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The Latest Rett Syndrome Pipeline Developments and Their Implications for Patients | DelveInsight

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The Latest Rett Syndrome Pipeline Developments and Their Implications for Patients | DelveInsight

May 01
21:32 2023
The Latest Rett Syndrome Pipeline Developments and Their Implications for Patients | DelveInsight

DelveInsight’s, “Rett Syndrome Pipeline Insight 2023” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in the Rett Syndrome pipeline landscape. It covers the Rett Syndrome pipeline drug profiles, including Rett Syndrome clinical trials and nonclinical stage products. It also covers the Rett Syndrome therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Latest Developmental Activities in the Rett Syndrome Treatment Landscape

 

  • In March 2023, Neuren Pharmaceuticals, based in Melbourne, announced that its North American partner Acadia Pharmaceuticals had received US Food and Drug Administration (FDA) approval for Neuren’s lead candidate Daybue (trofinetide) to treat Rett syndrome in adults and children.

 

  • In February 2023, Unravel Biosciences, Inc., a therapeutics company that advances drugs for complex diseases by leveraging a machine-learned model of human health, announced the successful completion of its Type B pre-IND meeting with the US Food and Drug Administration (FDA) for its RVL001 program in Rett Syndrome.

 

  • In February 2023, PharmaTher Holdings Ltd., a leader in specialty ketamine pharmaceuticals, announced that the United States Food and Drug Administration had designated KETARX (racemic ketamine) as an orphan drug for the treatment of Rett Syndrome, a rare genetic neurological disorder.

 

  • In February 2023, Anavex Life Sciences Corp. announced the enrollment of 92 Rett syndrome patients in the ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 study in Rett syndrome patients aged 5 to 17 years (inclusive).

 

  • In January 2023, Neurogene Inc., a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced that the FDA has approved the Company’s Investigational New Drug (IND) application for NGN-401 to treat Rett syndrome.

 

Discover the recent breakthroughs happening in the Rett Syndrome Pipeline landscape @ Rett Syndrome Pipeline Outlook

 

Key Takeaways from the Rett Syndrome Pipeline Report

 

  • DelveInsight’s Rett Syndrome pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Rett Syndrome treatment.

 

  • The leading Rett Syndrome Companies include Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, Q-State Biosciences, AveXis, AMO Pharma, Neurolixis, Biohaven Pharmaceuticals, Echo Pharmaceuticals, Vyant Bio, Prilenia Therapeutics, StrideBio, Relmada Therapeutics, VICO Therapeutics, Taysha Gene Therapies, Sarepta therapeutics, and others

 

  • Promising Rett Syndrome Pipeline Therapies include Blarcamesine, EPI-743, Fingolimod, REL 1017, Triheptanoin, Cannabidivarin, STRX 230, Research programme: Rett syndrome therapeutics, AVXS 201, AMO 04, Pridopidine, NLX 101, BHV 5000, and others.

 

  • The Rett Syndrome companies and academics are working to assess challenges and seek opportunities that could influence Rett Syndrome R&D. The Rett Syndrome therapies under development are focused on novel approaches to treat/improve Rett Syndrome.

 

Rett Syndrome Emerging Drugs Profile

 

  • Trofinetide: Neuren Pharmaceuticals

Trofinetide (previously designated by Neuren as NNZ-2566) and NNZ-2591 are patented synthetic analogs of molecules which are derived from IGF-1 and occur naturally in the brain. IGF-1 is a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and for responding to injury and disease. . The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted Orphan Drug designation to Neuren for trofinetide in Rett syndrome. The FDA has also granted Fast Track designation. The drug also has a Rare Pediatric Disease designation. Neuren’s North American partner for trofinetide, ACADIA Pharmaceuticals, has announced positive top-line results from a Phase III trial in Rett syndrome.Neuren Pharmaceuticals reported that the US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) of trofinetide for the treatment of Rett syndrome, that was submitted by Neuren’s US partner Acadia Pharmaceuticals (Nasdaq: ACAD). The FDA had granted a Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of 12 March 2023.

 

  • ANAVEX2-73 (blarcamesine): Anavex Life Sciences

ANAVEX2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring cellular homeostasis. The drug is in phase III stage of clinical trial evaluation for the treatment of patients with Rett syndrome.

 

Get More Information on the Rett Syndrome Drugs and Companies of Report @ Rett Syndrome Ongoing Clinical Trials Analysis

 

Scope of the Rett Syndrome Pipeline Report

 

  • Coverage- Global

 

  • Rett Syndrome Companies- Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, Q-State Biosciences, AveXis, AMO Pharma, Neurolixis, Biohaven Pharmaceuticals, Echo Pharmaceuticals, Vyant Bio, Prilenia Therapeutics, StrideBio, Relmada Therapeutics, VICO Therapeutics, Taysha Gene Therapies, Sarepta therapeutics, and others

 

  • Rett Syndrome Pipeline Therapies- Blarcamesine, EPI-743, Fingolimod, REL 1017, Triheptanoin, Cannabidivarin, STRX 230, Research programme: Rett syndrome therapeutics, AVXS 201, AMO 04, Pridopidine, NLX 101, BHV 5000, and others.

 

  • Rett Syndrome Pipeline Segmentation: Product Type, Molecule Type, Route of Administration

 

Find out more about the list of FDA-approved drugs for Rett Syndrome @ Rett Syndrome Treatment Landscape

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Rett Syndrome: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Rett Syndrome – DelveInsight’s Analytical Perspective
  7. Late Stage Products (Preregistration)
  8. Trofinetide: Neuren Pharmaceuticals
  9. Drug profiles in the detailed report…..
  10. Late Stage Products (Phase III)
  11. Blarcamesine: Anavex Life Sciences
  12. Drug profiles in the detailed report…..
  13. Mid Stage Products (Phase II)
  14. Drug Name: Company Name
  15. Drug profiles in the detailed report…..
  16. Early Stage Products (Phase I)
  17. BHV 5000: Biohaven Pharmaceuticals
  18. Drug profiles in the detailed report…..
  19. Preclinical and Discovery Stage Products
  20. Drug Name: Company Name
  21. Drug profiles in the detailed report…..
  22. Inactive Products
  23. Rett Syndrome Key Companies
  24. Rett Syndrome Key Products
  25. Rett Syndrome- Unmet Needs
  26. Rett Syndrome- Market Drivers and Barriers
  27. Rett Syndrome- Future Perspectives and Conclusion
  28. Rett Syndrome Analyst Views
  29. Rett Syndrome Key Companies
  30. Appendix

 

Got Queries? Find out the related information on Rett Syndrome Preclinical and Discovery Stage Products @ Rett Syndrome Drugs, Unmet Needs and Recent Trends

 

About Us

DelveInsight is a Business Consulting and Market research company, providing expert business solutions for the healthcare domain and offering quintessential advisory services in the areas of R&D, Strategy Formulation, Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions.

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