The US Food and Drug Administration (FDA) has given orphan drug designation to a Friedreich’s ataxia drug called omaveloxolone

General
1

Omaveloxolone is a treatment for Friedreich’s ataxia that has been developed by Reata Pharmaceuticals. The US FDA has now granted omaveloxolone orphan drug designation based on promising results from the company’s early-stage research.

What is omaveloxolone?

Omaveloxolone works by activating Nrf2, which is a signalling process within our cells that is important for producing antioxidants. Antioxidants help defend against oxidative stress, which can damage cells.

Research has shown that Nrf2 signalling is impacted in people with Friedreich’s ataxia, which may contribute to the cause of the disease. Omaveloxolone is thought to restore Nrf2 signalling, thereby reducing the effects of the disease.

What has research shown so far?

The first part of the Phase II trial looked at the safety and effectiveness of omaveloxolone. It found that the drug was effective in restoring Nrf2 function and that this was associated with improved neurological function in patients. The second part of this Phase II trial will begin in the second half of 2017.

What is orphan drug designation?

https://raremark.com/friedreich-ataxia/articles/friedreichs-ataxia-treatment-new-orphan-drug-designation--566

1 Like
2

Thanks Alan🙂 I bet I’m not the only one who wonders if their own specific ataxia could benefit from improved neurological function😏xB

3

Hi,
I am presently in a double blind clinical trial for a drug called bhv-4157. It has only been a month, but will keep you posted

4

Yes, have always said that as the symptoms of ATAXIA overlap with so many other conditions, we can only benefit from “Looking outside the box”

(plus the more “others” do the less we have to do {in a financial context]) :grinning:

5

Looking forward to hearing more
-The patient is the Expert in their own condition

6

Thanks Alan,
You are so right. The patient knows his condition the best. For instance I am convinced my condition was brought about by too much dilantin- I also have epilepsy. I have since changed my meds ( my neurologist switched me over)

7

:smirk:Bob, I had one seizure in my sleep (1991​:thinking:) and experienced deja vous on a regular basis. After a scan, I was diagnosed with Temporal Lobe Epilepsy.

Because I needed to drive ( after a one year ban because of the seizure) I was obliged to take antiseizure medication.

I had several changes in medication but nothing ever stopped the deja vous🙄 After Carbamazaphine Retard ( slow release) I started to notice eye problems. The Neurologist ( an Epilepsy Consultant) dismissed my concerns… I went on to develop Iritis, uveitis, double vision and Nystagmus.

Around the same time, I started to experience what I now know, are Cerebellar ataxia symptoms. Disorientation coping with spatial awareness, lack of concentration when multitasking, dizziness, brain fog etc… But, because my balance was ‘off’ prior to taking the medication…:thinking:

Despite being back and forth to my doctor, it was all dismissed as depression. This went on for many years…leading to actual deep depression.

To end a long story​:smirk: After experiencing falls, I was officially taken off antiseizure medication in 2011, by a different Neurologist who diagnosed Spinocerebellar Ataxia (Idiopathic- unknown cause):smirk:

To date, I strongly suspect I have an Episodic Ataxia connected to Epilepsy (specifically deja vous episodes) and BPPV ( a type of vertigo). Recently, my Neurologist ( a genetics Professor) ) prescribed Acetazolamide, to try to alleviate BPPV ( I suffer dizziness turning over in bed). It made me extremely ‘foggy headed’ during the day, so I stopped taking it. Raising my neck and head using 3 pillows has proved helpful instead. I await the results of yet another blood test😏

So, the search continues​:smirk: Taking things day by day :blush:xB