In a recent press release, Atossa Therapeutics announced that their novel drug, (Z)-Endoxifen, received Rare Pediatric Disease (RPD) designation for patients with Duchenne muscular dystrophy (DMD).
“Unlike more recent therapeutic approaches, (Z)-Endoxifen does not target specific exon defects, thus potentially offering a broader and more accessible treatment approach for this patient population,” said Senior Vice President of Research Janet Rea. “RPD designation provides a regulatory framework and an enhanced level of interaction with the FDA [Food and Drug Administration] as we define the clinical development path in DMD.”
The FDA grants RPD designation to drugs designed to treat rare diseases that mainly impact individuals younger than 18 years of age. Companies that receive this designation may qualify for vouchers that expedite the drug review process. The goal of this program is to incentivize therapeutic companies to develop drugs that may otherwise not be profitable.
(Z)-Endoxifen is a selective estrogen receptor modulator, meaning that it regulates estrogen levels throughout the body. In some tissues, it blocks the receptors that interact with estrogen, while in others, it activates those very same receptors. Estrogen receptors have emerged as a potential therapeutic target for DMD due to their role in maintaining muscle health and function.
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In addition to DMD, Atossa Therapeutics is investigating (Z)-Endoxifen for breast cancer, particularly among patients with metastatic disease. “While oncology remains our core focus, this milestone highlights (Z)-Endoxifen’s potential as a platform therapy in both cancer and rare diseases,” explained Dr. Steven Quay, president and chief executive officer at Atossa.
Congress did not renew the RPD Priority Review Voucher Program in 2024. This means that currently, the FDA cannot grant vouchers to companies whose drug products received RPD designation after December 20, 2024.
However, the House of Representatives recently approved the Give Kids a Chance Act, which would extend the program until 2029. The bill has now been passed on to the Senate. If approved, Atossa may become eligible for a voucher, which they can choose to either utilize or sell to another company.
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