- Last week, PTC Therapeutics Inc's (NASDAQ:PTCT) Upstaza (PTC-AADC; AAV2-based DDC gene therapy) received a CHMP opinion for approval under exceptional circumstances, says Raymond James. It maintains Outperform rating on the stock.
- The CHMP recommended Upstaza for AADC deficiency (AADCD) patients aged 18 months and older with a severe phenotype.
- According to the analysts, with the exceptional circumstances designation, PTCT will be required to provide registry study data to characterize long-term safety/efficacy further (since unable to conduct a placebo-controlled trial with so few patients).
- Little color was provided on price point/patient numbers. The management did indicate that they have identified over 300 patients across the globe and anticipate ~$20 million - 40 million in Upstaza revenues in 2022
- Raymond James believes the AADCD opportunity is relatively modest but views the recommended approval of an AAV-based therapy (especially one directly administered into the brain) as a positive step for the broader gene therapy space.
- Price Action: PTCT shares are down 2.55% at $31.38 during the market session on the last check Tuesday.
