News
Market Authorisation in the EU

On Monday 12 February 2024 the European Commission announced its approval for market authorisation of Skyclarys in Europe. This was subsequent  to the positive recommendation of Skyclarys on the 15 December 2023 by the European Medicines Agency (EMA).

The European Commission’s approval of Skyclarys is a significant milestone towards expanding global access, bringing the first approved treatment to the Friedreich’s Ataxia community in the EU.

FARA Ireland is grateful to all the researchers, clinical sites, individuals with Friedreich’s Ataxia and their families, patient organizations, Biogen and

the European Medicines Agency for the research, drug development and advocacy efforts that led to this approval. We look forward to continuing our collaboration with the Friedreich’s Ataxia community with the goal of expanding access where it is needed.

It is hoped that Skyclarys can address some of the significant unmet need by bringing the first treatment to people living with Friedreich’s ataxia in Ireland.

However, it is important to understand that European regulatory approval does not mean that patients can be prescribed a medicine and that the HSE will fund it. The HSE is the decision-making body which determines whether a medicine is both clinically and cost-effective for patients.

For more details please sign up for the advocacy campaign at the link below:

https://forms.gle/vXabvW9T31kAfLXY6

News
Positive News Regarding Skyclarys® Approval in the EU

Like many of you, we have been closely following the developments in the approval of Skyclary’s in Friedreich’s ataxia (FA). We were very excited to see that the FDA approved Skyclary’s for the treatment of adults with FA in the US on 28th February 2023.

Whilst this is an amazing step forward, we are obviously keen for people with FA to potentially get access to the drug in the Ireland too. 

Reata pharmaceuticals (now owned by Biogen), who developed Skyclary’s, submitted an application to the regulatory agency in Europe (the EMA) at the end of 2022.

On 15 December 2023 we got some great news, this was from the European Medical Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), the regulatory body that makes recommendations on drug approvals in the EU. The CHMP has recommended a market authorization for Skyclarys® for people (16 and older) with FA in the EU.  

The final decision will need to be made by the European Commission, in the first quarter of 2024. However, this was a significant hurdle to overcome.

In order for Skyclarys® to be approved in the Ireland, the EMA approval now has to signed off by European Commission which normally takes 67 days. Once this is done Biogen Ireland must make a rapid review to the Irish regulator. This usually takes about 4 weeks but due to the cost of the drug it is assumed the rapid review will be denied. Biogen Ireland would then need go through the ‘Health technology assessment’ process, where decisions are made on funding of the drug. 

FARA Ireland is working with Biogen and doing all we can to ensure access to Skyclarys® for people with FA in the Ireland as soon as possible. As part of this Process information is needed to help people get access to the drug if it is approved. We will continue to work with Biogen and will keep you updated with progress. 

See Biogen’s press release at the link below:

https://investors.biogen.com/news-releases/news-release-details/chmp-issues-positive-opinion-biogens-skyclarysr-omaveloxolone

Uncategorized
Useful Equipment

I have had FA for a number of years and I have compiled a list of items I have found useful over the last 20 years as I have progressed.

  • When standing or walking un-aided became challenging I started to use a posterier walker called a Kaye walker (Posterier as it offer me more stability)
  • For standing upright stationary I used an easy stand stander
  • For walking and transfering I have ankle foot orthoses (AFO) {also known as splints]
  • i have a manual wheelchair [be careful not to get one to wide]
  • I use a scooter for rough ground.
  • I have an electric wheelchair [of which there is varies types] and a folding electric wheelchair (handy for traveling)
  • Wheelchair assistive devices I found useful were Wijits and New Drive
  • I use a wet room with a shower wheelchair to shower
  • To help transferring I use hoists and banana boards
  • For cooking and eating I use a lap tray and a dishwasher
  • I use assistive technology such as google home and alexa, and wifi bulbs
  • I drive with Hand controls
Personal Experiences
Speech and Swallow

I am an FAer and I have problems with my speech and swallow. I have noticed people with FA often rank speech and communication problems as one of the top three symptoms of their condition.

Did you know it takes about 100 muscles speak? Muscles that have to work in close collaboration and with precision. FA attacks the body’s ability to coordinate its muscles, including those used to speak and communicate.

Problems communicating can have significant impact on people:

  • frustration with others and themselves about not getting the message across
  • negative self-image or depression
  • less motivation to speak with others, leading to social withdrawal and isolation

Tools I have been given to help me are;

  • Exercises to strengthen the head and neck control to aid with swallowing difficulties.
  • Humming and vocalisation exercises to enhance voice projection.
  • Diaphragmatic Breathing to reduce breathlessness.

Another area I am told I could explore is Voice Banking;

Voice banking has 3 options, Voice Build, Voice Repair and Voice Design which will allow anyone with FA to have a synthetic voice that sounds how you want, no matter how badly your voice is already affected. Ask your speech and language therapist if you are a candidate for voice banking.

  

Voice Build 

Voice Build is a synthetic replica of your own voice that can be used in your communication device. This is ideal for people whose FA is likely to impact their speech but who have not yet had any significant symptoms.  

Voice Repair 

This is for people who have already noticed considerable changes in their speech. For example, if you struggle articulating some words or sound more breathy than normal, Voice Repair can repair this damage in your synthetic voice.  

Voice Design 

This is for people who have no intelligible speech and enables you to design your synthetic voice based on your chosen characteristics such as regional accent, age, and gender.  

News
Call for Action

To support the advocate(s) please complete the google form below on the link below:

https://docs.google.com/forms/d/e/1FAIpQLSfqhN3O7nINLBsiX3lfBUeX1sSF_ntFpOPrty508-RNqClGVA/viewform?usp=sf_link

Aims

  1. To find out the exact number of people with FA in Ireland.
  2. To assist with getting drugs approved and funded in Ireland.

Background to Advocacy Campaign

On the 28 February 2023 the Food and Drug Administration (FDA) in USA approved SKYCLARYS (Omaveloxolone, ‘Omav’ for short) for those over 16 years of age with FA. This is the first treatment ever approved for people with FA.

In Ireland, ‘Omav’ will first need to be approved by the European Medicines Agency (EMA), which is based in Amsterdam before the Irish agency, Health Products Regulatory Authority (HPRA), will review it. Assuming, the EMA and HPRA approve ‘Omav’ for use in FA and given that the price of ‘Omav’ is $370,000 per person per year, it will then be necessary to apply to the Health Service Executive (HSE) to fund the drug.

Creating a Friedreich’s Ataxia (FA) Database in Ireland 

Currently in Ireland, it is not clear how many people have FA. The coding system that is currently used in Irish hospitals does not have a specific code for FA. Therefore, we need to find out the exact number of people in Ireland with FA to help with drug approval specifically for:

  1. Reata (Drug company producing ‘OMAV’) 
  2. For the drug to be approved in Ireland Reata must apply to the Irish Authorities. This application takes time and costs money for Reata. As we are a small country, we need to show them the amount of potential FAer’s in need of the drug. An estimate is not strong enough reason we need concrete numbers so Reata can see potential customers. 
  • Health Service Executive (HSE) 
  • The drug is currently $370,000 per person per year. At this price, a cost versus benefit analysis [also known as a Health Technology Assessment (HTA)] will need to be done by the HSE.
  • The more FAer’s we can prove in Ireland the more ethical responsibility on the government to provide funding for the drug for FAer’s to have the same human rights as non-FAer’s. 
  • The HSE also need to know their potential financial exposure which is only possible if we have an exact number of people with FA in Ireland.

Become an Advocate for FA in Ireland 

Together we are stronger. If the FA community are not all together, we could send mixed messages to Reata and the HSE, etc. Therefore, it is crucial that we work together and have one voice. I am proposing myself, Brona Kearney, as an advocate now. In time I hope other FAer’s/family members, etc. will volunteer and assist in this work. I would hope the advocate(s) will:

  • Ask the FA community their thoughts, opinions, and get feedback from them.
  • Be a representative for FAer’s. 
  • Give the FA community updates on the drug approval process. 
  • Ask for help or advice from the FA community when needed.
  • Get the FA community to assist with a petition (if necessary).

About Brona Kearney – Current FA Patient Advocate 

I’m in my early 30’s, I was diagnosed with scoliosis at 9 years and FA at 10 years. I live in Dunlavin, Co Wicklow in the family home with my parents. I have an older brother and sister. My sister also has FA. I did Accounting in College and since then I have completed professional qualifications in accounting and tax.

Currently, I am a Civil Servant, working 4 days a week from home.  I take a Wednesday off to rest, mid-week.  When I’m not working, I take the opportunity to exercise i.e. horse riding, riding my trike (three wheel bike), physiotherapy exercises, e.g. walking in posterior walker using ankle foot orthosis (AFO), hydrotherapy and floor exercise.  I use a manual wheelchair, predominately to get around. I have a car and I use hand controls to drive it.

I became an advocate for FA because I want to help others with FA and I feel I have something to offer on the topic. Something, I live by, is ‘if it feels like it’s the right thing to do, just go and do it’. Being an advocate for FA in Ireland feels right for me now and I hope we can all help one another.

Contact the advocate(s) at advocateforfainireland@gmail.com

News
Omav!

The first treatment for Friedreichs Ataxia it has been approved in the US

The video below explains a little bit about this new drug. It was produced by FARA