Amgen seeks FDA approval for breakthrough orphan leukemia drug
- Blinatumomab is Amgen’s first BiTE antibodies, which are based on a novel immunotherapeutic approach whrein antibodies are modified to engage two different targets at the same time.
- Amgen has filed a biologic license application (BLA) seeking approval to market blinatumomab for treatment of Philadelphia-negative (Ph-) relapsed/refractory B-precursor forms of acute lymphoblastic leukemia (ALL).
- Blinatumomab has been granted breakthrough and orphan drug status by the FDA, signaling the importance of this new drug as a potential new treatment option for an unmet medical need.
Experts estimate that 6,000 cases of ALL will be diagnosed this year. Blinatumomab represents an important potential treatment option, because currently there is no widely accepted standard treatment for refractory or relapsed ALL. Median overall survival for patients with refractory disease is just three to five months.
The submission package includes phase II data demonstrating that 43% of patients met the primary endpoint -- a complete response, meaning no leukemia cells were detectable with microscopy.
- Pharma Times Amgen files ‘breakthrough’ leukaemia drug in the US