Abstract Details

Title A Single-center Experience with Omaveloxolone in Friedreich Ataxia

Topic Movement Disorders

Presentation(s) P4 - Poster Session 4 (8:00 AM-9:00 AM)

Poster/Presentation
Number 16-013

Objective Evaluate the real-world use of Omaveloxolone in Friedreich Ataxia (FA).

Background FA, is an autosomal recessive neurodegenerative disorder, showing a suppressed Nrf2 pathway, increase oxidative stress, mitochondrial dysfunction and damage to cells. Omaveloxolone (150 mg per day) significantly improved neurological function compared to placebo when evaluated in a phase II trial in FA patients.

Design/Methods Patients starting Omaveloxolone underwent baseline and follow-up visits after 3 and 6 months. Neurological assessments included the modified FA rating scale (mFARS), and FA activities of daily living (FA-ADL) scores. Laboratory analyses included liver enzymes and lipid profile. Variables were assessed with a linear mixed model for repeated measures. Patients were divided based on their median baseline mFARS score (£63.5 or >63.5), and baseline FA-ADL score (£17 or >17).

Results We enrolled 60 patients (31 female), with a mean age of 41.3±13.6 and a disease duration of 22.1±10.6 years. Mean±SD baseline mFARS was 58.8±18.1 and decreased to 58.5±16.9 after 3 months and 56.7±15.2 after 6 months (p=0.111). Patients with higher baseline mFARS score were more prone to improvement during the 6-month follow-up (mean difference 2.68, p=0.046). The mean FA-ADL did not show any change (p=.645) during follow-up from baseline (15.2±7.5) to month 3 (15.2±7.5) and month 6 (15.5±6.9). Patients with higher baseline FA-ADL showed a small improvement during follow-up (mean difference 1.309, p=.043). AST and ALT increased during follow-up, with a peak at the 3-month time-point and then decreased (p<0.001 for both). Eleven patients underwent temporary discontinuation for liver enzyme increase, pyrosis, stomatitis, patient’s decision, followed by a re-start with slow titration. Two patients discontinued Omaveloxolone permanently because of gastrointestinal side effects.

Conclusions Omaveloxolone was safe and well tolerated. mFARS and FA-ADL showed a higher improvement in patients with a more advanced disease and higher baseline scores at both scales.

Authors/Disclosures
Martina Gramaglia, MD PRESENTER	Dr. Gramaglia has nothing to disclose.
Alessio Sarnataro, MD	Dr. Sarnataro has nothing to disclose.
Giorgia Puorro	Giorgia Puorro has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Elma Research. Giorgia Puorro has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Alexion.
Angela Marsili	Angela Marsili has nothing to disclose.
Alessia Bonfini Rendina, PA	Miss Bonfini Rendina has nothing to disclose.
Chiara Pane, MD	Dr. Pane has nothing to disclose.
Francesco Sacca, MD, FAAN (University Federico II)	Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lexeo. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genpharm. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medpharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Madison Pharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zai Lab. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reata. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sandoz. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neopharm Israel. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson&Johnson. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Sacca has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Astrazeneca. The institution of Dr. Sacca has received research support from AIFA. The institution of Dr. Sacca has received research support from FARA.

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