RARE Daily

FDA Grants Rare Pediatric Disease Designation to Knopp Biosciences for Treatment of Rare Epileptic Encephalopathy

July 15, 2020

The U.S. Food and Drug Administration has granted Knopp Biosciences Rare Pediatric Disease designation for its experimental therapeutic KB-3061 for the treatment of KCNQ2 epileptic encephalopathy.

Photo: Michael Bozik, CEO of Knopp Biosciences

KCNQ2 epileptic encephalopathy (KCNQ2-EE) is a rare genetic disease associated with seizures beginning in the first days of life and profound neurodevelopmental delay. The disease is caused by dominant-negative mutations in the KCNQ2 gene, which encodes for Kv7.2, a potassium channel that plays a critical role in early brain development.

KB-3061 is an activator of voltage-gated Kv7.2/7.3 potassium channels that, in cells transfected with gene variants that cause KCNQ2-EE, has demonstrated the ability to fully restore mutated Kv7.2 potassium channel function in in vitro experiments.

Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes KB-3061 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its Rare Pediatric Disease voucher to Vifor Pharma $111 million in February 2020.

“The Rare Pediatric Disease Designation highlights the significant unmet medical need facing patients with KCNQ2-EE,” said Michael Bozik, CEO of Knopp Biosciences.  

Editor’s note: This story was updated to correct the most recent sales of a rare pediatric disease voucher.

Author: Rare Daily Staff

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