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News & Press Releases

Here you will find the most recent organizational news from FARA, including information on events, and awareness and advocacy initiatives. To locate an article from a certain date, please use the archives on the right side of your screen.


Information on COVID-19 and Friedreich's Ataxia

There are now several vaccines for COVID-19 that have demonstrated safety and efficacy in human clinical trials. While FARA does not make medical recommendations and does not endorse any particular vaccine, as a resource for the community we have researched COVID vaccines and reached out to FA experts to create this information document on COVID-19 vaccines.

There are no special concerns with COVID vaccines for persons with FA. However, those with advanced disease may be especially vulnerable to COVID-19 complications; thus, while weighing the current safety and efficacy data, the risk/benefit balance is in favor of vaccination.

What is Immunity? Protection from an infectious disease. If you are immune to a disease, you can be exposed to it without becoming infected. Immunity can be acquired through previous infection with a disease or through Immunization, which is the process by which a person becomes protected against a disease through vaccination.

What is a Vaccine? A product that stimulates a person's immune system to produce antibodies to a specific disease, protecting the person from that disease. Vaccines are usually administered through needle injections but can also be administered by mouth or sprayed into the nose.

What COVID-19 vaccines are available? In the US, COVID-19 vaccines are being used under an Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA). The FDA has issued EUAs for the Pfizer-BioNTech COVID-19 Vaccine and the Moderna COVID-19 Vaccine to be distributed in the U.S. The Pfizer vaccine is approved for those 16 years of age and older while the Moderna vaccine is approved for those 18 years of age and older. The European Medicines Agency (EMA) has granted a conditional marketing authorization for the COVID-19 vaccines from Pfizer-BioNTech, Moderna and AstraZeneca. The Medicines and Healthcare products Regulatory Agency (MHRA) in the UK has also approved use of the Pfizer-BioNTech, Moderna and AstraZeneca vaccines.

Who is eligible to receive the first vaccine? The initial supply is limited and for the next few months, it may be that some groups will be vaccinated first based on scientific data and ethical considerations. For example, in many US states, first responders, medical personnel and residents of long-term care facilities have been prioritized to be vaccinated first.

As supply of the vaccinations increase, there will be more opportunity to access it. We encourage you to follow your local guidance on how vaccine is being distributed as there is much variability across countries, states and local counties. Many guidelines list underlying conditions where there was sufficient evidence from the COVID trials to draw conclusions on risk. There was not enough evidence on rare diseases for those conditions to be listed. However, individuals with FA should consult their healthcare provider and advocate to be given priority based on your specific underlying conditions (i.e., respiratory, heart, diabetes, etc.)

What are the differences between the types of vaccines?

mRNA vaccines - A new technology was used by both the Pfizer and Moderna to create the first vaccines that use messenger RNA, or mRNA. The mRNA directs cells to produce COVID virus protein and the immune system then responds by making protective antibodies to the COVID-19 virus. Note: The mRNA vaccines don't use any type of virus for delivery, the mRNA is inside lipid nanoparticles, very small particles of fats, to protect it until it is taken up into the cell. Both of these vaccines require two doses to be effective. They also need to be stored cold, but Pfizer's vaccine has to be stored at ultra-cold temperatures, which has made it a bit harder to a ship and distribute. Because these vaccines are based on a newer technology, there are less long-term data on safety and efficacy in many different age groups. These data are being generated now as larger and larger numbers of people are being vaccinated.

Virus Vaccines - Viruses are naturally good at getting genetic material into human cells and therefore can be used to carry the genetic code (“the gene”) for a COVID-19 protein into the body. Once there, the gene allows the body to develop immunity to COVID-19. Furthermore, although these viruses are good at carrying genetic material into cells, they are highly modified and unable to make people sick.

The virus delivery system being used for COVID is called adenovirus (AdV). AdV is a completely different virus than Adeno Associated Viruses (AAV) which are being used for gene therapy. Taking a vaccination using AdV will not cause your immune system to make antibodies against AAV.

The AstraZeneca/Oxford (AZ) vaccine approved in the UK contains a modified adenovirus (AdV), as does the vaccine being developed by Janssen Pharmaceuticals (a division of Johnson and Johnson). Each uses a modified virus to deliver the COVID protein. Novavax has produced a vaccine that carries a COVID protein on nanoparticles (nano is one billionth of a unit) given together with a proprietary adjuvant, a substance that hypes up the immune response. This vaccine has not yet been approved.

Are there any special considerations for FAers regarding COVID vaccines? Individuals should discuss COVID vaccines with their health care provider.

Currently, there is no reason to believe that any adverse effects of any of these vaccines is different in FA patients than in the general population. As these vaccines have now been administered to an ever-growing population, we now have a better understanding of the side effects. Note that the experts we consulted suggested that, once the vaccines are approved, the risk-benefit ratio in FA patients are weighted in favor of vaccination given the higher vulnerability of FA patients to COVID 19 complications.

As discussed above, some COVID vaccines are using a virus to help deliver the vaccination material. The viruses being used are modified versions of AdenoVirus (AdV). An antibody or immune response to adenovirus used in these vaccines does not generate a similar immune response to other viruses, including those currently being used to develop FA gene therapy. There is currently no reason to expect that a patient's eligibility for an FA gene therapy trial or the patient's safety in such a trial would be impacted by having received COVID 19 vaccines that use AdV or adenovirus.

Most of the gene therapy approaches currently under development for the treatment of FA are using Adeno-Associated Viruses (AAVs). Although the name may be confusing, these are completely different viruses than those used in COVID vaccine. AAVs were discovered because they were found with adenovirus, but they do not have the same genetic or protein makeup. Therefore, an antibody response to adenovirus used in several of the COVID vaccines does not generate a similar immune response to AAV. Just to reiterate, none of the vaccines that have been approved (Pfizer, Moderna, AstraZeneca) or are close to approval (Janssen or Novavax) raise concerns with development of antibodies to viruses currently used in FA gene therapy.

Anyone considering receiving a COVID vaccination should discuss the matter with his or her physician. FARA does not endorse or recommend any particular vaccination.

Please note: This is an evolving situation. The comments provided are based on information available at the time of posting - February 4, 2021.

References:
https://www.nature.com/articles/d41586-020-03326-w
https://www.nature.com/articles/s41401-020-0485-4
https://cen.acs.org/pharmaceuticals/vaccines/Adenoviral-vectors-new-COVID-19/98/i19
https://www.youtube.com/watch?v=nXn4KBJ4oD0
https://www.gov.uk/government/collections/mhra-guidance-on-coronavirus-covid-19#vaccinesand-vaccine-safety
https://www.fda.gov/emergency-preparedness-and-response/coronavirus-disease-2019-covid-19/covid-19-vaccines
https://www.ema.europa.eu/en/human-regulatory/overview/public-healththreats/coronavirus-disease-covid-19/covid-19-latest-updates

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Monthly Update - January 2021


Dear friends,

As many of you know, FARA has prepared a letter to send to the Food and Drug Administration (FDA) and Reata Pharmaceuticals to request that they Allow Individuals with Friedreich Ataxia Access to Omaveloxolone.
Read and Sign on to the letter
This letter requests Reata to submit a New Drug Application (NDA) on an urgent basis and FDA to exercise the flexibility granted by law and contained in FDA guidance in considering approval of an NDA for Omaveloxolone in FA based on the existing evidence from clinical trials.

In just four days, you have facilitated over 25,000 signatures in support of this letter! We are grateful to everyone who has signed the letter, especially members of our FA families who make up 23% of respondents. The statements and photos shared by FA families will be instrumental in drawing attention to the patient experience and voice. If you have not signed onto the letter, you can do so up until January 20th. The entire FA community (individuals with FA, family, friends, caregivers, supporters, other rare disease advocates) is invited to sign on.

More...

Read the Full Monthly Update
 

Webinar: FA Community Call to Action on January 5, 2021

Webinar: FA Community Call to Action - held on January 5, 2021
The sign-on period will remain open until January 20, 2021 at 5pm (Eastern)
Have Your Voices Heard: Be a Part of the FA Community's Request for Access to Reata and FDA

Watch the Recorded Webinar

(See below for more detailed information about MOXIe)

After consultation with regulatory advisors, FARA plans to submit a letter to both Reata and the Food and Drug Administration (FDA) that requests they work together to provide access to omaveloxolone for individuals with Friedreich's Ataxia as soon as possible. The letter will highlight the voice of the patient with a request for Reata to submit a marketing application to the FDA and for the FDA to review and consider approval of this application. The strength of this letter will depend in large part on input and testimony from the FA community. The voices and lived experiences of this community matter and will help add critical perspective and context to the MOXIe trial data points that are being discussed. Even if you did not participate in this clinical trial, it is important to hear your voices too. It is crucial that our response is respectful, informative, and representative of the FA patient experience.

We are at an important crossroads in Reata's development of omaveloxolone as a potential treatment for FA. Our clinical investigators and FARA have confidence in the data from the completed studies and would like FDA to use tools given to it under the law to exercise regulatory flexibility to avoid a delay of at least several years for access which would occur if Reata conducts another clinical trial prior to the drug being approved for use by FA patients in the U.S. Please register for this important session and learn how you can participate. Please also share this message within the FA community. The greater our unity, the stronger our message.   more...

Read the Full Text
 

Friedreich's Ataxia Center of Excellence at CHOP Awarded $1.275 Million to Advance Medical Research

The Friedreich's Ataxia Center of Excellence (COE) at Children's Hospital of Philadelphia (CHOP) was awarded $1.275 million by the Friedreich's Ataxia Research Alliance (FARA), along with the Hamilton and Finneran families and the CureFA Foundation, to support the development of breakthrough therapies to improve the quality of life for individuals with Friedreich's ataxia.

Friedreich's ataxia (FA) is a neurodegenerative condition that limits the production of frataxin, a protein in the mitochondria, the cells' energy generators. The condition damages the nervous system and impairs muscle coordination, causing problems with movement and sensation that get progressively worse. Individuals may also experience cardiac dysfunction, scoliosis, diabetes, vision and hearing difficulties. About 1 in 50,000 individuals worldwide have FA, including 5,000 cases in the United States. Symptoms typically appear between age 5 and 15, though the condition can sometimes appear later in life as well.   more...

Read the Full Article
 

Monthly Update - December 2020


Dear friends,

As 2020 draws to a close, I reflect on the challenges, accomplishments, and continued generosity of our FA our community. One of the challenges we faced this year was the combination of increased research opportunities and fewer fundraising events.

This past Tuesday, aka Giving Tuesday, we launched our #GiftOfResearch campaign to help us close the funding gap and accelerate research momentum. As always, our community answered the call. We are excited to announce that Giving Tuesday kicked off our year-end campaign by raising over $95,000 which included a $25,00 generous match from Foley, Baron, Metzger & Juip, PLLC and a FARA Board Director.

Thank you to everyone that participated by creating an online fundraiser, donating, or by sharing photos and videos of why the #GiftOfResearch is important to you.

When you give the gift of research this holiday season, you are providing research tools and insights into Friedreich's ataxia. As you plan your holiday shopping, please consider giving the #GiftOfResearch to a loved one- gifting them an opportunity to be a part of the effort to find meaningful treatments and a cure for FA.

More...

Read the Full Monthly Update
 

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