Another step forward for Australian rare disease company

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Investors have welcomed positive news for Australian company Antisense Therapeutics (ASX:ANP) with the FDA moving its treatment for duchenne muscular dystrophy (DMD) one step closer to possible approval.

The US regulator has granted rare pediatric disease (RPD) designation for ATL1102 for the treatment of DMD.

DMD is a rare and fatal muscle-wasting disease where inflammation leads to fibrosis and death of tissue.

Investors welcomed the result with a 26 per cent increase in the company's share price.

The RPD designation was based on the submission from a phase two trial. It means Antisense Therapeutics will be able to apply for expedited regulatory review. The company could also sell any voucher it receives granting an expedited regulatory review. These vouchers can be worth several hundred million dollars.

CEO Mark Diamond said, “We are very encouraged by the granting of the rare pediatric disease designation to ATL1102 by the FDA, which recognizes a great need for new and improved therapies for boys with DMD. We look forward to future interactions with FDA as we refine our strategy for development of ATL1102 in DMD in the US.”