Product information and disease description Place of product in therapy Approaches to treatment

Ongoing post-approval monitoring

6 evidence topics · 6 sources

FDA guidance on long-term follow-up after gene therapy: duration recommended for AAV vectors

“In general, our current recommendations for the duration of a LTFU protocol based on product type are as follows:” (opens the source at this quote in a new tab)
“Up to five years for AAV vectors.” (opens the source at this quote in a new tab)
“Additionally, a risk-based approach for determining the duration of a LTFU protocol may be considered for vectors capable of latency (e.g., Herpesvirus), long term expression without integration (e.g., AAV), or vectors carrying gene editing components.” (opens the source at this quote in a new tab)

Long-term follow-up after gene therapy: company statement on FDA guidance

“FDA guidance advises that patients treated with some types of gene therapy undergo follow-up observations for potential adverse events for as long as 15 years.” (opens the source at this quote in a new tab)
“Furthermore, if undesirable side effects caused by our product candidate are identified following regulatory approval of a product candidate, such as in long-term follow-up studies, several potentially significant negative consequences could result, including reputational harm and regulatory authorities suspending or withdrawing approvals of such product candidate, requiring additional warnings on the label or requiring that we change the way a product candidate is administered or that we conduct additional clinical trials.” (opens the source at this quote in a new tab)

Follow-up planned in the pivotal phase 2 trial: 5 years after administration

“The trial will include a total of 5 years of follow-up after administration of RP-A501.” (opens the source at this quote in a new tab)

Long-term follow-up of phase 1 participants: registry record states 2 to 5 additional years

“All patients are planned to be followed for 36 months after investigational product administration.” (opens the source at this quote in a new tab)
“After the end of the follow-up period, patients will enter a Long-Term Follow-Up (LTFU) study enabling follow-up for an additional 2 to 5 years post-IP administration.” (opens the source at this quote in a new tab)

Long-term follow-up of phase 1 participants: October 2026 presentation states up to 10 years

“Phase 1 LTFU: up to 10 years (ongoing)” (opens the source at this quote in a new tab)

EMA paediatric investigation plan: long-term safety and efficacy concerns for paediatric use

“Concerns on potential long term safety/efficacy issues in relation to paediatric use: Yes” (opens the source at this quote in a new tab)

Post-approval monitoring requirements for RP-A501 (registry, risk evaluation and mitigation strategy, or labeling)

Not applicable.