ProQR Therapeutics N.V. (NASDAQ: PRQR) shares jumped early on Thursday after the firm announced that it received Orphan Drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for its autosomal dominant retinitis pigmentosa (adRP) drug, QR-1123.
QR-1123 is an investigational antisense oligonucleotide designed to address the underlying cause of vision loss associated with adRP due to the P23H mutation in the rhodopsin (RHO) gene. In simpler terms, QR-1123 interacts with the messenger RNA to prevent the translation of a certain gene; in this case, it’s to prevent vision loss.
For some quick background: adRP is a severe and rare genetic disease that causes progressive problems in night vision during childhood, leading to visual field loss and frequently resulting in blindness in mid-adulthood.
The ODD provides a special status for investigational drugs being developed for rare diseases. The ODD program offers development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S. following market approval.
Daniel de Boer, CEO of ProQR, commented:
We are pleased to have orphan drug designation for our QR-1123 program targeting autosomal dominant retinitis pigmentosa, or adRP. It highlights the unmet need for patients with this progressive disease causing blindness. Our goal is to develop and actively advance a pipeline of programs that can treat inherited retinal diseases like adRP in a targeted manner.
Shares of ProQR closed Wednesday at $7.69, in a 52-week range of $5.49 to $20.00. The consensus price target is $28.55. Following the announcement, the stock was up about 9% at $8.40 in early trading indications Thursday.