Skyclarys (omaveloxolone)
Research Synopsis
- * The drug acts by activating the Nrf2 pathway, which enhances antioxidant activity and helps protect cells from the oxidative stress that leads to nerve cell damage associated with Friedreich's ataxia.
- * A study published in June 2023 confirms that Skyclarys is currently the only FDA-approved treatment for managing the symptoms of Friedreich's ataxia, a condition caused by frataxin protein deficiency and mitochondrial dysfunction.
- * While showing promise, omaveloxolone does not cure the disease, highlighting a need for continued research into potential gene therapies and other innovative treatments for Friedreich's ataxia.
- * Recent research emphasizes the Nrf2 pathway's importance in regulating cellular stress responses and its potential impact on other neurodegenerative diseases, suggesting that future therapies might aim to enhance its activation while minimizing side effects.
- * In studies regarding administration methods, it was found that omaveloxolone can be effectively mixed with applesauce for patients having trouble swallowing capsules, maintaining similar drug absorption and tolerability as intact capsules.
- * Analysis of genetic variants linked to Friedreich's ataxia has revealed a majority have detrimental effects on frataxin protein function, contributing to condition severity and underscoring the urgency for targeted treatment options like omaveloxolone.
- * Skyclarys' development signifies crucial progress in therapeutic strategies against Friedreich's ataxia, yet patients and healthcare providers are encouraged to explore complementary treatments and ongoing clinical trials.
- * Overall sentiment in the literature indicates cautious optimism regarding the utility of omaveloxolone, recognizing its role as a foundational treatment while continuing to seek advancements in therapy development.
- * Future research is focused on integrating omaveloxolone with novel treatment strategies to enhance its efficacy and address the unique challenges posed by Friedreich's ataxia.
Skyclarys (omaveloxolone)
Omaveloxolone: First Approval.
London, UK
2 hours ago
1 Received
- - Omaveloxolone (SKYCLARYS™) is a new oral medication developed by Reata Pharmaceuticals to treat Friedreich's ataxia, a condition linked to nerve cell damage caused by oxidative stress.
- - The drug works by activating the Nrf2 pathway, which helps protect cells from damage by increasing antioxidant activity and preventing the breakdown of Nrf2.
- - In February 2023, omaveloxolone received FDA approval for treating Friedreich's ataxia in adults and adolescents aged 16 years and older, marking a significant milestone in its development.
$100 - $150
Hourly Rate
Omaveloxolone (Skyclarys) for patients with Friedreich's ataxia.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Emerging small molecule inhibitors of Bach1 as therapeutic agents: Rationale, recent advances, and future perspectives.
London, UK
2 hours ago
1 Received
- Nrf2 is a key transcription factor that regulates the cellular response to stress and promotes the expression of genes that protect cells by detoxifying drugs, modulating the immune system, and managing iron metabolism.
- Current Nrf2 activators, used in treating diseases like multiple sclerosis and Friedreich's ataxia, have side effects due to their non-specific actions, and Nrf2 is negatively regulated by Bach1, a protein that can interfere with Nrf2's activation, especially in neurodegenerative diseases like Parkinson's.
- To maximize the therapeutic effects of Nrf2 activation, combining strategies to stabilize Nrf2 and inhibit Bach1 is essential, and recent research is focusing on developing small molecule inhibitors of Bach1 to
$100 - $150
Hourly Rate
Pharmacotherapeutic strategies for Friedreich Ataxia: a review of the available data.
London, UK
2 hours ago
1 Received
- Friedreich ataxia (FRDA) is a rare genetic disorder that leads to various health issues like coordination loss and problems with the nervous and cardiovascular systems, with only one FDA-approved treatment available.
- The disease is caused by a lack of the frataxin protein, which results in mitochondrial dysfunction; currently, omaveloxolone (Skyclarys) is the sole approved treatment for managing FRDA symptoms.
- While omaveloxolone marks significant progress in treatment, it's not a cure, and future therapies involving gene therapy or other innovative approaches may offer more effective, long-term solutions.
$100 - $150
Hourly Rate
An In Silico Analysis of Genetic Variants and Structural Modeling of the Human Frataxin Protein in Friedreich's Ataxia.
London, UK
2 hours ago
1 Received
- * The disease is linked to mutations in the frataxin (FXN) gene that disrupt iron regulation within cells, causing harmful iron buildup and oxidative stress that lead to neuron death.
- * A study analyzed 226 FXN genetic variants, confirming a majority have harmful effects on protein function, and created a 3D model of FXN to understand significant mutations (I154F and W155R), revealing they disrupt essential protein interactions critical for its maturation. *
$100 - $150
Hourly Rate
[Not Available].
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Relative Bioavailability of Omaveloxolone When Capsules Are Sprinkled Over and Mixed in Applesauce Compared With Administration as Intact Omaveloxolone Capsules: A Phase 1, Randomized, Open-Label, Single-Dose, Crossover Study in Healthy Adults.
London, UK
2 hours ago
1 Received
- Omaveloxolone (SKYCLARYS) is now approved to treat Friedreich ataxia in patients 16 and older in the U.S. and EU, with a recommended dose of 150 mg daily.
- A study tested the option of sprinkling the capsule contents on applesauce for patients who struggle to swallow capsules, with results showing similar drug absorption compared to taking intact capsules.
- The sprinkled method was found to be well tolerated, with no serious side effects, and is now included in prescribing information for both the U.S. and EU.
$100 - $150
Hourly Rate