Cellectis obtains 'orphan' and 'rare' status in the USA

(CercleFinance.com) - Cellectis jumped 9% on the Paris Bourse on Friday, after announcing that the FDA had granted orphan drug and rare pediatric disease status to its experimental treatment for acute lymphoblastic leukemia (ALL).


ALL, which accounts for around 10% of leukemia cases in the USA, progresses rapidly and can be fatal within weeks or months if left untreated.

It is estimated that 6660 new cases of ALL are diagnosed each year in the USA, and 1560 die from the disease.

There is an urgent need to develop new therapies for ALL for patients who are not candidates for transplantation or who relapse after transplantation", emphasized Mark Frattini, Cellectis' Chief Medical Officer.

Cellectis is a genome-editing company that has developed UCART22, an allogeneic CAR T product candidate targeting CD22 currently being evaluated in Phase 1/2 clinical trials on a dose-escalation basis in patients with relapsed or refractory ALL.

Orphan drug status can help speed up and reduce the cost of developing, approving and marketing a therapeutic agent.

Drug status for a rare pediatric disease can lead to a 'priority review' at the time of the marketing authorization decision.

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