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Neurofibromatosis Therapeutics Pipeline is Driven by Unmet Medical Needs

Published Date:   January 2018

Neurofibromatosis currently exhibits a growing pipeline with 25 therapeutic candidates, P&S Intelligence

Neurofibromatosis therapeutics pipeline in 2018

The study analyzed that the Neurofibromatosis therapeutics pipeline comprises of approximately 25 therapeutic candidates in different stages of development.

Access Report Summary with TOC on "Neurofibromatosis Therapeutics - Pipeline Analysis 2018, Clinical Trials & Results, Patents, Designations, Collaborations, and Other Developments" at:

Insights on pipeline segments

Majority of the pipeline drug candidates are being developed to be administered by oral route. Celldex Therapeutics Inc. and Merck Co. Inc. are in the process of developing a monoclonal antibody drug candidate, CDX0158, and Pembrolizumab respectively for the treatment of NF. Lixte Biotechnology Holdings Inc. is in the process of developing two drug candidates, in the Pre-Clinical stage of development for the treatment of neurofibromatosis (NF). In September 2016, the USFDA granted Orphan Drug Designation to BXCL101, a drug candidate of BioXcel Therapeutics Inc. for the treatment of NF type 2.

Unmet medical needs driving the neurofibromatosis therapeutics pipeline

Since there is no treatment available for NF, the growing medical needs of the people suffering from the disease are driving its therapeutics pipeline growth. High rate of recurrence/progression of the disease and poor long-term benefit of surgery can be viewed as a limitation of this treatment option.

Positive clinical trial results propelling the growth of the pipeline therapeutics of the Neurofibromatosis

The drugs being developed by the different pharmaceutical companies demonstrated positive clinical trial results which in-turn increasing their further development. For instance, In June 2014, Array BioPharma Inc. announced positive results of Phase I/II, combination of binimetinib and LEE011. Selumetinib being developed by the AstraZeneca plc. And it met primary endpoint as confirmed partial responses (tumor volume decreases from baseline of ≥20%) in 17 of the 24 children (71%) in Phase I study.

Some of the key players developing drugs for the treatment of neurofibromatosis include Novartis AG, AstraZeneca Plc, Pfizer Inc., GlaxoSmithKline plc, Merck & Co. Inc, BioXcel Therapeutics, Inc.

Neurofibromatosis therapeutics pipeline analysis

  • By Phase
  • By Molecule Type
  • By Route of Administration
  • By Company