Tuberous sclerosis

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.

Tuberous Sclerosis Alliance and Seizure Tracker™ Partner to Promote Data Sharing and Biosample Collection

Friday, June 19, 2020 - 11:00am

SILVER SPRING, Md., June 19, 2020 /PRNewswire/ --Today the Tuberous Sclerosis Alliance (TS Alliance) and Seizure Tracker announced a partnership to promote data sharing and biosample collection from people with tuberous sclerosis complex (TSC), a rare genetic condition that causes tumors to form in vital organs.

Key Points: 
  • SILVER SPRING, Md., June 19, 2020 /PRNewswire/ --Today the Tuberous Sclerosis Alliance (TS Alliance) and Seizure Tracker announced a partnership to promote data sharing and biosample collection from people with tuberous sclerosis complex (TSC), a rare genetic condition that causes tumors to form in vital organs.
  • The Data Share system is designed to enable website and mobile diary users to link their personally reported data to partner organizations' managed databases.
  • "Combining the TS Alliance's Natural History Database and seizure outcome data from Seizure Tracker has the potential to change the way we think about and treat seizures in people living with tuberous sclerosis complex," says Robert Moss, Co-Founder and President of Seizure Tracker.
  • Reports generated on the Seizure Tracker system provide extensive data visualizations comparing therapies against seizure activity changes while highlighting trends.

Tuberous Sclerosis Complex: 2020 Pipeline Insights - Therapeutics, Products, Companies and More - ResearchAndMarkets.com

Monday, April 20, 2020 - 4:25pm

Tuberous Sclerosis Complex Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Tuberous Sclerosis Complex market.

Key Points: 
  • Tuberous Sclerosis Complex Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Tuberous Sclerosis Complex market.
  • A detailed picture of the Tuberous Sclerosis Complex pipeline landscape is provided, which includes the disease overview and Tuberous Sclerosis Complex treatment guidelines.
  • The assessment part of the report embraces in-depth Tuberous Sclerosis Complex commercial assessment and clinical assessment of the Tuberous Sclerosis Complex pipeline products from the pre-clinical developmental phase to the marketed phase.
  • How many Tuberous Sclerosis Complex emerging therapies are in early-stage, mid-stage, and late stage of development for the treatment of Tuberous Sclerosis Complex?

GW Pharmaceuticals submits Type II Variation Application to the European Medicines Agency (EMA) to expand the use of EPIDYOLEX®, (cannabidiol) oral solution, for the treatment of seizures associated with Tuberous Sclerosis Complex (TSC)

Friday, March 13, 2020 - 11:00am

If approved, this will be the third licensed indication for GWs cannabidiol oral solution in Europe.

Key Points: 
  • If approved, this will be the third licensed indication for GWs cannabidiol oral solution in Europe.
  • TSC is typically diagnosed in childhood.1
    The Type II Variation Application is based on data from a positive Phase 3 safety and efficacy study.
  • It is associated with an increased risk of autism and intellectual disability.1 The severity of the condition can vary widely.
  • A supplemental New Drug Application (sNDA) was submitted to the FDA in early 2020 for the treatment of seizures associated with Tuberous Sclerosis Complex (TSC).

GW Pharmaceuticals and Greenwich Biosciences Submit Supplemental New Drug Application to U.S. FDA for Epidiolex® (cannabidiol) for the Treatment of Tuberous Sclerosis Complex

Monday, February 3, 2020 - 12:00pm

The sNDA seeks to expand the Epidiolex label to include the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a rare genetic condition.

Key Points: 
  • The sNDA seeks to expand the Epidiolex label to include the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a rare genetic condition.
  • Epidiolex is currently approved in the U.S. to treat seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome and has been granted Orphan Drug Designation from theFDAfor the treatment of TSC.
  • We look forward to working with the FDA toward an expected approval later this year."
  • A supplemental New Drug Application (sNDA) has been submitted to the FDA for the treatment of seizures associated with tuberous sclerosis complex (TSC).

Tuberous Sclerosis Alliance Lauds Lundbeck And FDA For Sabril Label Expansion For Patients 2 To 10 Years Old

Monday, January 27, 2020 - 8:21pm

This new label expansion now means there will be no age gap for patients needing critical access to this medication, particularly those in the tuberous sclerosis complex (TSC) community.

Key Points: 
  • This new label expansion now means there will be no age gap for patients needing critical access to this medication, particularly those in the tuberous sclerosis complex (TSC) community.
  • "Two years ago, the TS Alliance became aware insurance companies were denying coverage for vigabatrin for our community members whose children were 3 to 9 years old," said Kari Luther Rosbeck, President & CEO.
  • These discussions eventually resulted in the TS Alliance funding a study led by Darcy Krueger, MD, PhD, Director of the Cincinnati Children's Hospital Medical Center TSC Clinic.
  • An updated Sabril label can be downloaded at www.tsalliance.org/sabril-change to show insurers the broadened approved age group since the label won't be immediately available in the medication's package insert.

Bcureful and Tuberous Sclerosis Alliance Announce Plans to Combine Efforts

Thursday, December 12, 2019 - 11:00am

SILVER SPRING,Md., Dec. 12, 2019 /PRNewswire/ --Today, Bcureful and the Tuberous Sclerosis Alliance (TS Alliance) announced plans to formally combine efforts, specifically by establishing the Bcureful Travel Fund at the TS Alliance.

Key Points: 
  • SILVER SPRING,Md., Dec. 12, 2019 /PRNewswire/ --Today, Bcureful and the Tuberous Sclerosis Alliance (TS Alliance) announced plans to formally combine efforts, specifically by establishing the Bcureful Travel Fund at the TS Alliance.
  • "We are grateful to Bcureful for all its work over the past eight inspiring years on behalf of the tuberous sclerosis complex (TSC) community," said Kari Luther Rosbeck, TS Alliance President & CEO.
  • The Tuberous Sclerosis Alliance is the only US-based nonprofit dedicated to finding a cure for tuberous sclerosis complex (TSC) while improving the lives of those affected.
  • Bcureful is dedicated to connecting patients to expert medical care and creating opportunities to advance research toward a cure for tuberous sclerosis complex.

Greenwich Biosciences, a GW Pharmaceuticals Company, Presents New Data for EPIDIOLEX® (cannabidiol) in Patients with Tuberous Sclerosis Complex (TSC) at the American Epilepsy Society Annual Meeting

Saturday, December 7, 2019 - 5:00pm

Caregivers and patients also reported overall improvement with EPIDIOLEX in 69% of patients on 25 mg/kg/day and 62% of patients on 50 mg/kg/day compared to 39% on placebo (p=0.0074 and p=0.0580, respectively).

Key Points: 
  • Caregivers and patients also reported overall improvement with EPIDIOLEX in 69% of patients on 25 mg/kg/day and 62% of patients on 50 mg/kg/day compared to 39% on placebo (p=0.0074 and p=0.0580, respectively).
  • We are pleased to present these results which demonstrate the potential of EPIDIOLEX to reduce both focal and generalized seizures associated with TSC, said Justin Gover, CEO, GW Pharmaceuticals.
  • Eight patients on EPIDIOLEX 25 mg/kg/day, 10 on 50 mg/kg/day and 2 on placebo discontinued treatment due to an AE.
  • The Company continues to evaluate EPIDIOLEX in additional rare conditions including tuberous sclerosis complex (TSC) and Rett syndrome.

GW Pharmaceuticals and U.S. Subsidiary Greenwich Biosciences to Present Data on EPIDIOLEX® (cannabidiol) Oral Solution at the American Epilepsy Society Annual Meeting

Thursday, December 5, 2019 - 12:00pm

; & Robert Wechsler, M.D., Ph.D.

Key Points: 
  • ; & Robert Wechsler, M.D., Ph.D.
    Cannabidiol (CBD) Treatment in Patients with Seizures Associated with Tuberous Sclerosis Complex: A Randomized, Double-blind, Placebo-controlled Phase 3 Trial Thiele et al.
  • (Sunday, December 8, Poster #2.437)
    Time to Onset of Cannabidiol (CBD) Treatment Effect and Resolution of Adverse Events Madan Cohen et al.
  • The Company continues to evaluate EPIDIOLEX in additional rare conditions including tuberous sclerosis complex (TSC) and Rett syndrome.
  • Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.