#Amsterdam #Switzerland #Norgine #CISPLATIN #PEDMARQSI®

Norgine's Groundbreaking Approval for PEDMARQSI®

In a significant milestone for pediatric healthcare, Norgine B.V., a notable European specialty pharmaceutical firm, has secured marketing authorization from Swissmedic for its innovative treatment, PEDMARQSI®. This drug is specifically designed to prevent cisplatin-induced ototoxicity, a type of hearing loss that affects children undergoing chemotherapy.

The Importance of PEDMARQSI®

PEDMARQSI (sodium thiosulfate infusion) stands out as the first and only approved treatment in Switzerland aimed at counteracting hearing loss caused by cisplatin, an essential but potentially harmful chemotherapeutic agent used in the treatment of localized, non-metastatic solid tumors in children aged 1 month to under 18 years. Hearing loss resulting from chemotherapy can have long-lasting effects on children's speech development, education, and social skills, presenting a pressing need for effective preventive measures.

Clinical Evidence Supporting Approval

The authorization granted by Swissmedic is supported by robust clinical data from two pivotal Phase 3 studies, SIOPEL 6 and COG ACCL0431. Results indicated that children treated with both cisplatin and sodium thiosulfate exhibited approximately a 50% reduction in the incidence of hearing loss compared to those receiving cisplatin alone, with the efficacy of the chemotherapy remaining intact.

Rüdiger Merkel, General Manager of Norgine DACH, emphasized the importance of this approval for families and healthcare professionals in Switzerland, noting that until now, there were no approved pharmacological options to help prevent this severe complication associated with cisplatin treatment. The consequences of hearing loss can be profound and transformative, underscoring the urgency of making this treatment accessible.

Previous Approvals and Future Prospects

PEDMARQSI® was previously granted approval by the European Medicines Agency (EMA) for use in pediatric patients within the European Union and has also received national authorization in the UK. These milestones illustrate Norgine's capability in navigating diverse regulatory pathways across different European jurisdictions for specialized and rare medications.

What is PEDMARQSI®?

PEDMARQSI® is a novel formulation of anhydrous sodium thiosulfate that has been meticulously developed for preventing cisplatin-induced hearing loss. Its unique formulation caters to those patients who are among the most vulnerable, ensuring that they receive quality care designed for their specific circumstances.

Side Effects and Efficacy

The studies validating PEDMARQSI® have shown a favorable safety profile, with nausea and vomiting as the most commonly reported adverse effects, while severe adverse events included infections, anemia, and neutropenia. The significant reduction in ototoxicity risk highlights the potential of this treatment to transform pediatric oncology care.

About Norgine

Norgine operates as a medium-sized pharmaceutical company within the EU, employing around 1,500 professionals and generating annual revenues of approximately 650 million US dollars. The company’s mission focuses on delivering life-altering medications for various diseases, including common ailments and severe conditions such as childhood cancers. Their commitment to addressing unmet medical needs is clear as they strive to ensure that every medical breakthrough reaches patients who need it the most.

The recent approval of PEDMARQSI signifies not only a major advancement in pediatric oncology but also reinforces the importance of innovation in pharmaceutical development amidst pressing healthcare challenges. By overcoming barriers in the marketplace, Norgine is paving the way for better health outcomes for children facing life-threatening illnesses.