Regulation

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).

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.