|Pamplona, Spain November 6, 2023 |
Palobiofarma S.L. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s phosphodiesterase 10 (PDE-10) inhibitor, PBF-999, for the treatment of patients with Prader-Willi Syndrome (PWS). “Pediatric patients living with PWS suffer from hyperphagia, a significant unmet need with very limited treatment options,” said Julio Castro, PhD, Chief Executive Officer of Palobiofarma S.L. “Obtaining Rare Pediatric Disease Designation from the FDA is an outstanding achievement for a small Spanish Biotech company and supports our mission to provide PBF-999 as a potential new treatment option for patients suffering from PWS.”
About Rare Pediatric Disease Designation
Rare Pediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a New Drug Application (NDA) for PBF-999 for the treatment of PWS is approved by the FDA, Palobiofarma S.L. may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application. This program is intended to encourage the development of new drugs and biologics for the treatment of rare pediatric diseases.
About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a variable and complex genetic neurobehavioral disorder resulting from abnormality on the 15th chromosome. It occurs in males and females equally and in all races. Prevalence estimates range from 1:15,000 to 1:25,000 (approximately 400 000 to 530 000 affected people worldwide). The most common and challenging symptom among those diagnosed is hyperphagia, an intense, insatiable hunger characterized by a constant desire to eat. Individuals experiencing hyperphagia may engage in continuous eating, leading to life-threatening obesity. There is currently no pharmacological treatment for hyperphagia.
PBF-999 is a novel, potent and selective PDE-10 inhibitor discovered and patented by Palobiofarma. PBF-999 is in Phase 2 clinical development for the treatment of Prader-Willi Syndrome (PWS).
About Palobiofarma S.L.
Palobiofarma S.L., Inc. is a biopharmaceutical company committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics based on the modulation of the adenosinergic pathways. Palobiofarma exhibits one of the more robust and diversified pipeline of the Biotech sector worldwide, with six different compounds in clinical development targeting diseases of high unmet medical need and is financially supported by well known Spanish VCs like Inveready and Sodena. For more information, please visit www.palobiofarma.com.