Approvals

FDA approves Incyte’s Pemazyre as first targeted treatment for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma

Incyte announced that the U.S. Food and Drug Administration (FDA) has approved Pemazyre™ (pemigatinib), a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

Seattle Genetics gets FDA nod for breast cancer drug Tukysa

Seattle Genetics has secured approval from the US Food and Drug Administration (FDA) for its Tukysa (tucatinib) to treat people with advanced unresectable or metastatic HER2-positive breast cancer.

FORMA Therapeutics receives FDA orphan drug designation for FT-4202 to treat sickle cell disease

FORMA Therapeutics, a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for FORMA’s lead investigational agent, FT-4202, currently in clinical development as a potentially disease-modifying treatment for sickle cell disease (SCD).

Alnylam receives fast track designation for Vutrisiran to treat polyneuropathy of hATTR amyloidosis

Alnylam Pharmaceuticals, the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to vutrisiran, an investigational therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Ascletis receives IND approval for its HIV drug ASC09F

Ascletis Pharma announces that it received IND approval from National Medical Products Administration (NMPA) for its human immunodeficiency virus (HIV) protease inhibitor ASC09F (ASC09/Ritonavir Fixed-Dose Combination).

AstraZeneca, Merck secure FDA nod for Koselugo to treat paediatric patients with NF1 genetic disorder

AstraZeneca and Merck have secured approval from the US Food and Drug Administration (FDA) for Koselugo (selumetinib) to treat paediatric patients aged two years and older with neurofibromatosis type 1 (NF1) who have symptomatic and inoperable plexiform neurofibromas (PN).

FDA approves Pfizer’s Braftovi plus cetuximab to treat BRAFV600E-mutant metastatic CRC

Pfizer has secured approval from the US Food and Drug Administration (FDA) for its Braftovi (encorafenib) plus cetuximab (Erbitux) to treat BRAFV600E-mutant metastatic colorectal cancer (CRC) after prior therapy.

IVERIC bio announces fast track designation from US FDA for Zimura to treat geographic atrophy secondary to dry age-related macular degeneration

IVERIC bio announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Zimura® (avacincaptad pegol), a novel complement C5 inhibitor, in development for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).

Bristol Myers Squibb, Acceleron get FDA nod for Reblozyl to treat anaemia with lower-risk MDS

Bristol Myers Squibb (BMS) and Acceleron Pharma have secured approval from the US Food and Drug Administration (FDA) for Reblozyl (luspatercept-aamt) to treat anaemia in adults with lower-risk myelodysplastic syndromes (MDS).