We will be attending the Utah Medicaid meeting.
They are reviewing Exondys and Emflaza, in addition to developing criteria for FDA approved drugs approved through the AA pathway, which could potentially put future DMD drugs in jeopardy. We have seen positive decisions where we've actually sent DMD patients/caregivers to these meetings so they can better understand DMD.
We'll be addressing three agenda items that impact the DMD community are 4 and 5. Obviously, we don't want Utah to develop criteria that prevent access to Exondys and Emflaza. And, #5 is particularly important if they develop a policy that prevents access to FDA approved drugs that are approved through the accelerated approval program; not only will a negative policy impact current and future drugs for Duchenne, but will also impact other rare disease communities as they see drugs get approved by the FDA AA pathway (for example: rare, fatal cancers).
