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Fly News Breaks for May 26, 2015
PTCT
May 26, 2015 | 07:55 EDT
After PTC announced that Germany determined that its Translarna drug offs "quantifiable additional benefit" to ambulatory-patients aged 5+ with nmDMD, Oppenheimer thinks the determination could lead the EU to make reimbursement payments for the drug above the $300,000 per year guided by the company. The firm keeps a $155 price target and Outperform rating on the shares.
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.