Neurofibromatosis Therapeutics Analysis, Clinical Trials and Developments
Neurofibromatosis (NF) Therapeutics currently exhibits a proliferating pipeline with 15+ therapeutic candidates.
Neurofibromatosis (NF) is a genetic disorder that causes tumors on nerve tissues. These are usually non-cancerous, but few can be developed into cancer. The disorder becomes prominent by the age of 10–15 years. The NF is of three types: NF1, NF2, and schwannomatosis. NF1 is caused due to the defect in chromosome 17, NF2 is a result of defect in chromosome 22, and schwannomatosis is a rare form of NF in which people develop multiple tumors consisting of schwann cells on cranial, spinal, and peripheral nerves.
According to the research findings, majority of the pipeline drug candidates are being developed for oral administration. It has been observed that the oral route of medication is convenient, available in delayed or rapid release formulation, less risk of systemic infections, and inexpensive in nature, and also provides improved patient’s compliance. Administration of therapeutics for NF through the oral route has shown promising results in clinical studies. Moreover, many of the drug candidates are mitogen-activated protein kinase (MEK) inhibitors, which inhibit the growth of tumor cells.
Though there are no marketed products for the disease, there are many drugs in the pipeline. For instance, selumetinib is a MEK inhibitor developed by Array BioPharma, which is under phase 2 clinical trial. Due to many such drug candidates in clinical phases, there is a strong anticipation that the portfolio for NF is going to expand widely in the upcoming years. Moreover, BXCL101 manufactured by BioXcel Therapeutics Inc. received the Orphan Drug Designation from the U.S. Food and Drug Administration (USFDA) for curing NF2.
Some of the key players involved in the development of NF are Pfizer Inc., Novartis International AG, AstraZeneca PLC, Merck & Co. Inc., GlaxoSmithKline plc, and BioXcel Therapeutics Inc.