Experimental Neurofibromatosis Type 1 Therapy Earns Fast Track Designation

According to a story from Global Genes, the drug developer SpringWorks Therapeutics announced that the US Food and Drug Administration (FDA) has granted the company Fast Track Designation for its investigational product candidate PD-0325901. This therapy is being developed as a treatment for patients aged two and older with plexiform neurofibromas associated with neurofibromatosis type I (NF-1). These tumors are known for their aggressive growth that can inflict severe pain and disfigurement.

About Neurofibromatosis Type I

Neurofibromatosis Type I (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 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 Fast Track Designation

The FDA’s Fast Track program is specifically intended to accelerate the development cycle for therapies that have demonstrated the potential to treat severe and life threatening diseases or conditions, particularly when said therapy has the potential to satisfy medical needs that are not being met by current treatments. The designation gives the recipient company access to more frequent collaboration and correspondence with the agency throughout the drug development and approval process.

About PD-0325901

PD-0325901 has also earned Orphan Drug designation from the FDA. The drug is an inhibitor of MEK1 and MEK2 proteins. These proteins play a key role in the MAPK pathway which is responsible for the regulation of cell survival and growth. This pathway could play a role in a number of rare tumors and cancers, meaning that PD-0325901 has the potential to play a role in treatment beyond just neurofibromatosis type I.


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