Regulation - Approvals

X4 Pharmaceuticals receives breakthrough therapy designation from FDA for Mavorixafor to treat WHIM Syndrome

X4 Pharmaceuticals, a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for mavorixafor (X4P-001) for the treatment of adult patients with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.

FDA approves Reblozyl for treatment of anaemia in beta thalassemia

The US Food and Drug Administration (FDA) has approved Reblozyl (luspatercept-aamt) for the treatment of anaemia in beta thalassemia in adults needing regular red blood cell (RBC) transfusions.

Sandoz secures FDA approval for pegfilgrastim biosimilar Ziextenzo

Novartis’ Sandoz unit has secured approval for Ziextenzo (pegfilgrastim-bmez), a biosimilar to Amgen’s Neulasta (pegfilgrastim) from the US Food and Drug Administration (FDA).

Sanofi’s Fluzone quadrivalent formulation gets FDA nod in older people

Sanofi’s Fluzone High-Dose Quadrivalent (Influenza Vaccine) has secured approval from the US Food and Drug Administration (FDA) for its use in adults, aged 65 and over.

FDA grants fast track status to Terns’s FXR agonist to treat NASH

Terns Pharmaceuticals has secured fast track designation from the US Food and Drug Administration (FDA) for its TERN-101 investigational farnesoid X receptor (FXR) agonist to treat non-alcoholic steatohepatitis (NASH).

EC approves Pfizer’s Bavencio plus axitinib to treat advanced RCC

Pfizer and Merck KGaA announced that the European Commission (EC) has approved Bavencio (avelumab) in combination with axitinib as a first-line treatment for adult patients with advanced renal cell carcinoma (RCC).

AVROBIO receives orphan-drug designation from FDA for AVR RD 02 to treat Gaucher disease

AVROBIO announced that the US Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease.

EC approves BMS melanoma drug Opdivo two and four-week flat dosing schedule

Bristol-Myers Squibb (BMS) has secured approval from the European Commission (EC) for melanoma drug Opdivo (nivolumab) two and four-week flat dosing schedule.

FDA grants expanded approval for GSK’s ovarian cancer drug Zejula

GlaxoSmithKline (GSK) has secured expanded approval from the US Food and Drug Administration (FDA) for its Zejula (niraparib) for the late-line treatment for women with recurrent ovarian cancer.