- Antisense Therapeutics’ (ANP) treatment for Duchenne Muscular Dystrophy (DND), ATL1102, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration
- The designation provides support and incentives — such as tax credits, wavering prescription drug filing fees and exclusive marketing authorisation — to companies which are developing drugs for the treatment of rare diseases
- A phase two study confirmed the drug’s safety and tolerability and demonstrated either an improvement or stabilisation in several measures of muscle strength and function
- Last month, Antisense also received Rare Paediatric Disease Designation for the drug, adding further commercial value to the clinical development of the drug
- Shares have been trading 2 per cent higher at 13 cents
Antisense Therapeutics’ (ANP) treatment for Duchenne Muscular Dystrophy, ATL1102, has been granted Orphan Drug Designation (ODD).
The designation from the U.S. Food and Drug Administration (FDA) provides incentives and support to companies developing drugs for the treatment of rare diseases, defined as those affecting fewer than 200,000 people in the United States.
These incentives include tax credits towards the cost of clinical trials, wavering U.S. prescription drug filing fees and exclusive marketing authorisation for seven years.
CEO Mark Diamond said the ODD represents a significant commercial milestone for Antisense.
“ODD provides a cost reduction and regulatory streamlining mechanism made available by the FDA for companies developing therapies for rare diseases along with key additional intellectual property protection for ATL1102 in DMD with seven years of marketing exclusivity from generic competition.”
“This FDA recognition spotlights the significant unmet need for patients living with DMD and ATL1102’s potential to markedly improve the quality of life of those boys so desperate for an effective treatment,” he said.
A phase two study confirmed the drug’s safety and tolerability and demonstrated either an improvement or stabilisation in several measures of muscle strength and function.
Last month, Antisense also received Rare Paediatric Disease Designation for the drug, adding further commercial value to the clinical development of the drug.
Shares have been trading 2 per cent higher at 13 cents at 11:30am AEDT.