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Norgine's Groundbreaking Approval for PEDMARQSI in Switzerland

On March 26, 2026, Norgine B.V., a prominent European specialty pharmaceutical firm, announced that Swissmedic, Switzerland's authority for therapeutic products, officially authorized PEDMARQSI® (sodium thiosulfate solution for infusion). This marks an important milestone, as it becomes the first and only treatment approved to prevent cisplatin-induced ototoxicity (hearing loss) specifically in pediatric patients aged from one month to less than 18 years, who are undergoing treatment for localized, non-metastatic solid tumors.

Cisplatin is a key drug in pediatric cancer therapies. However, it is notorious for its severe side effects, particularly the risk of irreversible, bilateral hearing loss, which can start impacting children from their very first dose. This condition can profoundly affect a child’s ability to communicate, learn, and develop socially, leading to long-term repercussions on their overall quality of life.

In light of such significant unmet medical needs, the Swissmedic decision is particularly notable. Rüdiger Merkel, Norgine's General Manager for the DACH region, expressed the importance of this approval for children and families in Switzerland. Until now, there has been no approved pharmacological option for mitigating hearing loss associated with cisplatin treatment, and this milestone helps fill that critical gap.

The Swissmedic authorization was largely based on compelling data from two open-label, randomized Phase 3 trials: SIOPEL 6, which played a pivotal role in demonstrating the drug's efficacy, and the Clinical Oncology Group’s ACCL0431 study. Results showed that patients who received sodium thiosulfate alongside cisplatin experienced about a 50% reduction in the incidence of hearing loss compared to those treated with cisplatin alone, while also maintaining effective chemotherapy outcomes.

PEDMARQSI® was first granted a pediatric use marketing authorization (PUMA) by the European Medicines Agency (EMA) in May 2023, alongside a national marketing approval in the UK. These combined approvals demonstrate Norgine's adept navigation through complex European regulatory environments for specialized and rare medications.

Understanding PEDMARQSI®

PEDMARQSI® represents a novel formulation of anhydrous sodium thiosulfate, designed specifically for pediatric patients. It is unique in that it is the only preventative treatment aimed at addressing cisplatin-induced ototoxicity in children. The clinical trials that led to its approval evidenced that sodium thiosulfate could significantly lower the risk of hearing loss in patients undergoing cisplatin chemotherapy.

The trials monitored the safety and tolerability profiles of PEDMARQSI®, revealing that nausea and vomiting were the most commonly reported adverse events, while more severe reactions, including infection, anemia, and neutropenia, were categorized as grade 3–4 adverse events. However, these findings suggest that the use of sodium thiosulfate alongside chemotherapy provides a favorable risk-benefit balance, making it an essential option for protecting young patients’ hearing during critical cancer treatment periods.

The Role of Norgine

Norgine is a mid-sized pharmaceutical company based in Europe, employing around 1,500 staff and generating approximately $650 million in annual revenue. The company is devoted to innovation in developing medicines that address urgent and unmet medical requirements. By tackling issues ranging from common ailments to extreme diseases like childhood cancers, Norgine showcases its commitment to enhancing patient lives through innovative therapeutic solutions.

Their significant expertise in navigating complex development, commercialization, and regulatory processes, paired with strategic partnerships, empowers them to expand access to transformative treatments across regions including Europe, Australia, and New Zealand.

As pediatric patients and their families can now look forward to better protection against one of the alarming side effects of crucial cancer therapies, Norgine's efforts in bringing PEDMARQSI® to the market represent a significant step forward in the quest to improve treatment outcomes and quality of life for young cancer patients.

Conclusion

The Swissmedic approval of PEDMARQSI® is not only a testament to Norgine's dedication to healthcare innovation but also a beacon of hope for young cancer patients and their families affected by the harsh realities of cisplatin. This groundbreaking treatment is a vital and timely addition to pediatric oncology, offering protection against hearing loss and enriching the lives of those undergoing one of the most challenging journeys.