Orally-administered Translarna is for Duchenne patients to reduce the effect of nonsense mutations in the DMD gene that lead to a lack of functional dystrophin protein, leading to irreversible muscle damage.
Translarna has been approved in the European Union for Duchenne patients ages 5 and older, but it hasn’t been approved in the U.S. The Food and Drug Administration granted Translarna an independent advisory committee review in June this year after telling PTC in 2016 that its new drug application was not sufficiently complete for the FDA to review.
Since then, the FDA has assigned an action date of Oct. 24, 2017, for PTC’s new drug application for Translarna. PTC is preparing for the advisory committee meeting, which is tentatively scheduled for Sept. 28.
Emflaza, approved by the FDA in February 2017, is indicated for all forms of Duchenne, regardless of mutation. Emflaza is a corticosteroid that decreases inflammation and the activity of the immune system.
Stuart W. Peltz, PhD, the CEO at PTC Therapeutics, said in a press release that PTC is dedicated “to working with patients and their families to bring Translarna to the U.S. We are also pleased with the successful early launch of Emflaza. Consistent with our mission, we are working to ensure broad access to Emflaza to all eligible Duchenne patients regardless of financial or insurance status.”
The positive second quarter data of PTC reflect the results of Translarna and Emflaza. The company observes that the results of Translarna reflect the continued uptake, sustainable pricing, and high (more than 90 percent) compliance to treatment. The company is studying market access regarding funding on a country-by-country basis.
“Since our founding nearly 20 years ago, it has been our mission to provide treatments to patients living with rare diseases who have limited treatment options,” Peltz said.
Research and development expenses also increased compared to the same period in 2016. The company attributes this mainly to the beginning of clinical activities and regulatory expenses.
Regarding its full-year 2017 activities, PTC anticipates increased sales of Translarna and Emflaza. The company also anticipates increased expenses in 2017. These expenses will primarily support the global commercial availability of Translarna, the commercial launch of Emflaza in the U.S., and the continued research and development of other product candidates.
Among key second quarter highlights, PTC announced the successful launch in the U.S of Emflaza, with over 1,200 DMD patients being treated in the 12 weeks after the drug’s launch. The company also established the EMFLAZACares Program, which aims to give access to Emflaza to all eligible patients regardless of financial or insurance status.
PTC has also developed a spinal muscular atrophy (SMA) program in a joint collaboration with Roche and the SMA Foundation. This program is expected to proceed into clinical trials in the second half of 2017.
Preliminary data from the first cohort of the SUNFISH trial (NCT02908685) was presented at the Cure SMA Conference, demonstrating a dose-dependent improvement of up to 400 percent in the RNA of survival motor neuron (SMN)2, a protein produced in insufficient levels in SMA, with no relevant toxicity.
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