RE:RE:RE:RE:RE:RE:ONC/Y and the FDAHI WestCoast,
This may be ONCY's achilles heel actually... Pela is to be used with the current standard of care, Paclitaxel, which has the following commonly reported side effects.
Bleeding, blistering, burning, coldness, discoloration of the skin, feeling of pressure,
hives, infection, inflammation, itching, lumps, numbness, pain, rash, redness, scarring, soreness, stinging, swelling, tenderness, tingling, ulceration, or warmth at the injection site, cracked lips,
diarrhea, difficulty with swallowing,
hair loss,
nausea or
vomiting, numbness, burning, or tingling in the hands or feet pain in the joints or muscles, especially in the arms or legs, thinning of the hair
The point being, that whilst the side effects of Pela are quite tolerable, we still need to consider that Paclitaxel is a cytotoxic chemotherapeutic... The goal should be to find a treatment that doesn't have a chemo like side effects profile. I think it would be very different if Pela showed the same efficacy alone as it does in combo with Pax.
Bear in mind also, that FDA requires data from two suitably powered randomised trials. IND-213 alone is not enough, and Bracelet-1 is not going to show adequate statistical powering. If symptoms weren't apparent for ten years if untreated, then accelerated approval makes sense, also for rare conditions, where it may not be possible to find enough patients.
For medicines approved decades ago that have a very poor safety profile, recall that if submitted and assessed by today's standatrds, even Aspirin would never get approved... Is it the use of Pax that is keeping would be suitors away ?