Epilepsy

Researchers Combine Genetic Information with Electronic Medical Records to Pinpoint When Epilepsies Affect Children

Tuesday, August 11, 2020 - 3:06pm

Large initiatives to link genomic data with electronic medical records (EMR) are already underway to determine how existing genetic data can be linked to a lack of information about clinical outcomes.

Key Points: 
  • Large initiatives to link genomic data with electronic medical records (EMR) are already underway to determine how existing genetic data can be linked to a lack of information about clinical outcomes.
  • "This is really important as we need to understand the clinical features that children with genetic brain disorders, especially children with genetic epilepsies, develop over time.
  • "Electronic medical records are an untapped resource to learn about how very rare disorders present over time, which will allow us to include this information in our clinical practice.
  • Ganesan et al, "A longitudinal footprint of genetic epilepsies using automated electronic medical record interpretation."

Cenobamate Designated Promising Innovative Medicine (PIM) by the UK’s MHRA for Treatment of Drug-Resistant Focal-Onset Seizures in Adults

Tuesday, August 11, 2020 - 5:00am

Commenting on the announcement, Stuart Mulheron, General Manager UK & Ireland, Arvelle Therapeutics said: The MHRA PIM designation marks an important milestone for Arvelle.

Key Points: 
  • Commenting on the announcement, Stuart Mulheron, General Manager UK & Ireland, Arvelle Therapeutics said: The MHRA PIM designation marks an important milestone for Arvelle.
  • To date, cenobamate has been approved by the US Food and Drug Administration (FDA) for the treatment of partial-onset (focal) seizures in adults, and is commercially available in the U.S.
  • In March 2020, Arvelle announced the European Medicines Agencys (EMA) acceptance of the marketing authorization application (MAA) for cenobamate for the adjunctive treatment of focal-onset seizures in adults with epilepsy.
  • Cenobamate was discovered by SK Biopharmaceuticals and SK life science and is a new FDA-approved anti-seizure medication (ASM) for the treatment of partial-onset seizures in adults (also known as focal-onset seizures).

Global Dravet Syndrome Industry Assessment 2017-2030: Epidemiology, Pipeline, Drugs, Competitive Intelligence Analysis

Thursday, August 6, 2020 - 11:45am

The Dravet Syndrome epidemiology chapters provide insights about historical and current Dravet Syndrome patient pool and forecasted trend for every seven major countries.

Key Points: 
  • The Dravet Syndrome epidemiology chapters provide insights about historical and current Dravet Syndrome patient pool and forecasted trend for every seven major countries.
  • Incidence of Dravet Syndrome segments Dravet Syndrome epidemiology, Mutation positive cases, Mutation Cases by Types and Class, Seizures associated with Dravet Syndrome, Gender-specific cases and Age-specific cases of Dravet Syndrome.
  • Drug chapter segment of the Dravet Syndrome report encloses the detailed analysis of Dravet Syndrome pipeline drug.
  • The publisher performs Competitive and Market Intelligence analysis of the Dravet Syndrome Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc.

Zogenix Provides Corporate Update and Reports Second Quarter 2020 Financial Results

Wednesday, August 5, 2020 - 9:01pm

This was a significant milestone for Zogenix and those in the Dravet community seeking effective new treatment options.

Key Points: 
  • This was a significant milestone for Zogenix and those in the Dravet community seeking effective new treatment options.
  • Last week, we commenced the commercial launch of FINTEPLA in the United States and are very pleased with our early progress.
  • Research and development expenses for the second quarter endedJune 30, 2020, totaled$34.4 million, up from$27.1 millionin the second quarter endedJune 30, 2019, as the Company decreased spending in Dravet syndrome and expanded clinical activities in LGS and MT1621.
  • As ofJune 30, 2020, the Company had$390.2 millionin cash, cash equivalents, and marketable securities, compared to$251.2 millionatDecember 31, 2019.

Dravet Syndrome Market Insights, Epidemiology and Forecasts to 2030 - ResearchAndMarkets.com

Tuesday, August 4, 2020 - 2:34pm

The "Dravet Syndrome Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Dravet Syndrome Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The Dravet Syndrome epidemiology chapters provide insights about historical and current Dravet Syndrome patient pool and forecasted trend for every seven major countries.
  • Incidence of Dravet Syndrome segments Dravet Syndrome epidemiology, Mutation positive cases, Mutation Cases by Types and Class, Seizures associated with Dravet Syndrome, Gender-specific cases and Age-specific cases of Dravet Syndrome.
  • Drug chapter segment of the Dravet Syndrome report encloses the detailed analysis of Dravet Syndrome pipeline drug.

FDA Approves EPIDIOLEX® (cannabidiol) Oral Solution to Treat Seizures Associated with Tuberous Sclerosis Complex

Monday, August 3, 2020 - 12:00pm

FDA approval of this new indication is exciting news for those with refractory seizures due to tuberous sclerosis complex, said Justin Gover, GWs Chief Executive Officer.

Key Points: 
  • FDA approval of this new indication is exciting news for those with refractory seizures due to tuberous sclerosis complex, said Justin Gover, GWs Chief Executive Officer.
  • FDA approval of EPIDIOLEX in TSC is a tremendous step forward and our community applauds this positive development, said Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance.
  • In the U.S., EPIDIOLEX is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or tuberous sclerosis complex (TSC) in patients one year of age and older.
  • EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older.

Tuberous Sclerosis Alliance Lauds FDA Approval of Epidiolex® to Treat Seizures in People with Tuberous Sclerosis Complex

Saturday, August 1, 2020 - 2:43am

SILVER SPRING, Md., July 31, 2020 /PRNewswire/ --Today, the Tuberous Sclerosis Alliance (TS Alliance) lauds the U.S. Food and Drug Administration's (FDA's) approval of Epidiolex oral solution to treat seizures in people one year of age and older who have tuberous sclerosis complex (TSC).

Key Points: 
  • SILVER SPRING, Md., July 31, 2020 /PRNewswire/ --Today, the Tuberous Sclerosis Alliance (TS Alliance) lauds the U.S. Food and Drug Administration's (FDA's) approval of Epidiolex oral solution to treat seizures in people one year of age and older who have tuberous sclerosis complex (TSC).
  • "Up to 85 percent of those affected by TSC experience seizures at some point in their lifetime," explained TS Alliance President & CEO Kari Luther Rosbeck.
  • In 2018, the FDA approved the drug to treat people two years of age and older with Lennox-Gastaut syndrome (LGS) or Dravet syndrome.
  • Established in 1974, the Tuberous Sclerosis Alliance is an internationally recognized nonprofit organization dedicated to finding a cure for tuberous sclerosis complex while improving the lives of those affected.

Epilepsy Foundation is Challenging People to Wear Purple Lipstick on National Lipstick Day to Create Awareness about Epilepsy

Wednesday, July 29, 2020 - 12:30pm

LANDOVER, Md., July 29, 2020 /PRNewswire/ --To coincide with National Lipstick Day on July 29, the Epilepsy Foundation launched the #PurpleLipstickChallenge encouraging people to wear purple lipstick to bring awareness to epilepsy and seizures.

Key Points: 
  • LANDOVER, Md., July 29, 2020 /PRNewswire/ --To coincide with National Lipstick Day on July 29, the Epilepsy Foundation launched the #PurpleLipstickChallenge encouraging people to wear purple lipstick to bring awareness to epilepsy and seizures.
  • "In addition to raising funds, we want to create awareness about this brain disorder that can affect any of us.
  • We are challenging everyone to raise money for a good cause, including men because newsflash, us men can wear lipstick too!"
  • Based in Denver, CO, Kenzie started her charitable foundation, Fly 4 A Purpose , in support of epilepsy awareness and advancement.

Zogenix to Release Second Quarter 2020 Financial Results and Host Conference Call and Webcast on August 5

Wednesday, July 29, 2020 - 1:00pm

Zogenixis a global pharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.

Key Points: 
  • Zogenixis a global pharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.
  • The companys first rare disease therapy, FINTEPLA(fenfluramine) oral solution, C-IV has been approved by theU.S.FDA and is under review inEuropefor the treatment of seizures associated with Dravet syndrome, a rare, severe childhood onset epilepsy.
  • In addition, the company has two late-stage development programs underway: one for FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome, a rare childhood-onset epilepsy and one for MT1621, an investigational novel substrate enhancement therapy for the treatment of TK2 deficiency, a rare genetic disorder.
  • MT1621 is being developed through Modis Therapeutics, aZogenixcompany.

Praxis Precision Medicines Announces $110 Million Financing

Tuesday, July 28, 2020 - 1:59pm

We are pleased with and excited by this financing, which supports our continued mission to develop best-in-class therapies that deliver long-term value to human health, said Marcio Souza, president and chief executive officer of Praxis.

Key Points: 
  • We are pleased with and excited by this financing, which supports our continued mission to develop best-in-class therapies that deliver long-term value to human health, said Marcio Souza, president and chief executive officer of Praxis.
  • With three programs currently in clinical development for five indications, and additional programs in earlier stages of development, we are making great strides to bring new treatments to patients in the CNS space.
  • Praxis is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system, or CNS, disorders characterized by neuronal imbalance.
  • We have established a broad portfolio, including five disclosed programs across multiple CNS disorders, including depression, epilepsy, movement disorders and pain syndromes.