A majority of rare diseases are genetic in nature, with an estimated 50-75% of these conditions manifesting in childhood. The Rare Pediatric Disease designation was established to encourage and streamline…
Rare Pediatric Disease designation (RPDD) is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics in development for rare pediatric diseases. In the United States, “rare”…
The FDA created the Rare Pediatric Disease Designation and Priority Review Voucher Program to stimulate the development of therapies intended to treat, diagnose, or prevent rare pediatric diseases. These are…
In late September 2022, biotech company Oncoheroes Biosciences, Inc. ("Oncoheroes") shared via news release that its product dovitinib, which the company has an exclusive pediatric licensing agreement for from…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
In 2012, the FDA established its Rare Pediatric Disease designation initiative through the Food and Drug Administration Safety and Innovation Act. Rare Pediatric Disease designation is granted to drugs or…
EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia. As recently reported in the Business Insider, EDIT-301 has just been granted the…
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
Because there are many obstacles that stand in the way of developing drugs for rare diseases, the FDA grants a number of designations to incentivize companies to create these treatments.…
According to a November 3, 2021 news release from biopharmaceutical company OS Therapies, the company’s therapeutic candidate OST-HER2 (OST31-164 / Listeria monocytogenes) received Rare Pediatric Disease Designation from the FDA.…
In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…
The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…
In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
The clock is ticking. If you or someone you love is diagnosed with a rare disease like hypoplasminogenemia, days -- often even hours -- can mean the difference between life…
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