Evicenter
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RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionProduct descriptionProduct informationDrug/Drug, drug/disease interactions

Effects of RP-A501 on other drugs

2 evidence topics · 3 sources

Effect of RP-A501 on the pharmacokinetics or exposure of other drugs

No evidence found.

Effect of RP-A501 on the dosing of concomitant immunomodulatory agents

“Additionally, the protocol will specify a lower threshold for administering a C5 inhibitor (eculizumab) in response to impending complement activation.” (opens the source at this quote in a new tab)

“Given the recognized association between systemic AAV administration, complement activation, and thrombotic microangiopathy, a more reactive strategy was adopted for complement-mediated complications[15]. Accordingly, the protocol specified a lower predefined threshold for initiation of the C5 inhibitor eculizumab in the event of complement activation.” (opens the source at this quote in a new tab)

Effect of RP-A501 on laboratory values that guide concomitant therapy

“Elevation in transaminases was observed in all three low-dose patients and returned to baseline within the first one to two months post-treatment. There was also a transient and reversible decline in platelets observed in these three patients.” (opens the source at this quote in a new tab)