Summary:
The ACAD Trofinetide PDUFA is expected this Sunday March 12, 2023
Will we see an early decision before the weekend?
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ACAD PDUFA
ACAD's asset Trofinetide has a PDUFA target action date of March 12, 2023, this Sunday. We are interested to see if the FDA will announce an early decision on Friday, or if we will have to wait until Monday. This approval decision is a BPIQ Suspected Mover for March, so it will be interesting to see how the market reacts to the approval decision.
Topline data from the Phase 3 Lavender trial of Trofinetide to treat Rett Syndrome was announced in December 2021 and the NDA was submitted in July 2022. The stock moved +18% upon report of the Ph3 data. The ACAD PDUFA was not extended, never received a CRL, and no AdComm was completed. If approved, Trofinetide would be the first approved treatment for Rett Syndrome.
Trofinetide is a novel synthetic analog of the amino-terminal tripeptide of IGF-1. This small molecule analog of IGF1 is meant to reduce neuroinflammation and support synaptic function to treat the core symptoms of Rett Syndrome.
There is no cure for Rett syndrome so current treatments include focusing on and managing symptoms including medications for breathing irregularities, motor difficulties, controlling seizures, as well as physical and speech therapy. All treatments are currently to improve quality of life.
Current competitors to Trofinetide include TSHA's TSHA-102 (Phase 1), AVXL's ANAVEX 2-73 (Phase 2, Phase 2/3, and NDA), and JAZZ's EPIDIOLEX (Phase 3; no recent updates though). TSHA-102 (a gene therapy) is constructed from a neuronal specific promoter, MeP426, coupled with the miniMECP2 transgene, MECP2, and miRARE all packaged in AAV9. Data from this trial is expected in the first half of 2023.
We are very excited for the possible approval of Trofinetide and look forward to the FDA's decision hopefully by Monday!
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