Jul. 13 at 5:49 PM
$SRPT
CAPR’s drug ( CAP-1002 ) was rejected by the FDA for insufficient evidence, but two things indicate it could still be approved after HOPE-3 data.
First, in HOPE-2, patients who stopped treatment for 13 months saw their upper limb performance decline twice as fast. When CAP-1002 treatment resumed, the decline slowed again. This is a signal of treatment effectiveness and is unlikely to happen by coincidence.
Second, the drug was originally tested in post-heart attack patients (ALLSTAR trial, 142 enrolled), where it showed a clean safety profile.
For rare diseases, safe and likely effective therapies often get approved.
I believe Elevidys isn’t going obsolete though. CAP-1002 mainly slows DMD progression, while Elevidys can reverse some motor decline when used for young ambulatory children. But Elevidys doesn’t help with cardiac function, and CAP-1002 does.
For example, Elevidys may serve younger ambulant patients, while CAP-1002 could preserve function in later stages of DMD.
