RARE Daily

FDA Grants Rare Pediatric Disease Designation to Tellus Therapeutics’ TT-20 for Neonatal Brain Injury

October 21, 2020

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease and Orphan Drug designations to Tellus Therapeutics’ TT-20 for the prevention of diffuse white matter injury in preterm infants less than 32 weeks gestational age.

Diffuse white matter injury (DWMI) is the most prevalent form of preterm neonatal cerebral injury and is a strong predictor of poor neurologic outcomes in preterm neonates, leading to adverse neurodevelopmental events including cerebral palsy, intellectual disability, and neurosensory impairments. DWMI is characterized by diffuse, subtle changes in the white matter (myelin) microenvironment due to global hypomyelination. This disease process is driven by a reduction in the number of oligodendrocyte progenitor cells in the third trimester that can result from postnatal infections that induce systemic inflammation, including necrotizing entero-colitis or spontaneous intestinal perforations. There are currently no FDA-approved treatments for DWMI.

Tellus is developing novel small molecules derived from human maternal breast milk that have demonstrated the ability to induce regeneration of myelin-producing oligodendrocytes and reverse white matter injury in animal models of perinatal brain injury. The company is pursuing a First-in-Neonate regulatory path to evaluate safety and efficacy in newborns with brain injury for whom no treatments are available.

“Receipt of the Orphan Drug and RPD designations from the FDA is an important milestone for our TT-20 program and underscores the need to find treatments for DWMI, the predominate form of brain injury among neonates who survive preterm birth,” said Jason Kralic, CEO and co-founder of Tellus. “With no FDA-approved drugs for DWMI, we look forward to continued collaboration with the FDA as we further define a regulatory path for TT-20 in DWMI and other neonatal conditions.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes TT-20 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

The voucher program was due to expire at the end of September but Congress provided a short-term extension of the program. According to the current statutory sunset provisions, after December 11, 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by December 11, 2020. After December 11, 2022, FDA may not award any rare pediatric disease priority review vouchers.

Going forward, requests for rare pediatric disease designation submitted will be reviewed in a timely manner but the FDA’s previous 60-day response time no longer applies.


Photo: Jason Kralic, CEO and co-founder of Tellus

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