The Empire State Building, where Rocket Pharma has offices, will light up again for Rare Disease Day.

Rock­et Phar­ma to light up build­ings again for Rare Dis­ease Day as it awaits first FDA de­ci­sion

Rock­et Phar­ma is get­ting ready to light up na­tion­al land­marks on Thurs­day as part of its an­nu­al rare dis­ease aware­ness ef­fort, but this year the event is even more mean­ing­ful as Rock­et awaits an FDA de­ci­sion on its first drug can­di­date.

Rock­et’s gene ther­a­py can­di­date, Kres­la­di, to treat se­vere leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-I) is be­ing con­sid­ered un­der pri­or­i­ty re­view with a June 30 de­ci­sion date. Rock­et al­so plans to sub­mit an­oth­er bi­o­log­ics li­cense ap­pli­ca­tion (BLA) in the first half of the year for RP-L102 treat­ment for fan­coni ane­mia.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.