Below ANP announcement dated 30 September 2020. It should help. Quote:
US FDA Grants Rare Pediatric Disease Designation to ATL1102 for the treatment of DMD• Potentially eligible for rare pediatric disease priority review voucher (PRV);• PRV may be utilised to expedite marketing authorization review by FDA; or• PRV may be sold to provide additional non-dilutive capitalAntisense Therapeutics Limited [ASX:ANP | US OTC:ATHJY] is pleased to advise that U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation for ATL1102 for the treatment of DMD, 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 (ODD), a request for a rare pediatric disease designation was submitted in conjunction with the ODD application. The FDA has granted the designation of ATL1102 as a drug for a rare pediatric disease following submission of data from Phase II clinical trial of ATL1102. 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. Therefore, FDA has determined ATL1102 to be eligible for rare pediatric disease designation for treatment of DMD.Further, under the FDA’s Rare Pediatric Disease Priority Review Voucher Program*, a company that receives an approval for a product designated for a rare pediatric disease may qualify for a voucher that can be redeemed to receive an expedited priority marketing authorization review. The award or non-award of a rare pediatric disease priority review voucher is made at the time of marketing approval, should the Company request such a voucher in its marketing application, and subject to it meeting the eligibility criteria for a PRV. Priority review can cut the FDA review process by as much as 4-6 months, shortening the time it takes for the drug to reach the market.The sponsor of a rare pediatric disease drug receiving a priority review voucher may transfer or sell the voucher without limitation, subject to applicable FDA requirements for filing and use. An intangible benefit of the voucher is the value created for a company if the faster review leads to an expedited approval and therefore provides a commercial advantage via earlier sales of a new drug on the market. In recent years, a secondary market for the vouchers has emerged allowing companies to use the sale of PRVs to recoup expenses undertaken for drug research and development and present them with additional source of non-dilutive capital to support further advancement of clinical development. Since 2009 when the first PRV was awarded the values for these vouchers have ranged between US$68 million and US$350 million.
ANP Price at posting:
13.5¢ Sentiment: Hold Disclosure: Held
