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Fly News Breaks for September 25, 2017
SRPT, PTCT
Sep 25, 2017 | 06:48 EDT
JPMorgan analyst Anupam Rama says he remains "somewhat cautious" on shares of PTC Therapeutics (PTCT) with the FDA's Peripheral and Central Nervous Systems Drugs Advisory Committee convening on September 28 to review the company's Translarna for the treatment of non-sense mutation Duchenne muscular dystrophy. Translarna has an FDA action date of October 24. The analyst cites the drug's "mixed" Phase 2 and Phase 3 datasets and prior refuse-to-file letter for his "somewhat cautious" stance. For Sarepta Therapeutics (SRPT), Rama sees a net positive read-through from a favorable panel, on the potential for an accelerated path forward for golodirsen in Exon 53 DMD. Further, a negative panel is likely to be viewed as Translarna-specific, playing out as a net neutral for Sarepta, the analyst tells investors in a research note. He does not see "major fundamental downside risk" for Sarepta shares going into PTC's panel. Rama keeps a Neutral rating on PTC and Overweight rating on Sarepta.
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.