SOUTH PLAINFIELD, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT), a biopharmaceutical company, announced the submission of a Marketing Authorization Application (MAA) for sepiapterin to the European Medicines Agency (EMA) today. The application targets the treatment of both pediatric and adult patients suffering from Phenylketonuria (PKU), encompassing all disease subtypes.
The company's Chief Executive Officer, Matthew B. Klein, M.D., stated that this MAA represents the initial step in a series of global submissions aimed at making sepiapterin available to PKU patients worldwide. PTC (NASDAQ:PTC) Therapeutics anticipates submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) by the third quarter of 2024, with further submissions in other key countries, including Brazil and Japan, expected within the same year.
The MAA is supported by data from the phase 3 APHENITY trial, which showed a significant reduction in blood phenylalanine levels—a key marker of PKU—among patients treated with sepiapterin. The trial reported a mean decrease of 63% in phenylalanine levels across the treated population, with an even greater reduction observed in patients with classical PKU. Notably, 84% of subjects achieved phenylalanine control in line with treatment guidelines, with 22% reaching normalization.
Additionally, the ongoing APHENITY open-label extension study indicates the durability of these treatment effects. Moreover, a sub study on phenylalanine tolerance revealed that patients could liberalize their diets while maintaining phenylalanine control, which is considered a significant benefit of sepiapterin treatment.
Sepiapterin is a synthetic precursor to tetrahydrobiopterin, an essential cofactor in the metabolism of various metabolic products. It is designed to be more bioavailable than the externally administered synthetic form and could potentially cater to a broader range of PKU patients.
PKU is a rare genetic disorder that can lead to severe and irreversible neurological damage if not properly managed. It is caused by a defect in the gene responsible for breaking down phenylalanine, an amino acid found in proteins and most foods. Globally, there are an estimated 58,000 individuals living with PKU.
PTC Therapeutics focuses on discovering and developing medicines for patients with rare disorders. The company's strategy involves utilizing its scientific and clinical expertise along with its global commercial infrastructure to deliver therapies to patients with limited treatment options.
This news is based on a press release statement from PTC Therapeutics, Inc.
InvestingPro Insights
As PTC Therapeutics (NASDAQ: PTCT) advances its global regulatory strategy for sepiapterin, the company's financial and market performance offer insights into its current standing and future prospects. According to InvestingPro, the company's market capitalization stands at $2.14 billion, reflecting the market's valuation of the biopharmaceutical firm amid its latest developments.
InvestingPro Data indicates a notable revenue growth of 34.2% over the last twelve months as of Q4 2023, suggesting that the company is expanding its financial base. This is complemented by an impressive quarterly revenue growth of 83.41% in Q4 2023, which may signal strong market acceptance of its existing products and potential for its pipeline candidates like sepiapterin.
However, analysts following PTCT have raised some concerns, as reflected in the InvestingPro Tips. They anticipate a sales decline in the current year and do not expect the company to be profitable this year. PTCT has not been profitable over the last twelve months, a trend that investors will be keenly watching as the company seeks regulatory approvals for its treatments. Despite these challenges, PTCT has experienced a large price uptick over the last six months, indicating investor optimism or reaction to specific company or industry events.
For readers looking to delve deeper into PTCT's financial health and future outlook, there are additional InvestingPro Tips available. Readers can use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, which includes an extensive list of tips to guide investment decisions.
With the next earnings date set for April 30, 2024, investors and stakeholders will be closely monitoring PTC Therapeutics' performance metrics and strategic initiatives, as the company continues to navigate the complex landscape of rare disease treatments.
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