FDA grants Rare Pediatric Disease designation to Emcitate®
November 18, 2020
Stockholm, November 18, 2020. PledPharma AB (publ) (STO: PLED) announced today that the US Food and Drug Administration (FDA) has granted the company’s application for Rare Pediatric Disease designation (RPD) for its lead candidate Emcitate® in the treatment of MCT8 deficiency.
Emcitate was recently added to PledPharma’s portfolio through the acquisition of Rare Thyroid Therapeutics (RTT) and is under development for the treatment of MCT8 deficiency, a rare congenital disorder of thyroid hormone trafficking with detrimental natural history and no currently available therapy. Approximately 1 in 70,000 males is affected. A successful Phase IIb trial has been completed and a pivotal study is expected to start in Q4 2020. Emcitate holds Orphan Drug Designation (ODD) in both EU and the US.
“We are very happy that the US FDA through this designation confirms the severity of MCT8 deficiency and the need for effective therapies to treat this devastating condition” said Nicklas Westerholm, CEO of PledPharma, and continued “This is a valuable recognition and we look forward to continue working with the FDA as well as other regulatory agencies to bring Emcitate to the market as the first therapy for MCT8 deficiency.”
The FDA grants RPD to therapies for serious or life-threatening diseases primarily affecting children from birth to 18 years and affecting fewer than 200,000 people in the USA. Upon approval of a new drug application, sponsors holding a RPD can apply to receive a US Rare Pediatric Disease Priority Review Voucher (PRV), which can be used to obtain accelerated FDA review of a new drug application for any drug candidate, in any indication, shortening time to market in the US. The voucher may be sold or transferred to another sponsor. Up to the end of 2019 22 PRVs for rare pediatric diseases have been awarded by FDA, whereof 12 were sold to another drug sponsor with individual voucher sale prices ranging from $67 million to $350 million. Under the terms of PledPharma’s acquisition of RTT, 50% of the potential net proceeds from such a sale of a PRV related to Emcitate should be paid as an earnout to the sellers of RTT.
For more information about US Rare Pediatric Disease Designation and Voucher Program, see https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-pediatric-disease-priority-review-vouchers