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PTC has been named a 2026 Gallup Exceptional Workplace Award winner. This is the company’s fifth time earning this important recognition, and it reflects the culture we have cultivated by enabling our people do their best work by leading with their Strengths.
PTC has been honored with Gallup’s 2025 Exceptional Workplace Award, marking the fourth time the Company has been recognized for fostering a strong workplace culture that empowers employees by harnessing their unique strengths. PTC leverages the Clifton Strengths assessment, providing every employee with the opportunity to discover their innate talents. Our comprehensive approach to strengths development has resulted in remarkable engagement, as evidenced by: - 90% of leaders participating in Strengths-focused learning experiences - Our Coaching and Mentoring Center of Excellence, which fosters individual growth by providing employees access to a dedicated community of experienced coaches and mentors - Strong leadership commitment to a strengths-based culture - Integration of strengths into communications, development, and processes “At PTC, we take immense pride in the recognition we’ve received from Gallup,” says Hege Sollie-Zetlmayer, Chief Human Resources Officer. “This reflects our shared commitment to fostering a strengths-based culture that drives us towards excellence and fulfillment.” This achievement is a testament to our collective dedication to nurturing a work environment that enables employees to reach their full potential.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities. "This positive opinion is great news for the PKU patient community in Europe for whom there remains a significant unmet medical need," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We look forward to initiating the European launch immediately following the adoption of this positive opinion by the European Commission. The data collected to date continue to support the potential for Sephience to provide a safe and effective treatment that not only lowers phenylalanine levels but also enables diet liberalization even for those with the most severe disease subtypes." Launch planning in Europe is well underway with priorities in Germany and other key European markets where named patient access will be immediately available.
PTC Therapeutics announced that the FDA has accepted its filing for the New Drug Application (NDA) of sepiapterin to treat pediatric and adult patients living with phenylketonuria (PKU). A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter. "The FDA filing acceptance for sepiapterin is a critical milestone toward bringing this potential important therapy to children and adults living with PKU in the United States," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The clinical trial data, including the evidence of enabling diet liberalization, support that sepiapterin can provide significant benefit to PKU patients, helping to meet the persistent, significant unmet need in this community."
PTC Therapeutics announced that the FDA has granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. Fast Track designation is awarded to promising therapies for diseases of high unmet need. "The granting of Fast Track designation to the PTC518 program further supports the potential of PTC518 to provide a disease-modifying therapy to Huntington's disease patients," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We look forward to working collaboratively with FDA to continue to advance the program as efficiently as possible." PTC518 was discovered using PTC's innovative splicing platform, following the successful discovery and development of Evrysdi® (risdiplam) for the treatment of spinal muscular atrophy.
PTC has announced the submission of the sepiapterin MAA to the European Medicines Agency. The MAA submission is for the treatment of pediatric and adult patients with PKU, including the full spectrum of disease subtypes. "This MAA submission is the first in a series of global submissions for sepiapterin as we move closer to bringing this therapy to children and adults with PKU worldwide," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The strength of the sepiapterin data generated in clinical trials substantiate how differentiated sepiapterin is from currently available therapies and support how sepiapterin could address the significant unmet medical need for PKU patients."
There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to our scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This enables the identification of small molecules, which are known as splicing modifiers, that are designed to strengthen the interaction between the cellular splicing machinery and the pre-mRNA to help make splicing more efficient. PTC pioneered the idea that splicing can be modulated utilizing small molecules, built on the team’s expertise in RNA biology. Through this technology came the discovery of a treatment for spinal muscular atrophy (SMA), as well as ongoing development for a potential treatment for Huntington’s disease (HD).
PTC is pleased to present a no-cost interactive panel discussion with cell and gene therapy policy experts. Cohosted by the Alliance for Regenerative Medicine, this virtual event will explore how policy leaders can work together to advance access to these innovative therapies in Europe.
PTC is celebrating our 23rd anniversary with a global tribute video dedicated to our amazing team – now more than a thousand people in 25 countries around the world! Since 1998, our innovative biopharmaceutical company has been a leader in the small molecule and gene therapy space when it comes to rare diseases. We’re also working to treat rare cancers and even COVID-19 by way of smart science.
Science Education Day is observed on March 14 each year – an opportunity to celebrate the teaching and learning of science. PTC is honored to partner with Students 2 Science to bring exciting science labs and career-focused curriculum to Passaic Academy of Science & Engineering (PASE). “About 80% of my students speak another first language besides English,” explains one PASE teacher. “Then science is a third language.” Read more about how science is shaping the vision of middle school students in Passaic, New Jersey in our newsroom.
