WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) has received confirmation from the U.S. Food and Drug Administration (FDA) that its resubmitted New Drug Application (NDA) for Translarna™ (ataluren) is under review. Translarna is a proposed treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD), a rare genetic disorder characterized by progressive muscle degeneration.
The FDA's acceptance of the NDA is a pivotal step for PTC (NASDAQ:PTC) Therapeutics, as articulated by CEO Matthew B. Klein, who expressed the company's commitment to advancing the drug's availability in the United States. The resubmission is anchored in evidence from global clinical trials and the STRIDE registry, which collectively suggest that Translarna may delay the progression of disability in nmDMD patients.
Significant benefits were observed in a placebo-controlled trial (Study 041) involving 359 participants. Improvements were noted in six-minute walk distance, NorthStar Ambulatory Assessment, and other functional tests after 72 weeks of treatment with Translarna. Additionally, data from the STRIDE registry indicated that the drug could postpone loss of ambulation by 3.5 years and delay respiratory decline.
Despite the positive findings, the FDA is not bound to follow standard Prescription Drug User Fee Act (PDUFA) timelines because this is a resubmission following a complete response letter issued to the initial NDA filed in 2016. Consequently, no action date has been provided for the review's conclusion.
Translarna, a protein restoration therapy, is designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. It is already licensed in various countries outside the U.S. and has been used by approximately 150 patients in clinical trials and extension studies.
PTC Therapeutics, a global biopharmaceutical company, continues to focus on developing treatments for rare disorders. The review of Translarna by the FDA represents a potentially significant advancement for the nmDMD community, which comprises about 13% of all Duchenne muscular dystrophy cases.
The information in this article is based on a press release statement from PTC Therapeutics.
In other recent news, PTC Therapeutics has faced opposition from the European Medicines Agency's Committee for Medicinal Products for Human Use on the renewal of Translarna's conditional marketing authorization, a treatment for Duchenne Muscular Dystrophy. This development coincides with the U.S. Food and Drug Administration's acceptance of the company's New Drug Application for sepiapterin, a potential treatment for phenylketonuria. PTC Therapeutics has also recently completed the sale of its gene therapy manufacturing business, receiving an upfront payment of $27.5 million.
Analyst firms including Raymond James, Baird, and TD Cowen have issued ratings on PTC Therapeutics, with Raymond James and TD Cowen maintaining a Market Perform and Hold rating respectively, while Baird reaffirms an Outperform rating. However, Goldman Sachs (NYSE:GS) maintains a Sell rating on the company.
In terms of financials, PTC Therapeutics announced Q2 2024 revenue of $187 million and revised its full-year revenue guidance to range from $700 million to $750 million. These facts underscore the recent developments in PTC Therapeutics' journey in the pharmaceutical industry.
InvestingPro Insights
PTC Therapeutics' (NASDAQ: PTCT) recent FDA development for Translarna aligns with the company's strong market performance. According to InvestingPro data, PTCT has shown impressive growth with a 126.79% price total return over the past year and is currently trading near its 52-week high at 97.93% of that peak.
The potential approval of Translarna could significantly impact PTC's financial outlook. While the company reported revenue of $900.45 million in the last twelve months, an InvestingPro Tip indicates that analysts anticipate a sales decline in the current year. This makes the FDA's review of Translarna crucial for future growth prospects.
Despite the positive market performance, it's worth noting that PTC Therapeutics is not currently profitable, with an adjusted operating income of -$192.45 million in the last twelve months. Another InvestingPro Tip suggests that analysts do not expect the company to be profitable this year, underscoring the importance of Translarna's potential market entry in the U.S.
For investors seeking a more comprehensive analysis, InvestingPro offers 11 additional tips for PTC Therapeutics, providing deeper insights into the company's financial health and market position.
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