Regulation

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).

Acceleron receives FDA breakthrough therapy designation for sotatercept in pulmonary arterial hypertension

Acceleron Pharma announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH).

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.

Alnylam seeks FDA approval for lumasiran in primary hyperoxaluria type 1

Alnylam Pharmaceuticals has filed an application with the US Food and Drug Administration (FDA) seeking the latter’s approval for lumasiran for the treatment of primary hyperoxaluria type 1 (PH1).

Beam Therapeutics licenses SIRION Biotech’s LentiBOOST Technology for its CAR-T pipeline

SIRION Biotech announced that Beam Therapeutics licensed rights to use SIRION Biotech’s LentiBOOST for use in their CAR-T cell products.

Takeda gets EU approval for ALUNBRIG in ALK+ NSCLC

Takeda Pharmaceutical has secured expanded approval for ALUNBRIG (brigatinib) in the European Union (EU) to use the tyrosine kinase inhibitor for the treatment of a particular lung cancer population.

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).

Bristol Myers Squibb and bluebird bio submit BLA for Idecabtagene Vicleucel to FDA

Bristol Myers Squibb and bluebird bio submitted their Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for idecabtagene vicleucel (ide-cel; bb2121) for the treatment of adults with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.