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PTC Therapeutics sells FDA priority review voucher for $150M

Published 11/27/2024, 08:06 PM
PTCT
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WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT), a biopharmaceutical company, announced on Wednesday it has agreed to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million. The PRV was awarded to the company by the U.S. Food and Drug Administration (FDA) following the approval of KEBILIDI™ (eladocagene exuparvovec-tneq) on November 13, 2024, a treatment for AADC deficiency in children and adults.

The sale of the PRV is pending customary closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino (HSR) Antitrust Improvements Act. The PRV program is designed to incentivize the development of treatments for rare pediatric diseases by allowing faster review of a subsequent marketing application for a different product, or the voucher can be sold or transferred.

PTC (NASDAQ:PTC) Therapeutics focuses on discovering and developing medicines for rare disorders. With a mission to provide access to treatments for patients with limited options, the company leverages scientific and clinical expertise to bring therapies to market. This transaction is part of PTC's strategy to maximize value for stakeholders through global commercialization and a diversified pipeline.

The company's statements regarding the expected closing of the transaction and clearance under the HSR Act are forward-looking and subject to risks and uncertainties that could cause actual results to differ materially. These include negotiations with third-party payors, market conditions, regulatory changes, and the commercial potential of their products.

This news is based on a press release statement from PTC Therapeutics, Inc. The sale of the PRV underscores the company's ongoing efforts to capitalize on its regulatory achievements and support its broader mission in the rare disease treatment landscape.

In other recent news, PTC Therapeutics has reported an optimistic outlook for its 2024 revenue, now projecting an increase to between $750 million and $800 million. This promising forecast follows the company's strong third-quarter earnings, with total revenue reported at $197 million. A significant contribution to this financial performance came from the Duchenne muscular dystrophy (DMD) franchise, including Emflaza, which generated $124 million.

The company has submitted two New Drug Applications (NDAs) to the FDA and is preparing for global product launches, including those of sepiapterin and vatiquinone. The launch of sepiapterin is planned across 50 countries, with potential revenue exceeding $1 billion in the U.S. alone. The NDA submission for vatiquinone is scheduled for December 2024, targeting Friedreich ataxia (FA), with a potential launch in 2025.

PTC Therapeutics has also received positive feedback from key opinion leaders on vatiquinone's efficacy and safety. However, there is some uncertainty surrounding the approval timelines for Translarna, with potential risk in Europe depending on the European Commission's decision. Despite these developments, PTC Therapeutics maintains a solid financial foundation, with cash reserves exceeding $1 billion, supporting future product launches and business development opportunities.

InvestingPro Insights

PTC Therapeutics' decision to sell its Rare Pediatric Disease Priority Review Voucher for $150 million aligns with the company's financial position and market performance. According to InvestingPro data, PTC Therapeutics has a market capitalization of $3.55 billion, reflecting its significant presence in the biopharmaceutical sector.

The company's revenue growth of 12.84% over the last twelve months as of Q3 2024 demonstrates its ability to expand its commercial footprint. However, an InvestingPro Tip indicates that analysts anticipate a sales decline in the current year, which may explain the company's strategic move to monetize its PRV.

Despite not being profitable over the last twelve months, PTC Therapeutics has shown strong market performance. The stock has delivered a remarkable 114.2% return over the past year and is trading near its 52-week high, with the price at 97.4% of its peak. This positive momentum is further supported by an InvestingPro Tip highlighting significant returns over the last week, month, and three months.

The sale of the PRV could provide a substantial cash infusion, potentially addressing the company's financial needs. An InvestingPro Tip notes that PTC's liquid assets exceed short-term obligations, which may be further bolstered by this transaction.

For investors seeking more comprehensive analysis, InvestingPro offers 12 additional tips for PTC Therapeutics, providing deeper insights into the company's financial health and market position.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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