Significant Prevalence of Neurofibromatosis Type 1 and R&D of New Drugs to Augment Growth of Neurofibromatosis Type-1 Market

 

Neurofibromatosis Type-1 Market

Neurofibromatosis type 1 is a genetic disorder that cause tumors to form on nerve tissue in the skin, brain, and other parts of the body. The condition is usually diagnosed in childhood. Bone deformities and small bumps on the iris of the eye are some of the symptoms of neurofibromatosis type 1. The condition is reported in many ethnic groups and affects males and females equally. Treatment of neurofibromatosis type-1 includes, surgery, laser or electrocautery treatment, radiation therapy, chemotherapy, or both, symptomatic, and supportive treatment.

Market Dynamics

Significant prevalence of neurofibromatosis type 1 is expected to propel growth of the neurofibromatosis type 1 market. For instance, as per Orphanet, a portal for rare diseases and orphan drugs, prevalence of neurofibromatosis type 1 is 1/3,000 live births. Moreover, approval and launch of new products is also expected to aid in growth of the market. For instance, in 2020, the U.S. Food and Drug Administration approved Koselugo (selumetinib) for use in patients with inoperable plexiform neurofibromas, a common manifestation in neurofibromatosis type 1.

R&D of new drugs is expected to offer lucrative growth opportunities for players in the neurofibromatosis type 1 market. For instance, in January 2021, researchers at Children's Hospital of Philadelphia, U.S., demonstrated that the drug, cabozantinib, reduces tumor volume and pain in patients with neurofibromatosis type 1.Similarly, in 2019, SpringWorks Therapeutics, Inc. dosed the first patient in the Phase 2b ReNeu clinical trial evaluating the efficacy and safety of mirdametinib, an oral, small molecule designed to inhibit MEK1 and MEK2, in children and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas.

Moreover, early diagnosis of neurofibromatosis type 1 and investment in R&D of the disease is also expected to aid in growth of the market. For instance, in 2019, The Children's Tumor Foundation, a neurofibromatosis research foundation, collaborated with DELopen, a DNA-encoded library technology platform, under which DELopen will provide researchers free access to several molecules for R&D in the treatment of neurofibromatosis under grants covered by Children's Tumor Foundation’s Drug Discovery Fund.

Competitive Analysis

Major players operating in neurofibromatosis type-1 market include, Merck & Co., Inc, AstraZeneca PLC, NFlection Therapeutics, Healx, and GL Pharm Tech Corporation.

Major players operating in neurofibromatosis type-1 market are focused on approval and launch of new products to expand their product portfolio. For instance, in July 2020, AstraZeneca received orphan drug designation for its selumetinib, co-developed and co-commercialized with MSD Inc., in Japan for the treatment of neurofibromatosis type 1.

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