OPERATING REVIEW CONTINUED THE IMPORTANCE OF ORPHAN DRUG DESIGNATION The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have both granted Orphan Drug designation for trofinetide in Rett syndrome and Fragile X syndrome and for NNZ-2591 in each of Phelan-McDermid, Pitt Hopkins and Angelman syndromes. The FDA has also granted Orphan Drug designation for NNZ-2591 in PraderWilli syndrome. Orphan Drug designation is a special status that the regulators may grant to a drug to treat a rare disease or condition. Amongst other incentives, Orphan Drug designation qualifies the sponsor of the drug for exclusivity periods during which the regulators will not approve a generic competitor product. These marketing exclusivity periods are extremely valuable for the commercialisation of Orphan Drugs. They provide additional protection, along with patents, against generic competitors and potentially can continue to provide protection after patent expiry. The exclusivity periods after marketing authorisation of products approved for pediatric use are 7.5 years in the United States and 12 years in the EMA region. Japan, South Korea and Taiwan also have Orphan Drug programs. As well as the exclusivity periods, Orphan Drugs have many other commercial advantages compared with existing markets that have apparently attractive large sales in which established products and companies have to be displaced. The serious and urgent unmet need results in a more supportive regulatory and pricing environment and strong engagement from the patient community and leading physicians. Historical data indicates a higher probability of achieving regulatory approval and the potential for immediate access to known patients means that a large sales organisation is less important. In short, the Orphan Drug business model targets a leadership position in markets with urgent need, at an attractive price and with a higher probability of getting to market. The conditions that Neuren is aiming to treat are “rare diseases”, however they are not “ultra-rare”, and in each condition there are tens of thousands of potential patients around the world. COMMERCIAL EXCLUSIVITY In addition to the primary protection of the important exclusivity periods from Orphan Drug designation explained above, Neuren has additional commercial protection from issued patents extending as far as 2040 and pending patent applications extending as far as 2046. Since trofinetide and NNZ-2591 are new chemical entities, following the first marketing authorisation for each drug, the term of one patent may potentially be extended by up to 5 years in many countries, including the United States, Europe and Japan. OTHER REGULATORY INCENTIVES The FDA has granted Fast Track and Rare Pediatric Disease designations for NNZ-2591 in each of Phelan-McDermid, Pitt Hopkins and Angelman syndromes. Trofinetide also received Fast Track and Rare Pediatric Disease designations before its approval in the US. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. A drug that receives Fast Track designation is eligible for some or all of the following: – More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval – More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers – Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met – Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA The Rare Pediatric Disease Priority Review Voucher (PRV) program provides for the award of a PRV to drug developers that receive FDA approval for a drug for a designated rare pediatric disease. The voucher entitles the holder to priority review of a different drug or may be transferred or sold to another drug developer. The Rare Pediatric Disease PRV program has been reauthorized by the United States Congress to 30 September 2029. Recently in January and February 2026, two drug developers announced the sale of vouchers for US$200 million and US$205 million respectively. Neuren Pharmaceuticals Limited Annual Report 2025 5
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