Abeona Therapeutics Inc ABEO has completed a Type B meeting with the FDA regarding the pivotal trial for its ABO-102 gene therapy for Sanfilippo syndrome type A (MPS IIIA).
- ABO-102 is currently being evaluated in the single-arm Transpher A study in patients with MPS IIIA.
- Based on the Type B meeting with the FDA, the ongoing Transpher A study will serve as the pivotal study for ABO-102 and could potentially support a marketing application submission depending on the data set.
- In addition, Abeona also aligned with the FDA to define the primary endpoint for the study.
- Abeona intends to work closely with the FDA through the regenerative medicine advanced therapy (RMAT) mechanism to assemble the most robust pivotal data package possible for the registration of ABO-102.
- The Company is hopeful of having an evaluable data set in 2022.
- Recently, new data from gene therapy showed that brain volume in three young patients increased at 24 months.
- Price Action: ABEO shares are up 1.60% at $1.27 during the market session on the last check Wednesday.
- Related content: Benzinga's Full FDA Calendar.
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