News

Orphan Drugs Are Neglected No More
The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.
BioSpace5d
Trump’s Tax Law Restores Orphan Drug Exemptions, Cuts Medicaid, Threatens 340B Program and Gives PBMs a Pass
President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs ...
Targeted Oncology4d
FDA Orphan Drug Status Granted for CAR T-Cell Therapy in Malignant Gliomas
FDA designates MB-101 CAR T-cell therapy for glioblastoma, highlighting its potential in treating aggressive brain tumors ...
Applied Clinical Trials Online2d
Arcus’ Quemliclustat Earns Orphan Drug Designation as Phase III Pancreatic Cancer Trial Advances
The FDA has granted orphan status to Arcus Biosciences’ CD73 inhibitor quemliclustat for metastatic pancreatic cancer, as the ...
Zacks.com on MSN5d
MBIO Soars on Orphan Drug Nod for Investigational Brain Cancer Therapy
Mustang Bio surges 180% after its brain cancer therapy, MB-101, earns FDA Orphan Drug status for two rare brain tumor types.
Targeted Oncology5d
FDA Grants Orphan Drug Designation to Liposomal Gemcitabine in Biliary Tract Cancer
FDA designates FF-10832 as an orphan drug for biliary tract cancer, promising enhanced treatment options for this aggressive ...
Arcus says Gilead-partnered cancer drug won FDA orphan status
Arcus Biosciences' cancer drug candidate, quemliclustat, earns FDA orphan drug status, ensuring incentives and exclusivity.
Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-101 (IL13Ra2-targeted CAR T-cells) to Treat Astrocytomas and Glioblastoma
In an ongoing Phase 1 trial published in Nature Medicine, MB-101 was well-tolerated and 50% of patients achieved stable ...
PharmExec3d
FDA Grants Orphan Drug Designation to Adcentrx Therapeutics’ ADRX-0405 STEAP1 ADC
ADRX-0405 is uniquely being tested during its 1a/b clinical trials. ADRX-0405 is labeled as a STEAP1 ADC (primarily ...
US Orphan Drug Market Size Trends FDA Designation By Indication Orphan Drug Sales Clinical Research Insight
US$ 190 Billion By 2030. Insight On FDA Designated Orphan Drugs In Clinical Trials: > 850 Orphan Drugs. Clinical Trials ...
CURE6d
FDA Grants Orphan Drug Status to MB-101 for Glioblastoma, Astrocytomas
The FDA granted orphan drug designation to a CAR T-cell therapy for recurrent glioblastoma and high-grade astrocytomas.
Pharmabiz2d
US FDA grants orphan drug designation to Klotho Neurosciences’ KLTO-202 to treat amyotrophic lateral sclerosis
US FDA grants orphan drug designation to Klotho Neurosciences’ KLTO-202 to treat amyotrophic lateral sclerosis: New York Saturday, July 12, 2025, 15:00 Hrs [IST] Klotho Neurosci ...