Neurofibromatosis (NF) is an autosomal-dominant genetic disorder characterized by neurofibromas of the nerves and skin, café-au-lait spots on the skin (spots that look like coffee with milk), and developmental anomalies of the bones, muscles, and viscera. Usually there are two distinct types of NF namely; NF type 1 and NF type 2, also known as NF1 and NF2, respectively. Recently, a third type, known as schwannomatosis has been identified.
NEUROFIBROMATOSIS THERAPEUTICS DRUG CANDIDATES UNDER DEVELOPMENT (2018)
NF type 1 and NF type 2 are caused by defect on chromosome 17 and chromosome 22 respectively. Clinical diagnosis of NF1 includes blood tests, and a medical examination of the body by a physician. The presence of bilateral vestibular schwannomas (also known as acoustic neuroma) is the confirmatory diagnosis for NF type 2. AstraZeneca plc is in the process of Phase II stage of development by the selumetinib an orally active, small molecule for the treatment of NF.
The neurofibromatosis therapeutics pipeline has been analyzed based on different phases of development. The pipeline analysis of drugs by phases includes information about product description, research and development activities, and other development activities. The strategic development section provides information regarding designations, collaborations, licensing, grants, funding, technology and others.
As of January 2018, the neurofibromatosis therapeutics pipeline comprises of 25 drug candidates in different stages of development.
Some of the key players developing drugs for the treatment of neurofibromatosis include Novartis AG, AstraZeneca Plc, Pfizer Inc., GlaxoSmithKline plc, Merck & Co. Inc, and BioXcel Therapeutics Inc.
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