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- Biotech company Antisense Therapeutics (ANP) has been granted a rare paediatric disease designation from the U.S. Food and Drug Administration (FDA)
- The designation is for its Duchenne muscular dystrophy (DMD) treatment ATL1102
- Being granted FDA approval in this category gives the company an opportunity to be eligible for a rare paediatric disease priority review voucher
- The voucher can be used to speed up its FDA process by up to four to six months or it could be sold for some extra capital
- Antisense is currently up 17.4 per cent and is trading for 13.5 cents in early afternoon trade
Biotech company Antisense Therapeutics (ANP) has been granted a rare paediatric disease designation from the U.S. Food and Drug Administration (FDA) for ATL1102.
The Federal Food, Drug, and Cosmetic Act defines a rare paediatric disease as a serious or life-threatening disease which impacts individuals from birth to 18 years and affects less than 200,000 people in the U.S.
ATL1102 is for the treatment of Duchenne muscular dystrophy (DMD) which is a rare and fatal muscle-wasting disease. The disease inflames the muscle and leads to fibrosis and death of muscle tissue.
In early August, the company submitted the application for the DMD treatment to the FDA.
As part of advancing the U.S. regulatory strategy and the recent application for an Orphan Drug Designation (ODD), a request for a rare disease designation was also submitted.
The FDA approved the designation of ATL1102 as a rare pediatric disease following the submission of data from phase two clinical trial.
“The FDA has also determined that DMD meets the definition of a rare pediatric disease based on the information submitted by the company and reliance upon additional supportive information,” the company explained.
“Therefore, FDA has determined ATL1102 to be eligible for rare pediatric disease designation for the treatment of DMD,” it added.
Additionally, a company that receives approval for a product designated for a rare pediatric disease priority could qualify for a voucher that can be used to receive an expedited priority marketing authorisation review.
This priority review can cut the FDA process by as much as four to six months.
CEO, Mark Diamond, is very encouraged by the granting.
“[The granting] recognises a great need for new and improved therapies for boys with DMD,” he said.
“We look forward to future interactions with FDA as we refine our strategy for the development of ATL1102 in DMD in the U.S.,” he added.
The voucher may also be sold to provide extra capital for the company. Previously, these vouchers have been sold from between US$68 million (around A$95 million) and US$350 million (approx. A$490 million).
Antisense is currently up 17.4 per cent and is trading for 13.5 cents in early afternoon trade.
