AFT Pharmaceuticals Secures FDA Tentative Approval for Scomara in Facial Angiofibromas, US Launch Delayed Until 2029
核心洞察
The US FDA (搜索) granted tentative approval to AFT Pharmaceuticals (搜索)' Scomara (搜索) (rapamycin 0.5% cream) for treating facial angiofibromas in tuberous sclerosis (搜索), a rare disease affecting 15,000–30,000 US patients.
US commercialization is blocked until March 22, 2029, due to orphan drug exclusivity held by a competing product granted in 2022.
AFT plans to leverage the FDA approval to pursue regulatory clearances in Canada, Australia, New Zealand, and Asian markets where US orphan exclusivity does not apply.
AFT Pharmaceuticals (搜索) (NZX: AFT; ASX: AFP) announced that the US Food and Drug Administration has issued a tentative approval for Scomara (搜索) (rapamycin 0.5% cream), a topical treatment for facial angiofibromas in tuberous sclerosis (搜索) (FA in TSC). The FDA has evaluated and accepted the quality, efficacy, and safety data, along with the product's labelling and claims. However, full commercial launch in the United States cannot proceed due to existing orphan drug exclusivity held by a competing product.
The orphan exclusivity, a regulatory incentive designed to encourage rare disease drug development, was granted to another manufacturer in 2022 and provides seven years of market protection. Under this framework, the FDA is barred from approving any other application for the same drug targeting the same rare disease indication. The exclusivity period expires on March 22, 2029, meaning Scomara (搜索)'s US market entry is delayed until at least that date.
Facial angiofibromas in tuberous sclerosis (搜索) affects an estimated 15,000 to 30,000 patients in the United States, representing a significant unmet need within the rare disease landscape.
International Pathway Unaffected by US Exclusivity
While the US orphan exclusivity blocks immediate commercialization in that market, AFT Pharmaceuticals (搜索) emphasized that the FDA's tentative approval serves as a valuable regulatory foundation for pursuing market authorizations in jurisdictions where the US exclusivity does not apply. These markets include Canada, Australia, and New Zealand — where AFT operates directly — as well as numerous markets across Asia.
Dr Hartley Atkinson, Managing Director of AFT Pharmaceuticals (搜索), stated: "We are pleased to achieve this tentative approval following FDA review of our regulatory application but are disappointed that the US launch will be delayed at least until 22 March 2029. However, the tentative approval is a catalyst for AFT to develop new and significant markets for the medicine outside the US which is positive."
Competitive Differentiation
AFT believes Scomara (搜索) holds meaningful competitive advantages over the existing orphan product in international markets. The medicine is administered only once daily and can be stored at ambient temperature. In contrast, the competing product requires twice-daily application and must be kept under refrigeration. These features may enhance patient convenience and adherence, as well as simplify supply chain logistics.
Financial and Commercial Structure
AFT Pharmaceuticals (搜索) has not incorporated any revenue from Scomara (搜索) into its financial forecasts, meaning the delayed US launch does not affect the company's FY27 outlook. Under the commercial arrangement, AFT receives a 65% share of worldwide earnings from the opportunity following recovery of development costs. The remaining 35% of profits accrues to the 35% owner of AFT Orphan Pharmaceuticals. Additionally, AFT separately receives a royalty for the use of its proprietary technology in the medicine, payable prior to any profit share calculations.