- Biotech company Antisense Therapeutics (ANP) has submitted its Duchenne muscular dystrophy (DMD) treatment to the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD)
- DMD leads to muscle wasting that gets more debilitating over time
- Orphan drug designation can be granted by the FDA to drugs that intended to treat rare diseases that affect less than 200,000 Americans
- In May, the company announced a positive second phase results with ATL1102, which confirmed the drug’s safety and tolerability
- Antisense expects to apply for Orphan Drug designation in the European Medicines Agency this quarter
- Antisense is up 1.28 per cent on the market today and is trading for 7.9 cents per share
Antisense Therapeutics (ANP) has submitted its Duchenne muscular dystrophy (DMD) treatment, ATL1102, to the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).
DMD is a genetic condition that impacts the muscles — it leads to muscle wasting and gets more debilitating over time.
In May, the company announced positive second phase results with ATL1102. The trial confirmed the drug’s safety and tolerability.
Managing Director and CEO Mark Diamond is pleased with the progress of ATL1102 to date.
“This is an important regulatory and commercial milestone in the further development of ATL1102 for DMD and represents another key step forward towards bringing this potentially transformative therapy to patients with a significant unmet need,” he said.
Orphan drug designation can be granted by the FDA to drugs that intended to treat rare diseases that affect less than 200,000 Americans.
The FDA provides incentives to help speed up the development of products for rare diseases, including tax credit towards clinical trials, the waiver of U.S. prescription drug filing fees and seven-year exclusivity for orphan products upon marketing authorisation.
Antisense is expecting to apply for Orphan Drug designation in the European Medicines Agency this quarter.
Antisense is up 1.28 per cent on the market today and is trading for 7.9 cents per share at 2:06 pm AEST