Pasithea Therapeutics receives FDA Rare Pediatric Disease Designation for PAS-004, a next-generation MEK inhibitor targeting Neurofibromatosis type-1 (NF1).
Written By: Pharmacally Medical News Desk
Pasithea Therapeutics a clinical-stage biotechnology company, has announced that its lead drug candidate PAS-004 has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Neurofibromatosis type-1 (NF1).
The designation is granted to therapies targeting serious or life-threatening conditions that primarily affect individuals from birth to 18 years old and impact fewer than 200,000 people in the United States. NF1, a rare genetic disorder, affects an estimated 115,000 individuals in the U.S.
PAS-004 is a next-generation macrocyclic MEK inhibitor currently under development. With this latest milestone, the therapy has now received three key FDA designations: Orphan Drug, Fast Track, and Rare Pediatric Disease.
The Rare Pediatric Disease Designation also opens the door for Pasithea to potentially receive a Priority Review Voucher (PRV) upon approval of PAS-004. These vouchers can significantly accelerate the FDA review process for another drug and may be sold or transferred. Recent PRV sales have ranged between $150 million and $205 million, highlighting their substantial market value.
“We are pleased to have received this designation, which reinforces the potential of PAS-004 to address a serious unmet medical need,” said CEO Dr. Tiago Reis Marques.
Pasithea is currently conducting a Phase 1/1b multicenter, open-label dose-escalation trial (NCT06961565) evaluating PAS-004 in adult patients with symptomatic, inoperable, or recurrent NF1-associated plexiform neurofibromas (PN).
Plexiform neurofibromas are complex tumors that grow along nerve pathways and can affect multiple branches. Occurring in approximately 30–50% of NF1 patients, these tumors can lead to severe complications, including organ compression and potential malignant transformation.
If successful, PAS-004 could represent a meaningful advancement in the treatment landscape for NF1, particularly for patients with limited therapeutic options.
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