FDA & Reata Pharmaceuticals:
Allow Individuals with Friedreich Ataxia Access to Omaveloxolone
74,070 individuals signed on to the FA Community Response letter requesting Reata Pharmaceuticals submit a New Drug Application (NDA) on an urgent basis and FDA consider approval of an NDA for omaveloxolone in FA based on the existing evidence from clinical trials.
FARA issued a Call to Action in the form of the FA Community Response letter asking individuals to sign-on in support of a request to FDA and Reata that they work together to approve omav for FA as quickly as possible. FARA shared the FA Community Response letter via our website, email lists and social media channels. Sign-on opportunity was open from January 5-20, 2021.
Click the buttons below to read the full petition and cover letter that were submitted to FDA and Reata Pharmaceuticals.
Community Petition Cover Letter
View the Submitted Petition
51 FA clinical experts and the investigators who participated in the MOXIe studies have provided a separate letter that discusses the clinical significance of the MOXIe trial and omav results.
Healthcare Provider Letter of Support
At the links below, you will find a copy of the FA Community Response Letter and 13 letters of support from FA and Ataxia organizations, writing on behalf of the FA patients they represent and joining FARA in making the request of FDA and Reata to urgently submit a marketing application, approve and provide access to omaveloxolone.
FA Community Response Letter
Letters from Advocacy Organizations
Click a heading below to read more on that topic:
Voice of the Patient
The voice of the patient is critical to the drug development process and FDA has been a strong advocate for identifying opportunities for the “patient voice” to inform and guide drug development. The 21st Century Cures Act requires sponsors to include and FDA to consider the patient perspective in making approval decisions for new drugs. The FA community contributed its patient voice in June 2017, when FARA hosted the externally-led Patient Focused Drug Development meeting on FA for FDA which revealed that nearly 100% of people with FA experience neurological symptoms including loss of balance and difficulty walking, loss of coordination of movement in the upper and lower limbs and fatigue. We described to FDA that these neurological symptoms have an enormous effect on quality of life as they lead to lost ability to perform activities of daily living and loss of independence. Many of these neurological symptoms (balance, gait, upper limb function, and speech) are measured in the clinic with a structured functional exam called the modified Friedreich Ataxia Rating Scale (mFARS). Data from an ongoing, large, prospective, longitudinal, FA natural history study has demonstrated that neurological symptoms as assessed by mFARS get progressively worse over time and that the mFARS score is highly correlated to activities of daily living in FA. We worked with FDA to improve this scale and ensure it is included in clinical trials in FA
Background
There are currently no approved treatments for FA. However, recently a well-controlled clinical trial of omav in 103 individuals with FA demonstrated a statistically significant, placebo-corrected 2.40-point improvement in mFARS after 48 weeks of treatment (p=0.014). In addition, individuals in the treatment arm reported improvements in activities of daily living, such as walking, quality of sitting position and swallowing compared to the placebo group. Given the positive clinical trial results, favorable safety profile of omav, and difficulty conducting clinical trials in FA especially during the current pandemic environment, we are asking FDA and Reata to work together to provide access to omav for people with FA as soon as possible.
Rationale
FA is a devastating, progressive and life-shortening rare genetic condition that affects children and adults. All individuals with FA suffer neurological symptoms which include loss of coordination of movement in the upper and lower limbs, loss of balance and gait ataxia leading to loss of ambulation and loss of independence in performing activities of daily living (eating, writing, dressing, bathing, etc.). Other common symptoms include dysarthria (speech difficulty), fatigue, cardiomyopathy, arrhythmia, and diabetes. The average life expectancy for individuals with FA is about 35 years.
Omaveloxolone targets a specific cellular dysfunction in FA and has been demonstrated in clinical trials to improve disease specific biomarkers and meaningful neurological clinical outcomes and activities of daily living. Omav has also been demonstrated to be safe and well-tolerated.
Given the clinical trial results, FA patient families and clinicians strongly encourage Reata and the FDA to work together promptly to give people with FA, who currently have no other choice for treatment, access to omav as soon as possible. FDA has emphasized the importance of the patient voice, especially for rare conditions without FDA-approved treatment options. As summarized in the report on The Voice of the Patient: Friedreich Ataxia, people with FA experience severely compromised quality of life, loss of independence and early mortality due to symptoms of FA. The majority of patients reported that balance/walking, upper limb function and fatigue have the highest impact on quality of life and treating even one of these individual symptoms would be meaningful. Ninety-five percent (95%) of individuals indicated that slowing or stopping disease progression would be extremely meaningful to them when considering a drug therapy. The patients and clinicians of the FA community are fully aware of the clinical trial results evaluating the use of omav in FA and are convinced that the results demonstrate meaningful benefit and low risk. More than 95 percent of eligible individuals who participated in the clinical trials elected to enter the open-label extension study to continue their access to omav while awaiting formal analysis of the trial data and regulatory review. We ask 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 omav in FA based on the existing evidence from clinical trials.