One of the Children’s Tumor Foundation’s partners, SpringWorks Therapeutics, just announced that the Food and Drug Administration (FDA) granted Fast Track designation for the MEK inhibitor PD-0325901, for NF1 patients over the age of 2 with inoperable plexiform neurofibromas. This will help expedite the development and review of this potential treatment for NF patients. SpringWorks expects to initiate a phase 2b clinical trial for pediatric and adult patients later this year.
Springworks’ press release is excerpted here:
SpringWorks Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PD-0325901, an investigational, oral, small molecule inhibitor of MEK1 and MEK2, for the treatment of patients ≥ 2 years of age with neurofibromatosis type 1-associated inoperable plexiform neurofibromas that are progressing or causing significant morbidity.
Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) is a rare genetic disorder characterized by mutations in the MAPK pathway, leading to the growth of peripheral nerve sheath tumors that cause significant pain, disfigurement and morbidity. NF1-PNs are most often diagnosed in the first two decades of life and are characterized by aggressive tumor growth, which is typically more rapid during childhood. There are currently no therapies approved for the treatment of NF1-PN.
“The Fast Track designation recognizes that plexiform neurofibromas have a substantial impact on the lives of patients, and that our MEK inhibitor has the potential to address the significant needs faced by this patient community who currently do not have an FDA-approved treatment,” said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. “We look forward to continuing to work closely with the FDA on our upcoming Phase 2b study, which will enroll pediatric and adult NF1 patients with plexiform neurofibromas.”
To read the rest of the press release, click here.
