Rare Daily Staff

The U.S. Food and Drug Administration granted SpringWorks Therapeutics Fast Track designation for its experimental therapy for the treatment of patients 2 and older 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.

SpringWorks experimental drug, PD-0325901, is an oral small molecule inhibitor of MEK1 and MEK2. MEK proteins play a critical role in the MAPK pathway, a key signaling network that regulates cell growth and survival, and whose activity is highly relevant in multiple oncology and rare disease indications. SpringWorks is evaluating PD-0325901 as a monotherapy for the treatment of patients with NF1-PN and is also pursuing PD-0325901 in combination with other rational anti-cancer agents across a range of solid tumors.

“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.”

The FDA’s Fast Track program is designed to expedite the development and review of drugs with the potential to treat serious or life-threatening conditions, and with nonclinical or clinical data that demonstrate the potential to address unmet medical needs. Fast Track designation enables a company to have frequent communication with the FDA throughout the drug development and review process.

In November 2018, the FDA granted Orphan Drug designation for PD-0325901 for the treatment of neurofibromatosis type 1. SpringWorks expects to initiate a phase 2b single-arm, open-label study of PD-0325901 in pediatric and adult patients with NF1-PN in the third quarter of 2019.

Photo: Saqib Islam, CEO of SpringWorks Therapeutics

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