RARE Daily

Marinus Sells Rare Pediatric Disease Priority Review Voucher to Novo Nordisk for $110 Million

July 14, 2022

Marinus Pharmaceuticals, a biotech focused on therapeutics for seizure disorders, said it is selling its Rare Pediatric Disease Priority Review Voucher for $110 million to Danish biopharma Novo Nordisk.

Photo: Steven Pfanstiel, chief financial officer of Marinus

The company received the voucher in March 2022 under a U.S. Food and Drug Administration program intended to encourage the development of treatments for rare pediatric diseases. The PRV was awarded with the FDA approval of Ztalmy (ganaxolone) oral suspension CV for the treatment of seizures associated with CDKL5 deficiency disorder, a rare form of genetic epilepsy, in patients two years of age and older.

“Upon completion, the sale of the PRV will significantly strengthen Marinus’ financial position, which we believe extends our cash runway into the fourth quarter of 2023,” said Steven Pfanstiel, chief financial officer of Marinus. “Importantly, this non-dilutive funding will allow us to maintain momentum advancing our clinical pipeline, including the two ongoing phase 3 trials in status epilepticus and tuberous sclerosis complex, and focus on the commercial launch of Ztalmy.”

The potential of ganaxolone is also being studied in other rare seizure disorders, including in phase 3 trials in tuberous sclerosis complex and refractory status epilepticus. Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is being developed in IV and oral formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings.

FDA’s Rare Pediatric Disease Priority Review program is intended to encourage the development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. Under this program, a PRV is issued to the sponsor of a rare pediatric disease product application and entitles the holder to priority review of a single New Drug Application or Biologics License Application. The sponsor may choose to sell or transfer the voucher upon approval of the rare pediatric disease product application.

Author: Rare Daily Staff

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