At the end of March 2022, ALS News Today announced that IFB-088, an experimental treatment for amyotrophic lateral sclerosis (ALS), earned Orphan Drug designation from the FDA. The treatment was…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Rare conditions are defined as those affecting…
Biotechnology company Genmab A/S recently shared that its investigational therapy epcoritamab (DuoBody-CD3xCD20), being developed with AbbVie, was granted Orphan Drug designation from the FDA. The therapy is designed for those…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A “rare” condition is one affecting under…
On February 16, 2022, biotechnology company Hillstream BioPharma Inc. (“Hillstream”) shared that its drug candidate HSB-1216 received Orphan Drug designation from the U.S. Food and Drug Administration. This particular designation…
In a news release from early February 2022, clinical-stage biotechnology company Eureka Therapeutics, Inc. (“Eureka”) shared that two of its therapies – ET140203 and ECT204 – received Orphan Drug designation…
In a news release from February 16, 2022, clinical-stage biopharmaceutical company AN2 Therapeutics, Inc. ("AN2") shared that its therapy epetraborole was granted Orphan Drug designation for the treatment of non-tuberculous…
On February 16, 2022, Healio shared that CT103A, a therapy for adult patients with relapsed or refractory (R/R) multiple myeloma (MM), had received Orphan Drug designation from the United States…
In a news release from late January 2022, biopharmaceutical company CASI Pharmaceuticals, Inc. ("CASI") shared that the Company's partner, BioInvent International AB ("BioInvent") recently earned an Orphan Drug designation from…
In a February 8, 2022 news release, late-stage clinical company Synthetic Biologics, Inc. shared that the FDA granted Orphan Drug designation to the therapeutic candidate VCN-01. VCN-01 is a therapeutic…
Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. For the purpose of the designation, “rare” is considered any condition…
In a recent news release, enzyme engineering company Codexis, Inc. shared that its product CDX-6512 received both Orphan Drug and Rare Pediatric Disease designations from the FDA. Both designations were…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this status, “rare” conditions…
The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this designation, "rare" conditions are those affecting…
Orphan Drug designation is typically granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases; in the United States, these are conditions affecting under 200,000 Americans. Drug…
In a news release from January 4, 2022, biopharmaceutical company SpliSense shared that their drug candidate SPL84-23-1 for cystic fibrosis (CF) earned Orphan Drug designation in both the United States…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. These are defined as conditions affecting under 200,000 Americans.…
In early December 2021, specialty pharmaceutical company Aytu BioPharma, Inc. ("Aytu") announced that its therapy AR101 (enzastaurin) earned Orphan Drug designation from the FDA. According to the announcement, the therapy…
On December 6, 2021, biotechnology company Longeveron Inc. ("Longeveron") announced that its investigational therapeutic candidate Lomecel-B earned Orphan Drug designation from the FDA. The therapy is designed to treat patients…
On December 2, 2021, Ultimovacs ASA ("Ultimovacs") announced via news release that its universal cancer vaccine, UV1, received Orphan Drug designation from the FDA. UV1 is designed for those…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, "rare"…
According to a November 23 article from Muscular Dystrophy News, the FDA recently granted Orphan Drug designation to clinical-stage biotechnology company Regenxbio's experimental gene therapy candidate, RGX-202. The gene therapy,…
According to a late October 2021 news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the company's therapeutic candidate alvelestat received Orphan Drug designation from the FDA. The therapy…
Orphan Drug designation in the United States is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions, defined as those affecting under 200,000 Americans.…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
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