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?