This Treatment for Neurofibromatosis Type 1 Just Got Orphan Drug Designation

According to a story from Business Wire, selumetinib, a drug in development for the treatment for neurofibromatosis type 1, was recently granted Orphan Drug Designation by the Food and Drug Administration (FDA). The companies responsible for development, AstraZeneca and Merck, announced the motion on February 15th.

Neurofibromatosis is a group of three conditions that are characterized by the growth of tumors in the nervous system.

The disease is caused by genetic mutations. In neurofibromatosis type 1 (NF1), various systems of the body are affected, which can lead to significant debilitation. Symptoms include structural abnormalities affecting the skull and spine, general muscle weakness and reduction in size of muscles, distinctive cafe au lait spot on the skin, deural neurofibromas, Lisch nodules in the iris, epilepsy, and various mental disabilities or abnormalities. Some people who have milder symptoms may live lives that are relatively undefined by the condition. Treatment is focused on reducing the many problems that can be caused by the complications associated with the condition, but there is no cure for neurofibromatosis. To learn more about neurofibromatosis, click here.

The granting of Orphan Drug Designation of selumetinib should increase the pace of the drug’s development.

The designation is typically only reserved for drugs that show promise during initial testing and fulfill a medical need that is currently unmet by presently available therapies. The designation allows for an expedited review process and waives several fees that the development company would normally be expected to pay. It also gives the company exclusive rights to development as well as a seven-year period of market exclusivity if the drug is successful in acquiring approval. The designation was developed specifically to give incentives for companies to develop treatments for rare conditions that would not be very profitable under normal circumstances.

Selumetinib is slated to begin Phase II trials later in the year and works by inhibiting the growth of the tumors that are commonly seen in NF1. The treatment has also entered Phase III trials as a therapy for differentiated thyroid cancer.

Selumetinib has previously obtained Orphan Drug Designation for the treatment of late stage differentiated thyroid cancer in 2016. This treatment also has potential for other uses as well, and is currently under study for use in combination with other drugs.

Selumetinib represents a notable improvement in treatment options for people with NF1!

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