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Fly News Breaks for August 23, 2017
PTCT
Aug 23, 2017 | 09:09 EDT
William Blair analyst Raju Prasad started PTC Therapeutics with a Market Perform rating and $17 fair value estimate, contending that it is not wise to view the FDA's demonstration of leniency for other rare disease therapies as raising the probability of success for ataluren, which he assigns only a 10% chance of success.
News For PTCT From the Last 2 Days
PTCT
Mar 28, 2024 | 08:34 EDT
PTC Therapeutics submitted the sepiapterin MAA to the European Medicines Agency, or EMA. The MAA submission is for the treatment of pediatric and adult patients with Phenylketonuria, including the full spectrum of disease subtypes. Phenylketonuria, or PKU, is a rare, inherited metabolic disease, which affects the brain. The sepiapterin MAA includes the results of the phase 3 APHENITY trial in which sepiapterin had a statistically significant and clinically meaningful reduction in blood phenylalanine levels in pediatric and adult PKU patients. PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024. Submissions in 2024 are planned in a number of additional key countries where PTC has existing rare disease commercial infrastructure including Brazil and Japan.