RNA biotech Dicerna has nabbed a priority review voucher as part of its new rare pediatric disease designation for leading drug nedosiran.
The RNAi drug is in the clinic for primary hyperoxaluria (PH) types 1, 2 and 3 and is Dicerna’s most advanced asset using the biotech’s so-called GalXC RNAi tech platform.
It’s set up to stop the lactate dehydrogenase (LDH) enzyme, an enzyme that catalyzes the final step in a common pathway resulting in oxalate overproduction in patients with PH1, PH2 and PH3.
The condition it treats is very rare and is linked to life-threatening genetic disorders that start off by hitting the kidneys. The three known types of PH (PH1, PH2 and PH3) each result from a mutation in one of three different genes. These genetic mutations cause enzyme deficiencies that result in the overproduction of a substrate called oxalate.
Irregular production and buildup of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease requiring intensive dialysis.
Now, the FDA has given Dicerna a rare pediatric disease designation for the ultra-rare drug. Under this program, a biopharma that gains approval for a rare pediatric disease can grab a voucher that can be redeemed to receive a priority review of a new, separate marketing application for a different drug.
Recently, these have become hot property and sold off for $100 million-plus to another buyer, given the seller a quick cash grab and the buyer a potentially quicker approval.
We’ll have to wait and see whether the biotech decides to hold on to the voucher for its other programs, many of which are partnered with an array of biopharmas.
“The FDA’s rare pediatric disease designation for nedosiran for primary hyperoxaluria reflects the agency’s continued recognition that PH is a serious and potentially fatal condition, with disease consequences that frequently manifest in childhood but progress over time into adulthood,” said Ralf Rosskamp, M.D., chief medical officer at Dicerna.
“These patients and their caregivers cope with burdensome and inadequate disease management techniques that ultimately cannot cure their underlying disease or prevent its potential to result in kidney stones, nephrocalcinosis, growth failure and failure to thrive, systemic oxalosis and end-stage renal disease—serious manifestations that often affect children.
“Our development program for nedosiran for the treatment of PH1, PH2 and PH3 reflects our recognition of the unmet need among all patients with PH, regardless of age or PH type, and we are gratified by this new designation as we continue to work toward our goal of providing a treatment for patients with all known forms of PH.”