30 September 2020 - Priority review voucher may be utilised to expedite marketing authorisation review by FDA.

Antisense Therapeutics is pleased to advise that U.S. FDA has granted rare paediatric disease designation for ATL1102 for the treatment of Duchenne muscular dystrophy, a rare and fatal muscle wasting disease where inflammation in the muscle leads to fibrosis and death of muscle tissue.

As part of advancing US regulatory strategy and recent application to US FDA for an orphan drug designation, a request for a rare paediatric disease designation was submitted in conjunction with the orphan drug designation application. The FDA has granted the designation of ATL1102 as a drug for a rare paediatric disease following submission of data from Phase 2 clinical trial of ATL1102. The FDA has also determined that Duchenne muscular dystrophy meets the definition of a rare paediatric disease based on the information submitted by the company and reliance upon additional supportive information. Therefore, FDA has determined ATL1102 to be eligible for rare paediatric disease designation for treatment of Duchenne muscular dystrophy.

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