Oct. 27 at 2:55 PM
$PMVP I'm still learning about biotech, so I wanted to share my thoughts and get some feedback. Please let me know where I've gone wrong!
The main reason I brought up the Breakthrough Therapy Designation (BTD) is this:
Typically, the process is Phase 1 \rightarrow Phase 2 \rightarrow Phase 3 \rightarrow NDA submission. However, if a drug, like lezatapopt, has virtually no alternative treatments and its Phase 2 results are exceptional, the Phase 3 trial can be skipped.
Usually, orphan drugs (for rare diseases) can apply for NDA or BLA with just Phase 2 data. Since the patient population for this disease is extremely small, it can practically be treated as a rare disease.
The clinical data released on September 10th was good, but perhaps slightly underwhelming. The company seems to have judged that the FDA might hesitate to grant BTD, which is why they updated the additional data.
Consequently, in this latest data update:
