Raymond James Says PTC Therapeutics' CHMP Backing Is 'Under Exceptional Circumstances' What's Next

Last week, PTC Therapeutics Inc's 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.

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