U.S. Capitol dome, Congress, Anerican flag

As the fairness of orphan drug exclusivities is debated in Congress, the U.S. Food and Drug Administration granted Orphan Drug designations to Editas Medicine and Neurocrine Biosciences.

FDA sign

China-based Hutchmed failed to get the U.S. Food and Drug Administration’s approval for the company’s proposed drug for pancreatic cancer.

AstraZeneca

AstraZeneca’s Fasenra (benralizumab) hit a roadblock in attempting to expand the medicine’s indications when the U.S. Food and Drug Administration issued a Complete Response Letter for chronic rhinosinusitis with nasal polyps (CRSwNP). 

AstraZeneca

AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.

The U.S. Food and Drug Administration placed a clinical hold on Boston-based Aprea Therapeutics’ lymphoid malignancy trial after the regulatory agency expressed concerns regarding the safety and efficacy data from a previous myelodysplastic syndromes (MDS) study.

Jazz Pharmaceuticals

Jazz Pharmaceuticals won U.S. regulatory approval for a new leukemia drug aimed at patients who have developed hypersensitivity to E. coli-derived asparaginase, an enzyme that is a component of the chemotherapy regimen used to treat acute lymphoblastic leukemia (ALL).

The Oncologic Drugs Advisory Committee (ODAC), as part of the U.S. Food and Drug Administration, voted 13-4 that a regulatory decision on Incyte’s PD-1 inhibitor retifanlimab for the treatment of advanced or metastatic squamous cell carcinoma of the anal canal (SCAC) should be deferred until further data are available from the clinical trial POD1UM-303.

The U.S. Food and Drug Administration rejected Orphazyme’s key drug candidate for Niemann-Pick disease type C (NPC), causing the Denmark-based biopharmaceutical company’s “meme stock” to decline in trading. 

Bold Therapeutics, a clinical-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration granted BOLD-100 an Orphan Drug Designation (ODD) in the treatment of gastric (stomach) cancer.

NASH

France-based Genfit launched NASHnext, a novel, non-invasive diagnostic test for nonalcoholic steatohepatitis (NASH) powered by the company’s proprietary diagnostic technology, NIS4. The test is offered in the United States through Labcorp.