Shots: The FDA’s BT designation is based on P-III HAVEN 3 study assessing Hemlibra vs no prophylaxis in 152 patients in ratio (2:2:1) with hemophilia A + prior treated with […]readmore
Regulatory
Proteostasis’ PTI-428 Receives FDA’s Breakthrough Therapy Designation for Cystic Fibrosis
Shots: The FDA’s BT designation is based on P-II study results assessing PTI-428 (50 mg, qd) vs PBO in 24 CF with F508del mutation on background treatment with Orkambi for […]readmore
Shots: The FDA’s BT Designation is based on P-II study assessing SAGE-217 vs PBO in 89 patients with moderate to severe major depressive disorder (MDD) The P-II study results: met […]readmore
Shots: 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 […]readmore
Shots: The FDA’s BT designation is based on P-III MONALEESA-7 study assessing Kisqali + tamoxifen/ aromatase inhibitor + goserelin vs tamoxifen/aromatase inhibitor + goserelin in 672 pre/perimenopausal women with HR+/HER2- […]readmore