Pasithea Therapeutics receives FDA rare pediatric disease designation for PAS-004

Pasithea Therapeutics Corp. (NASDAQ: KTTA) announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation to PAS-004 for treatment of Neurofibromatosis type-1 (NF1).

The FDA grants RPDD for serious or life-threatening diseases that primarily affect individuals from birth to 18 years and affect fewer than 200,000 people in the U.S. Approximately 115,000 individuals in the U.S. live with NF1.

Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a new drug application or biologics license application for a rare pediatric disease may be eligible for a Priority Review Voucher. The voucher can be redeemed for priority review of a subsequent marketing application for a different product and may be sold or transferred to another sponsor. In the last 12 months, disclosed voucher sales have ranged from $150 million to $205 million.

PAS-004 has previously received FDA Orphan Drug Designation and Fast Track Designation. The company is conducting a Phase 1/1b multicenter, open-label, dose escalation trial of PAS-004 in adult participants with symptomatic, inoperable, incompletely resected, or recurrent NF1-associated plexiform neurofibromas.

Plexiform neurofibromas are tumors originating from the nerve sheath that grow through and around nerves. Thirty to fifty percent of patients with NF1 harbor these tumors, which can undergo malignant transformation and can be life-threatening when they compress vital organs.

Pasithea Therapeutics is a clinical-stage biotechnology company focused on developing PAS-004, described as a next-generation macrocyclic MEK inhibitor for treating RASopathies and MAPK pathway-driven tumors.

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