Potential Treatment for Neurofibromatosis Type 1 Earns Fast Track Designation

According to a story from Acrofan, the biopharmaceutical company SpringWorks Therapeutics, Inc. has recently announced the that company’s experimental product candidate PD-0325901 has earned Fast Track designation from the US Food and Drug Administration (FDA). This experimental drug is being developed as a treatment for plexiform neurofibromas associated with neurofibromatosis type 1 (NF-1). SpringWorks is committed to the development of treatments for cancer and severe rare diseases.

About Neurofibromatosis Type 1 (NF-1)

Neurofibromatosis Type 1 (NF-1) is a genetic disorder that has impact on a number of the human body’s functional systems. NF-1 is caused by a mutation of a gene located on chromosome 17 which is responsible for the production of the protein neurofibromin. This mutation can be heritable, but about half of cases are the result of a spontaneous mutation. Symptoms of NF-1 include epilepsy, tumors affecting the nervous system and skin, spots on the skin, scoliosis and other skeletal deformations, learning and mental impairment, and vision disorders. People with the disorder also have greater risk of cardiovascular disease and cancer compared to unaffected people. The severity of symptoms can vary greatly; some people live fairly typical lives, whereas others are faced with serious quality of life challenges. There is no cure, and treatment generally involves managing serious symptoms and complications as they appear. To learn more about NF-1, click here.

About Plexiform Neurofibromas

Plexiform neurofibromas are peripheral nerve sheath tumors that can appear in severe cases of neurofibromatosis type 1. These tumors can inflict significant pain and disfigurement. These tumors are just one of several severe complications that can appear with this disorder and they have a major impact on quality of life.

About Fast Track Designation

Fast Track designation is a process that is designed to speed up the development cycle of therapies that have the potential to treat serious medical conditions and satisfy unmet medical needs. A drug can qualify for this designation if it is meeting an unmet need or demonstrates major advantages in effectiveness or safety over currently available treatments. A drug that earns this designation is subject to more frequent meetings and written communications with the FDA in relation to the drug’s development. The therapy may also be eligible for other benefits such as Priority Review and Accelerated Approval if other relevant criteria are met.

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