflaza available in an immediate-release tablet formulation at multiple dosages (6 mg, 18 mg, 30 mg, 36 mg) as well as in an oral suspension formulation (22.75 mg/mL).
How soon will Emflaza be available?
Marathon plans to make Emflaza available as quickly as possible. You can begin the process of obtaining a prescription for the drug now.
How can I get a prescription for Emflaza?
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, Marathon’s 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’s Expanded Access Program?
If you have been receiving deflazacort through Marathon’s AccessDMD Expanded Access Program, you already should have been informed that this program will end shortly after FDA approval. You will need a new prescription for FDA-approved Emflaza. Be sure to speak with your healthcare professional.
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 will receive communications by email (if email address was shared) and a call from an EMFLAZACares patient support specialist with information on next steps. In the meantime, patients and/or their caregivers should contact their healthcare 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 Marathon’s patient support service and assistance program, EMFLAZACares?
Marathon has established a website and toll-free number to provide information and help answer 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 Marathon’s 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
How is Marathon helping with access to Emflaza?
Marathon is committed to creating the most comprehensive, robust patient assistance programs allowed by law.
- Here is an overview of how the process will work for qualified patients:
- An EMFLAZACares patient support representative will verify coverage for Emflaza under a patient’s respective insurance.
- If patients with commercial insurance have out-of-pocket costs associated with Emflaza, Marathon will offer a copay assistance program.
- If patients need additional assistance or are recipients of state or federal insurance, patients will be referred to independent charitable assistance foundations who might be able to help, if the patient qualifies, as determined solely by the independent charitable foundation.
- If patients are uninsured, Marathon will offer a free goods program.
- In addition, patients taking Emflaza will receive convenient and complimentary specialty pharmacy home delivery. The pharmacy will monitor the status of refills and will contact patients and/or their caregiver 7-10 days before the next refill is due.
- The EMFLAZACares program also includes access to dedicated nurse case managers who can help with the following:
- Education and support
- Reimbursement information
- Tips for working with health care professionals
- Connect families with support groups
- Listen to and support caregivers
- Answer questions about Emflaza or Duchenne muscular dystrophy
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.
Based on feedback from the payer community and others, Emflaza will be priced in the bottom 10th percentile of rare disease drugs approved for a similar patient population size. Emflaza will have the same flat price for all tablet dosage strengths and nearly all tablet dispensing combinations, so there’s no disincentive to increase the dose as the patient weight increases.
What will be the anticipated cost of Emflaza to most patients?
Marathon expects that for U.S. patients there should be very low or no out-of-pocket costs for Emflaza, with limited exceptions.
Marathon has developed a comprehensive patient support services program and is committed to helping ensure U.S. patients who are prescribed Emflaza have access to it.
Will patients need to pay more than they were paying out of pocket by importing the drug?
Based on the EMFLAZACares patient assistance programs developed by Marathon, it is estimated that a standard prescription drug co-pay for Emflaza will be less than what families spent historically out of pocket to import deflazacort.
How does Marathon plan to ensure that patients with Duchenne already on deflazacort will continue to have access to Emflaza?
Marathon is committed to ensuring a smooth transition for patients who have been accessing deflazacort from overseas pharmacies. Marathon understands the importance of continuity of care and has oriented its EMFLAZACares patient and healthcare provider support programs to address this critical need.
Marathon previously set up the Deflazacort Transition Program (www.deflazacort.us), an optional program that allowed patients taking deflazacort in the U.S. to consent to be contacted by Marathon following U.S. FDA approval of Emflaza. This service was designed to connect patients and caregivers with Marathon’s patient support services as quickly as possible.
If a healthcare provider and patient (or caregiver/guardian) wishes to continue deflazacort therapy upon the approval of Emflaza, the healthcare provider can simply complete an Emflaza prescription start form and submit it to EMFLAZACares, Marathon’s patient support services program. A prescription start form is available at www.EMFLAZA.com.
Marathon is working with a top specialty pharmacy that understands the unique needs of patients with Duchenne. The specialty pharmacy will work with the patient’s third-party payer and the prescribing healthcare provider to provide any documentation required by the third-party payer. Marathon’s goal is to simplify the process for patients and caregivers.
Will insurance companies require the use of a generic corticosteroid before agreeing to cover Emflaza?
Marathon does not expect widespread insurance company requirements to use another generic corticosteroid that is not approved by the FDA for Duchenne prior to using Emflaza for Duchenne, especially for those currently on a version of deflazacort. However, insurance plans make independent decisions about their benefit design and prior authorization policies based on a balance of factors.
Marathon is fully committed to working with the payer and provider to establish coverage. In the interim, it’s important to continue therapy, especially with a drug like this. Therefore, Marathon will continue to provide free product via the bridge supply program.
For the most accurate information on insurance plan coverage of Emflaza, please call EMFLAZACares and speak with a patient support specialist.
What services are available if a prior authorization is required by the insurer?
Marathon will provide comprehensive reimbursement support services and work with healthcare providers during the prior authorization process:
- If a prior authorization for Emflaza is required, a dedicated team within the specialty pharmacy will work with the healthcare provider, patient/caregiver and insurance company/third-party payer to provide required information.
- HCPs could be asked to provide supporting literature, chart notes, medical necessity statements to support the prior authorization and/or appeals process.
- The specialty pharmacy will diligently follow up with the third-party payer and healthcare provider on a routine basis until a determination is made.Marathon will provide bridge medicine at no cost to help ensure access in the event that insurance coverage determination is delayed.
Now that Emflaza is approved, will patients still be able to get deflazacort from abroad? If so, for how long?
Marathon has no control over deflazacort availability from overseas pharmacies, and will be focused on making Emflaza available to patients in the U.S. Marathon is working very closely with healthcare providers and Masters, a UK-based pharmacy that has been a large supplier of deflazacort, to help ensure a smooth transition. To that end, Marathon has developed robust patient support services including patient assistance programs and will be using a specialty pharmacy to provide prescription fulfillment and support.
Marathon’s commitment is that every patient who needs Emflaza will have access to it.
What’s the difference between deflazacort currently available abroad and Marathon’s deflazacort product, Emflaza?
Both Emflaza (Marathon’s deflazacort) and Calcort (Sanofi’s version of deflazacort) met the formal criteria for bioequivalence for the active metabolite, based on the results of a bioavailability study conducted by Marathon. That is to say, 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. The labeling for Emflaza contains no limitation of use regarding the concurrent use of Emflaza and Exondys 51 in patients living with Duchenne.
What work was involved in getting Emflaza approved by the FDA? Why was this necessary?
Prior to Marathon’s involvement, no one had conducted the studies required for an FDA New Drug Application for deflazacort. Since 2011, Marathon has conducted those studies on Emflaza, taking the drug through a full clinical and pre-clinical program. Marathon has conducted 17 studies (eight clinical pharmacology and safety studies and nine preclinical studies). In addition, Marathon has invested in the licensure of two important clinical studies of deflazacort, one of which was among the largest clinical studies ever conducted in this patient population. Marathon worked with the initial investigators to obtain all available data which was then used to perform robust and comprehensive analyses. One of those studies became the pivotal phase 3 study, which was published in the peer-reviewed medical journal Neurology in August 2016.
Does Marathon plan any future additional studies of Emflaza in DMD?
Marathon is committed to further research of Emflaza to better understand its effect on pediatric populations as well as its cardiac and pulmonary effects in those who are non-ambulatory.
One planned study will examine various dosage regimens in younger patients with Duchenne, ages 3 to 5 years, to determine if earlier intervention is safe and effective and ultimately impacts the course of the disease in a more positive manner. A second planned study will examine various dosage regimens in non-ambulatory patients with Duchenne and characterize the pulmonary and cardiac effects of deflazacort in these patients. Finally, Marathon is planning to fund a study that examines potential new uses of Emflaza in Duchenne.
Does Marathon plan to pursue the development of other drugs for Duchenne, or other rare diseases? How robust is Marathon’s pipeline?
Marathon is committed to the development of drugs for Duchenne and other rare neurological diseases. Working with its Scientific Advisory Board, key opinion leaders and advocacy advisors, Marathon is constantly looking for opportunities that meet these criteria.
Marathon is also funding early research opportunities that its scientific team finds compelling.Marathon’s current pipeline is advancing therapies for Duchenne, infantile spasms and idiopathic inflammatory myopathies.
INDICATION Emflaza is indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.
IMPORTANT SAFETY INFORMATION
Contraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in Emflaza.
Do not stop taking EMFLAZA, or change the amount you are taking, without first checking with your healthcare provider, as serious side effects or even death can occur. Always talk to your doctor before stopping or changing your dose.
People taking Emflaza may be at increased risk for infection, diabetes, high blood pressure, osteoporosis, cataracts or glaucoma, serious skin rashes, stunted growth and behavioral and mood disturbances. It is important to talk with your doctor about whether Emflaza is appropriate for you or your child.
Emflaza may interact with other medications you are taking, including prescription drugs, over-the-counter medications, dietary supplements and vaccines. Tell your doctor about all of the medications you or your child are taking before beginning treatment with Emflaza.
Common side effects that could occur with EMFLAZA include:Facial puffiness or Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, frequent daytime urination, unwanted hair growth, central obesity, and colds.
Please see the accompanying full Prescribing Information
You may report side effects to ProPharma Group at 1-866-562-4620 or drugsafety@propharmagroup.com.
You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see the accompanying full Prescribing Information
You may report side effects to ProPharma Group at 1-866-562-4620 or drugsafety@propharmagroup.com.
You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
