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.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

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.

“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.”

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.”

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

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.