US regulator to expedite review of rare disease candidate

30 January 2020

Dutch rare disease specialist ProQR Therapeutics (Nasdaq: PRQR) has been given Rare Pediatric Disease (RPD) designation for its candidate QR-421a.

The US Food and Drug Administration awards the status, which provides priority review with the aim of expediting the process, to certain promising treatments for rare pediatric diseases.

ProQR is developing QR-421a for the treatment of people with retinitis pigmentosa caused by certain genetic mutations.

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