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BioWorld - Tuesday, February 3, 2026
Home » Topics » Regulatory » Orphan drug

Orphan drug
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Cancer

Researchers develop atlas of cell line models for high-risk pediatric cancers

April 3, 2023
By Tamra Sami
Researchers in Australia have developed new cancer cell line models for pediatric cancers with the lowest survival rates, with a focus on high-risk central nervous system tumors such as glioblastoma.
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Neurology/Psychiatric

Ractigen's therapeutic siRNA RAG-17 awarded US orphan drug designation for ALS

March 6, 2023
The FDA has awarded orphan drug designation...
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Neurology/Psychiatric

Asklepios' AB-1003 gene therapy for limb girdle muscular dystrophy granted EU orphan drug designation

Feb. 28, 2023
Asklepios Biopharmaceutical Inc.'s AB-1003 (LION-101) has been granted orphan drug designation...
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Conceptual image for brain cancer treatment
Cancer

Jubilant's PRMT5 inhibitor JBI-778 awarded US orphan drug designation for GBM

Feb. 14, 2023
Jubilant Therapeutics Inc.'s JBI-778 has been awarded orphan drug designation by the FDA for the treatment of glioblastoma multiforme (GBM).
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Cancer

Beatica's BEA-17 awarded orphan drug designation for glioblastoma

Feb. 1, 2023
The FDA has awarded orphan drug designation to Beactica Therapeutics AB's BEA-17 for the treatment of glioblastoma (GBM).
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Cancer cells
Cancer

Tango's PRMT5 inhibitors receive IND clearance, orphan drug designation

Jan. 26, 2023
The FDA has cleared Tango Therapeutics Inc.'s IND application for TNG-462, a next-generation methylthioadenosine-cooperative (MTA) inhibitor of protein arginine methyl transferase 5 (PRMT5) for the treatment of cancers with methylthioadenosine phosphorylase (MTAP) deletion.
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Gavel and pill blister packs

US FDA holding its position on orphan drug exclusivity

Jan. 23, 2023
By Mari Serebrov
As the FDA has in the past when a court issued an opinion it didn’t agree with, the U.S. agency is trying to limit the fallout from an appellate court ruling in Catalyst Pharmaceuticals Inc. vs. Becerra, which involved the breadth of orphan drug exclusivity, to that case alone.
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Cancer

Lantern's LP-284 awarded US orphan drug designation for mantle cell lymphoma

Jan. 9, 2023
Lantern Pharma Inc.'s LP-284 has been awarded orphan drug designation by the FDA for the treatment of mantle cell lymphoma (MCL). LP-284 is a novel small-molecule agent that preferentially damages DNA in cancer cells harboring mutations in DNA damage repair pathways.
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Concept art for adeno-associated viral-based gene therapy.
Hematologic

Belief's gene therapy candidate BBM-H803 for hemophilia A awarded US orphan drug designation

Dec. 23, 2022
The FDA has awarded orphan drug designation to Belief Biomed Inc.'s BBM-H803 injection for the treatment of hemophilia A.
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Real fluorescence microscopic view of human neuroblastoma cells
Cancer

AUM's multi-kinase inhibitor AUM-302 awarded US orphan drug designation for neuroblastoma

Dec. 1, 2022
The FDA has awarded orphan drug designation to AUM Biosciences Pte. Ltd.'s AUM-302 for the treatment of neuroblastoma.
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