Information Provided By:
Fly News Breaks for September 20, 2016
SRPT, PTCT
Sep 20, 2016 | 05:56 EDT
Jefferies analyst Gena Wang believes yesterday's FDA approval of Sarepta Therapeutics' (SRPT) eteplirsen will have a limited impact on PTC Therapeutics (PTCT). While PTC's potential treatment for Duchenne muscular dystrophy ataluren is considered safe, the FDA approval of eteplirsen is mainly based on the marginal increase of dystrophin level and PTC does not have any dystrophin data from Phase 3, Wang tells investors in a research note. Eteplirsen's approval could pressure the FDA to reconsider the review progress of ataluren, but the final outcome would likely remain the same, the analyst contends. She keeps a Hold rating on PTC with a $7 price target. The stock closed yesterday up 21%, or $1.88, to $10.99.
News For PTCT;SRPT 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.