Approved treatments are medications that have been deemed safe and effective by regulatory agencies such as the U.S. Food and Drug Administration. Each country has its own regulatory approval process that involves the review of clinical trial data. SKYCLARYS is an approved treatment for Friedreich’s ataxia (FA) in people age 16 and older.
There are also several potential treatments being investigated on the Drug Development Pipeline. Additionally, the FA Clinical Management Guidelines describe different general medications, interventions, and physical therapy for the management of FA.
Accessing a rare disease medication is usually a bit more complex than getting a prescription for a common medication. Click the button below to download a frequently asked questions guide about accessing rare disease medications in the US. Some of the different steps in the process are also explained to the right.
Some insurance companies may require a patient’s health care provider to seek approval for coverage of a medication prior to the prescription being written. This is called a prior authorization. The process of submitting a prior authorization request is different for each insurance company. Some insurance companies may have prior authorization criteria, or a list of standards, that the patient must meet for the prior authorization to be approved. Often, insurance companies use the Food and Drug Administration (FDA) label for the medication to create their prior authorization criteria. The FDA label indicates who is eligible to be prescribed the medication (for example, individuals with a diagnosis of FA within a specific age range). Sometimes insurance companies may use prior authorization criteria that does not follow the FDA label and creates extra restrictions on who can be prescribed the medication. If someone with FA does not meet an insurance company’s prior authorization criteria, they receive an insurance denial. This denial can be challenged through the appeals process.
There are several reasons why your insurance may have denied coverage for a medication. Some of these reasons include:
Your insurance company should have sent you a letter or other type of communication notifying you of the denial and explaining why you were denied. The specific reason(s) for denial should be referenced when appealing the denial.
The appeal process is different for each insurance company, and you should work with your doctor and insurance company to figure out how to proceed. Often, the first step is submitting an appeal letter arguing that the medication is a medically necessary treatment for FA. Sometimes, multiple rounds of appeals are necessary to gain approval. Higher levels of appeal could include a peer-to-peer conversation between the patient’s health care provider and a clinician who works with the insurance company. Use the resources below to help with your appeals.
SKYCLARYS (omaveloxolone) is a once daily oral medication that was shown in clinical trials to slow progression of FA symptoms. It is approved in many countries for the treatment of people with FA 16 years of age and older.
Watch the video on the right to learn how SKYCLARYS impacts mitochondrial function. Additional information about the research that led to SKYCLARYS’s approval can be found on the SKYCLARYS Drug Development Program page.
Like many rare disease drugs, SKYCLARYS cannot be picked up at a local pharmacy.
When prescribing SKYCLARYS, doctors first must enroll their patient in Biogen REACH, a program that assists people with FA and their doctors during the prescription process. Representatives from REACH will work with the patient’s insurance company to obtain coverage for SKYCLARYS. The prescription process is different from person to person depending on an insurance company’s policies and coverage. Once approved for SKYCLARYS, the patient will receive the prescription through the mail.
As of April 2025, SKYCLARYS is approved for people living with FA in the U.S., E.U., Canada, UK, and Brazil who are ages 16 and older. While approval is an important milestone, individual countries will still determine access through their own processes. Biogen, the company that owns and manufactures the drug, is currently undertaking efforts to expand approval in additional countries and in children, and is working to address gaps in access.
It is not uncommon for people affected by rare diseases to face difficulties when seeking insurance coverage for medications, medical equipment, or other types of care. It’s important to know that in most cases, there are steps patients and healthcare providers can take to appeal a denial. FARA provides resources about the insurance approval process, as well as template documents to support insurance appeals.
Some insurance companies may require a patient’s health care provider to seek approval for coverage of SKYCLARYS prior to the prescription being written. This is called a prior authorization. The process of submitting a prior authorization request is different for each insurance company. Some insurance companies may have prior authorization criteria, or a list of standards, that the patient must meet for the prior authorization to be approved. Many insurance companies use the SKYCLARYS Food and Drug Administration (FDA) label, which states that patients must be over the age of 16 and have a genetic diagnosis of FA. Other insurance companies create strict criteria that many FA patients are unable to meet, often leading to a denial. These denials can and should be appealed because they do not align with the FDA’s label.
There are several reasons why your insurance may have denied coverage for SKYCLARYS. Some of these reasons include:
Your insurance company should have sent you a letter or other type of communication notifying you of the denial and explaining why you were denied. The specific reason(s) for denial should be referenced when appealing the denial.
The appeal process is different for each insurance company, and you should work with your doctor and insurance company to figure out how to proceed. Often, the first step is submitting an appeal letter arguing that SKYCLARYS is a medically necessary treatment for FA. Sometimes, multiple rounds of appeals are necessary to gain approval. Higher levels of appeal could include a peer-to-peer conversation between the patient’s health care provider and a clinician who works with the insurance company. Use the resources below to help with your appeals.
The Biogen REACH program is a resource available to people with FA taking or trying to access SKYCLARYS. REACH Family Access Managers may be able to contact the insurance company to determine the proper steps for appealing a denial and answer questions about the process. They may also be able to investigate options for accessing SKYCLARYS without insurance approval through different free or reduced-price drug programs you may be eligible for.
Most insurance companies accept appeal letters from both doctors and patients or caregivers, and you can find a template below for each scenario. The letters explain results from the SKYCLARYS clinical trials, why SKYCLARYS is a medical necessity for people with FA, and that any insurance criteria excluding people with specific symptoms or disease states does not align with the Food and Drug Administration label for SKYCLARYS. You or your doctor must fill out [bracketed areas] of the letter templates with information specific to your FA symptoms, care, and reason for insurance denial.
Options may be available to help people in the U.S. afford their prescribed medication.*
Biogen Family Access Managers with Biogen REACH can help explore affordability options.
*Subject to eligibility and program terms and conditions. REACH affordability options are not healthcare insurance.