Volanesorsen

WAYLIVRA (volanesorsen): An Antisense Oligonucleotide Inhibitor of Apolipoprotein CIII (apoCIII) mRNA - Market Size, Forecasts, and Emerging Insights, 2019-2023 and 2024-2032 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, February 27, 2024

Further, it also consists of future market assessments inclusive of the WAYLIVRA market forecast analysis for lipodystrophy in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in lipodystrophy.

Key Points: 
  • Further, it also consists of future market assessments inclusive of the WAYLIVRA market forecast analysis for lipodystrophy in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in lipodystrophy.
  • WAYLIVRA (volanesorsen), an antisense oligonucleotide inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis Pharmaceuticals through its subsidiary company, Akcea Therapeutics to treat familial partial lipodystrophy (FPL).
  • Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA, leading to its degradation and preventing the translation of apoC-III protein.
  • What are the other emerging products available and how are these giving competition to WAYLIVRA for lipodystrophy?

Brazilian Regulatory Authority Grants Approval for Waylivra™ for Familial Partial Lipodystrophy

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Thursday, December 1, 2022

SOUTH PLAINFIELD, N.J., Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This is the first approval globally for Waylivra for the FPL indication. Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).

Key Points: 
  • SOUTH PLAINFIELD, N.J., Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agncia Nacional de Vigilncia Sanitria), has approved Waylivra (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil.
  • This is the first approval globally for Waylivra for the FPL indication.
  • Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).
  • ANVISA's approval for Waylivra for FPL was based on results from the Phase 2/3 BROADEN study in patients with familial partial lipodystrophy.

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 40th Annual J.P. Morgan Healthcare Conference

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Monday, January 10, 2022

SOUTH PLAINFIELD, N.J., Jan. 10, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST.

Key Points: 
  • SOUTH PLAINFIELD, N.J., Jan. 10, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST.
  • Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2021 accomplishments and highlight upcoming potential value-creating milestones.
  • The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com .
  • PTC successfully advanced its clinical pipeline in 2021:
    APHENITY, a Phase 3 registration-directed trial of PTC923 in phenylketonuria (PKU) was initiated.

Waylivra™ Receives Innovative Drug Category Pricing to Treat Familial Chylomicronemia Syndrome in Brazil

Retrieved on: 
Tuesday, January 4, 2022

Waylivra is the only treatment for familial chylomicronemia syndrome (FCS) in Brazil.

Key Points: 
  • Waylivra is the only treatment for familial chylomicronemia syndrome (FCS) in Brazil.
  • Category 1 classification is given to innovative treatments that provide greater efficacy than current standards of care, and it allows for pricing in line with international markets.
  • If approved, Waylivra will be the first approved treatment for FPL in Brazil, and this will mark the first approval globally for this indication.
  • "I am pleased withthe decision by CMED to grant FCS patients access to Waylivra in Brazil.

WAYLIVRA▼® (volanesorsen), the First and Only Therapy for Familial Chylomicronaemia Syndrome, an Ultra-Rare and Life-Threatening Condition, to Be Available on the NHS in Scotland

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Monday, November 9, 2020

This means that eligible patients will be able to access volanesorsen through NHS Scotland while additional clinical effectiveness data are gathered.

Key Points: 
  • This means that eligible patients will be able to access volanesorsen through NHS Scotland while additional clinical effectiveness data are gathered.
  • The SMC will review this data after three years and make a final decision on its routine use in NHS Scotland.
  • Volanesorsen, which received conditional marketing authorisation in Europe in May 2019, is the only therapy indicated for people with familial chylomicronaemia syndrome (FCS).
  • (2017) The APPROACH Study: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS).

WAYLIVRA▼® (volanesorsen), the First and Only Therapy for FCS, an Ultra-Rare and Life-Threatening Condition, to Be Available on the NHS in England

Retrieved on: 
Friday, September 18, 2020

This important milestone will allow patients with this severely debilitating condition to access treatment on the NHS, drastically improving their quality of life.

Key Points: 
  • This important milestone will allow patients with this severely debilitating condition to access treatment on the NHS, drastically improving their quality of life.
  • Akcea Therapeutics initially made volanesorsen available to patients with FCS through the Early Access to Medicines Scheme (EAMS).
  • Since initiation of the EAMS, 22 patients with FCS have been treated with volanesorsen, at no cost to the NHS.
  • Volanesorsen will be the second treatment that Akcea Therapeutics has made available through the NHS in the last year.