Neurofibromatosis (NF) Therapeutics Pipeline to Witness Significant Growth due to Positive Clinical Results in the Coming Years | Top Key players are Pfizer, Novartis, AstraZeneca, Merck, GSK, BioXcel Therapeutics Inc.

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.

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The diagnosis of the disorder is usually in early childhood due to its symptoms such as café-au-lait brown spots on the skin, abnormalities in bone development, appearance of freckles in groin or under armpits, and various others. So, the diagnosis can include genetic testing. Moreover, other ways of diagnosis are physical examination under special lamps, X-rays, computerized tomography (CT) scan, or blood test for NF1; and hearing and audiometry tests for NF2. The cure of the disease is not well-known, but developments are going on for minimizing the symptoms.

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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.

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The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of NF. Comprehensive insights on the pipeline products have been provided, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences. In addition, the report highlights the winning strategies of companies involved in the NF therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in this report. Furthermore, the report contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with respect to NF.

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