Novartis’ Promacta Receives FDA’s Breakthrough Therapy Designation for Severe Aplastic Anemia (SAA)

Novartis Reports Results of Zolgensma (onasemnogene abeparvovec-xioi) in P-III STR1VE Study for Spinal Muscular Atrophy (SMA) Type 1


  • The US FDA grants BT designation to Promacta (eltrombopag) + standard immunosuppressive therapy (SIT) for patients with 1L severe aplastic anemia (SAA)
  • The BT designation is based on research conducted at NIH results demonstrated complete response @6mos. (52%) & ORR (85%)
  • Promacta (eltrombopag) is a TPO receptor agonist and has received FDA’s & EU’s approval for thrombocytopenia in pediatric patients >1yr with insufficient response to corticosteroids and immunoglobulins

Click here to read full press release/ article | Ref: Novartis | Image: Twitter