Palobiofarma S.L., backed by Inveready, is pleased to announce that the FDA and the EMA have granted Orphan Drug Designation to our novel therapeutic candidate, PBF-999, for the treatment of Prader-Willi Syndrome (PWS). These designations represent a significant milestone in our commitment to addressing unmet medical needs and advancing breakthrough treatments for rare diseases.
Prader-Willi Syndrome is a complex genetic disorder that affects approximately 1 in 15,000 births globally, presenting challenges such as insatiable hunger, intellectual disabilities, and behavioral issues. Current treatment options are limited, making the need for innovative therapies paramount for patients and their families.
The Orphan Drug Designation indicates the acknowledgement by the Regulatory Authorities of USA and the EU that PBF-999 have the potential to address the specific needs of individuals suffering from Prader-Willi Syndrome. This designation offers several benefits, including scientific regulatory advice, reduced regulatory fees, and market exclusivity upon approval, fostering our ability to expedite the development of PBF-999 and bring this much-needed therapy to those affected by PWS.
"We are thrilled with the Authorities decision to grant Orphan Drug Designation to PBF-999 for the treatment of Prader-Willi Syndrome," stated Julio Castro, PhD, Chief Executive Officer of Palobiofarma S.L. "This designation underscores our commitment to advancing innovative therapies for rare diseases and brings us one step closer to providing hope and improved quality of life for individuals living with PWS."
Palobiofarma remains dedicated to the rigorous clinical development of PBF-999, working closely with regulatory authorities, medical community, and patient associations to progress towards potential approval and ultimately, to make a positive impact in the lives of those affected by Prader-Willi Syndrome.
About Palobiofarma S.L.
Palobiofarma S.L., is a biopharmaceutical company committed to discovering and developing first-in-class therapeutics based on the modulation of the adenosinergic pathways. Palobiofarma owns a robust and diversified pipeline, with six compounds in clinical development targeting diseases of high unmet medical need.
About PBF-99
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.
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