Orphan Drugs in Epilepsy (Topics in Epilepsy)

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Epygenix Therapeutics, Inc. Dravet Syndrome, a lifelong form of epilepsy, begins in the first year of life with frequent or prolonged seizures. Intellectual disability, behavioral abnormalities, gait and motor dysfunction, and increased mortality are commonly observed as the disease progresses.

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Patients with Dravet Syndrome also suffer with life-threatening seizures that cannot be adequately controlled by available medications. They also face a 15 to 20 percent mortality rate due to SUDEP Sudden Unexplained Death in Epilepsy , which are commonly seizure-related accidents such as drowning or infections.

The Difference between Seizures and Epilepsy

In most cases, Dravet Syndrome is caused by heterozygous de novo mutations or gene deletions of SCN1A, a gene encoding a brain voltage-gated sodium channel Nav1. The effectiveness of EPX was discovered using a proprietary phenotype-based zebrafish drug screening platform. The zebrafish harbors 82 percent of human disease-associated genes, and shares many physiological and metabolic pathways with humans. Using the zebrafish model for Dravet Syndrome, drug candidates were identified from a screen of more than 3, drugs that suppress seizures and symptoms associated with neurological diseases.

Acorda Therapeutics Committed to Working with FDA

It is characterized by multiple types of seizures. People with Lennox-Gastaut syndrome begin having frequent seizures in early childhood, usually between ages 3 and 5. More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably.

Orphan Drugs: Rare Diseases, Rare Funding

Almost all children with Lennox-Gastaut syndrome develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with Lennox-Gastaut syndrome require help with usual activities of daily living.


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Epidiolex, taken along with other medications, was shown to be effective in reducing the frequency of seizures when compared with placebo. The most common side effects that occurred in Epidiolex-treated patients in the clinical trials were: sleepiness, sedation and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue, malaise and weakness; insomnia, sleep disorder and poor quality sleep; and infections.

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As is true for all drugs that treat epilepsy, the most serious risks include thoughts about suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression and panic attacks. Epidiolex also caused liver injury, generally mild, but raising the possibility of rare, but more severe injury. Funding for research and development of drugs and treatments for orphan diseases can come from within an institution, from philanthropy or from patient advocacy groups like those represented by the National Organization for Rare Disorders NORD.

The federal government also has avenues through which funds are available; the FDA issues orphan grants, and the Rare Disease Clinical Research Network at NIH funds 22 orphan disease consortia, each focusing on a single rare disorder or family of like rare disorders. Although they have different approaches and specialties, the commonality of their goal remains—make progress for orphan drug research and development.

In addition, they provide orphan drug regulatory support and are heavily vested in educating the community, students and medical professionals about the rare lysosomal diseases they study—even hosting a Rare Disease Day program to attract attention from students and patient advocacy leaders. Having worked extensively with seizure disorders, Cloyd and his team are currently in active drug development for promising new therapies that may allow benzodiazepines to be administered even before a seizure starts, perhaps in the form of a pen or spray.

In addition, researchers at CODR are working on a rescue therapy for epileptic patients—one that may allow providers to determine the severity of a case even sooner to expedite treatment. She has worked with inherited metabolic disorders that occur because of a mutation in a gene resulting in a defective protein.

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We are conducting studies to understand how this medication works and determine when and how long boys should be treated. The two scientists, both reliant on laboratory-based research to conduct their studies, say they genuinely want to help those afflicted. You could see the stress and the desire to have something better. Together with a pediatric neurologist, we set out to deliver that something.

The RODC aims to model these orphan diseases in such a way that provides reproducibility and easy access to foundations, pharmaceutical companies and scientists.