FDA awards breakthrough designation for Novartis’ Promacta

The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation for Novartis’ Promacta (eltrombopag) as first-line treatment for severe aplastic anaemia (SAA).

Promacta has been indicated for conjunctive use with standard immunosuppressive therapy.

The drug is already approved as a second-line therapy to treat refractory SAA, and for thrombocytopenia in adults and children with chronic immune (idiopathic) thrombocytopenia (ITP).

An analysis of research by the National Heart, Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH) showed that 52% of treatment-naïve subjects experienced complete response at six months with Promacta and concurrent standard immunosuppressive treatment.

Novartis Oncology Global Drug Development head Samit Hirawat said: “Promacta is a promising medicine that, if approved for first-line use in severe aplastic anaemia, may redefine the standard of care for patients with this rare and serious bone marrow condition.

“We will continue to work closely with the FDA to make Promacta available to patients with SAA who are new to treatment as soon as possible.”

The firm’s cyclin-dependent kinase inhibitor drug Kisqali (ribociclib) has also obtained the FDA breakthrough therapy designation for initial endocrine-based treatment of breast cancer.

Kisqali can be used in combination with tamoxifen or an aromatase inhibitor for pre or perimenopausal patients suffering from hormone-receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer.

The FDA’s decision was based on the positive findings during Phase III MONALEESA-7 trial that demonstrated significantly prolonged progression-free survival (PFS) with the combination therapy.