Rocket Receives FDA Approval for Genetic Immunodeficiency Treatment

By Connor Hart

Rocket Pharmaceuticals said the Food and Drug Administration has granted accelerated approval for its therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I, or LAD-I.

Severe LAD-I is a rare and life-threatening pediatric genetic immunodeficiency characterized by recurrent infections and high early-childhood mortality without treatment, the biotechnology company said Friday.

Shares of Rocket climbed 6.6%, to $5.00, in premarket trading.

Chief Executive Gaurav Shah said the approval represents an important milestone, and that the company will continue working to make the treatment available to eligible patients in the U.S.

With the approval of the treatment, Kresladi, the FDA granted Rocket a rare pediatric disease priority review voucher, a program designed to encourage development of therapies for rare pediatric diseases. Rocket said it will evaluate options to monetize the priority review voucher "in a manner designed to enhance financial flexibility and maximize shareholder value."

Write to Connor Hart at connor.hart@wsj.com

(END) Dow Jones Newswires

March 27, 2026 07:44 ET (11:44 GMT)