The U.S. Food and Drug Administration (FDA) has granted an expanded indication for Novartis’ Promacta (eltrombopag), approving it for use in the first-line treatment of severe aplastic anemia (SAA) in combination with standard immunosuppressive therapy (IST). This approval extends to both adult and pediatric patients aged two years and older.

Promacta’s New FDA Approval: Enhancing Treatment Options

Promacta, known as Revolade outside the United States, is an oral thrombopoietin receptor agonist (TPO-RA). Prior to this approval, Promacta was only approved for use in SAA patients who did not respond adequately to immunosuppressive therapy. The drug is also approved for the treatment of chronic immune thrombocytopenia (ITP) in patients who are refractory to other treatments and for managing thrombocytopenia in individuals with chronic hepatitis C virus (HCV) infection.

This recent FDA approval for first-line use was influenced by a pivotal study demonstrating significant efficacy. The study revealed that 44% of treatment-naive SAA patients achieved a complete response at six months when treated with Promacta in conjunction with standard IST. This rate represents a 27% increase compared to historical responses with standard IST alone. Furthermore, the combination treatment achieved an overall response rate of 79% at six months.

Expert Opinions on the Approval

Liz Barrett, CEO of Novartis Oncology, emphasized the importance of this new approval: “Severe aplastic anemia can be a fatal diagnosis if left untreated, and many patients fail to respond to current initial treatment options. Today’s US approval for Promacta is an important step forward for people living with this challenging disease and shows how Novartis continues to reimagine care in areas where few treatment options exist.”

Dr. Phillip Scheinberg, Head of the Division of Hematology at Hospital A Beneficência Portuguesa de São Paulo in Brazil, added: “Patients with SAA sometimes do not respond to the current treatment standard of IST. With this approval, physicians now have an option to add Promacta to the standard IST in a regimen that has demonstrated significant overall and complete response rates upfront in SAA and reduce the numbers of those who are unresponsive to initial therapy.”

Additional Developments

In a related development, Novartis has also received breakthrough therapy designation from the FDA for Promacta as a countermeasure for hematopoietic sub-syndrome of acute radiation syndrome (H-ARS), further highlighting the drug’s potential in critical areas.

The FDA’s latest approval represents a significant advancement in the treatment of severe aplastic anemia, offering hope for improved outcomes in patients who have limited options available to them.

  Eltrombopag, Liz Barrett, Novartis, Phillip Scheinberg, Promacta, Revolade, Severe aplastic anemia, US FDA, USA