FDA decision on $SRPT
For FDA decision on $SRPT
ELEVIDYS (ELEV) DMD gene therapy
Currently ...
ELEV has conditional approval for 4-5 yr olds only
SPRT wants full approval & expanded label for all DMD pts
FDA is faced with challenge:
Hold a hard line on the 1' endpoint?
17 question NSAA test (rated as 0, 1, or 2) 📸
ELEV missed 1' endpoint 📸
Or give SRPT the benefit of the doubt?
DMD trials are very hard to run
1 time gene therapy with well founded MOA
ELEV was
Directionally favorable for all key endpoints 📸
&
Met stat sig across all age groups for key 2' endpoints
Time to rise
10-meter walk
Stride velocity
Ascend 4 steps
FDA decision does not have to be all or none
SRPT wants full approval for all DMD pts
But FDA could ...
Give full approval to only 4-5 yr old ambulatory pts
Give full approval to only 4-7 yr old amb pts
Give/retain only conditional approvals and require another study
Withdraw conditional approval/pull from market
+ more options
What do you think the FDA will do?
$SRPT - After looking at data further, considering FDA's Peter Marks comments and action last spring to get SRPT conditional approval, and listening to investor call, Amp plans to stay in SRPT with current positions (lowered last week before readout) and will likely increase positions if SRPT trades significantly lower in coming days.
I think they at least remain on the market with 4-5 yr olds and probably get full approval for 4-5 yr olds & at least conditional for 6-7 yr olds. The direct functional data from 2' endpoints is too significant for the FDA to not let at least 4-7 yr old families have the option, & Peter Marks appears to be willing to take some risks with DMD gene therapy approvals.
And let's not forget that SRPT has ~$1B revenue with their current DMD products even without Elevidys (currently trading at ~10X revenue, which is not a crazy multiple for a mid-cap biopharma).