PNH Oral Treatment Receives Orphan Drug Designation

As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA has officially designated the oral compound, BCX9930, as an “orphan drug”, which lessens typical barriers to approval, making the process cheaper and faster. The drug was additionally granted the Fast Track Designation, which makes for a speedy route to availability.

Paroxysmal Nocturnal Hemoglobinuria 

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease known to cause extreme inflammation, sometimes fatally.  This is because this serious disease kills red blood cells and can cause them to rupture, creating abnormalities in platelets. Symptoms include dark urine, tiredness, a fast heart rate, chest pain, headaches, breathing difficulty, swallowing difficulty, sexual dysfunction, and more. Patients on average survive 10 years after diagnosis.

Becoming an Orphan Drug

The orphan drug designation is meant to aid rare disease research which often falls behind due to less funding, smaller samples to research, and less political will. By designating BCX9930 as an orphan drug, research on the medication will qualify for tax credits and a time of market exclusivity, as well as several waivers of fees in order to speed up development.
BioSpace quoted Dr. William Sheridan, chief medical officer of BioCryst, who spoke of the value BCX9930 would bring to those with PNH. As there is a significant unmet medical need, this drug could change lives. BioCryst is very excited to continue working with the FDA to advance this treatment as quickly as possible.

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