Akebia Therapeutics(AKBA)2025-03-13 11:27Licensing and Revenue - The company received an upfront payment of 100.0 million and tiered royalties ranging from 10% to 30% of annual net sales in the Medice Territory[100][103]. - The company is eligible to receive up to approximately 25.0 million and a profit-sharing arrangement where the company receives approximately 66% of the profits from Vafseo sales[112]. - The company recorded 55.0 million upon the achievement of certain annual net sales milestones related to Riona[125]. - The company recorded 9.1 million in royalties due to Panion relating to the sales of Auryxia in the U.S. and JT and Torii net sales of Riona in Japan during the year ended December 31, 2024[129]. - The company is eligible to receive royalty payments based on a mid-single digit percentage of net sales of ferric citrate in licensed territories under the Panion Amended License Agreement[127]. Supply Chain and Manufacturing - The company relies on single-source suppliers for the manufacture of Auryxia, with pricing structured on a per-kilogram basis for the drug substance and tiered pricing for the drug product[97]. - The company has engaged Cardinal Health, Inc. as the exclusive third-party logistics distribution agent for commercial sales of Auryxia and Vafseo[98]. - The company has established CMO relationships for the supply of Auryxia to ensure sufficient material for ongoing commercial sales and clinical trials[97]. - The company entered into a supply agreement with Medice to supply Vafseo drug product for commercial and developmental use in the Medice Territory[106]. - The company controls all clinical and commercial manufacturing of praliciguat, which will be conducted by a third-party manufacturer[133]. Patent and Intellectual Property - The company holds 19 issued patents covering vadadustat, with expected expiration dates between 2027 and 2039[142]. - The company has exclusive rights under a series of patents and patent applications to commercialize Auryxia worldwide, with expected expiration dates between 2026 and 2030[145]. - The company has filed applications for patent term extensions for U.S. Patent Nos. 8,299,298, 8,093,423, 7,767,851, and 8,338,642 covering Auryxia due to FDA regulatory review delays[161]. - The company expects to apply for patent term extensions on issued patents covering future product candidates upon FDA or foreign regulatory approval[164]. - The company’s patent term extensions could be critical in maintaining market exclusivity for its products, potentially extending patent life by up to five years[160]. - Patent term restoration under the Hatch-Waxman Act can extend a patent by up to five years for time lost during development and FDA review[262]. Competition and Market Landscape - Auryxia competes in the hyperphosphatemia market against FDA-approved phosphate binders, including Renagel® and Renvela®, as well as newly approved XPHOZAH®[174]. - In the iron deficiency anemia market, Auryxia faces competition from over-the-counter oral iron and various intravenous iron formulations, including Feraheme® and Injectafer®[175]. - Vafseo competes with several established products, including Epogen® and Aranesp®, as well as new therapies like roxadustat and daprodustat in various markets[167][171]. - The company is aware of the competitive landscape, including potential new anemia therapies in development by other companies[169]. - The company faces significant competition from larger pharmaceutical firms with greater resources and R&D capabilities[165]. - The company is currently facing competition from products that may be less expensive or more effective than Auryxia[177]. - The revenue decline for Auryxia post-LoE may be slower than typical due to its inclusion in the ESRD bundle under Medicare Part B[180]. - The timing and number of generic versions entering the market will significantly impact future Auryxia revenues[180]. Regulatory and Clinical Trial Processes - Regulatory processes for drug approvals require substantial time and financial resources, impacting the development timeline[181]. - The FDA requires a 30-day waiting period after IND submission before clinical trials can commence, allowing for safety reviews[188]. - Clinical trials must comply with GCP requirements, including obtaining informed consent from all participants[201]. - The company must disclose clinical trial information on a public registry, with potential penalties for non-compliance[196]. - The FDA requires sponsors to develop and submit a diversity action plan (DAP) for each Phase 3 clinical trial, aimed at increasing diverse patient enrollment[203]. - The FDA's application fee for NDA submission in fiscal year 2025 is approximately 4.3 million, with an annual program fee of about $0.4 million per eligible prescription product[216]. - The FDA aims to review most NDAs within ten months of filing, with priority review products targeted for six months[218]. - The FDA may grant fast track designation for products addressing unmet medical needs in serious or life-threatening conditions[224]. - The FDA's review process includes a preliminary review within 60 days of receipt to determine if the application is sufficiently complete[217]. - The FDA conducts inspections of manufacturing facilities and clinical sites to ensure compliance with cGMP and GCP requirements before NDA approval[219][221]. - The FDA may require a Risk Evaluation and Mitigation Strategy (REMS) as a condition of approval to minimize risks associated with a product[222]. - The FDA's draft guidance in June 2023 aims to modernize clinical trial design and conduct, facilitating the development of medical products[204]. - The FDA's Pediatric Research Equity Act mandates that applications must include data to assess safety and effectiveness in pediatric populations[211]. - The FDA may designate a product as a breakthrough therapy if it shows substantial improvement over existing therapies for serious conditions[228]. - The FDA may designate a product for priority review, reducing the review time from ten months to six months for drugs that treat serious conditions and provide significant safety or effectiveness improvements[229]. - Accelerated approval can be granted based on clinical trials showing effects on surrogate endpoints, allowing quicker access to therapies for serious or life-threatening illnesses[230]. - New legislation allows the FDA to require confirmatory clinical trials to be underway before granting accelerated approval and mandates progress reports every six months[231]. - The FDA prefers randomized controlled trials for accelerated approval to ensure robust efficacy and safety assessments, particularly for oncology therapeutics[232]. - The FDA's review process includes a benefit-risk assessment, considering the severity of the condition and the adequacy of existing therapies[236]. - A Complete Response Letter (CRL) indicates deficiencies in an application, requiring substantial additional testing or information for reconsideration[237]. - An approval letter authorizes commercial marketing but may impose specific conditions, including post-approval trials and risk management mechanisms[238]. - The Ensuring Innovation Act mandates the FDA to publish action packages summarizing decisions on new drug approvals within 30 days[239]. - Post-approval requirements may include additional studies and monitoring to assess drug safety and effectiveness[240]. Data Privacy and Compliance - The California Consumer Privacy Act (CCPA) and the California Privacy Rights Act (CPRA) impose new privacy obligations on companies handling personal data[264]. - At least eighteen states have enacted privacy laws similar to the CCPA and CPRA, impacting data processing obligations[265]. - Companies face increasing lawsuits related to privacy violations, particularly concerning the use of web trackers and pixels[266]. - Non-compliance with privacy and data security laws could result in significant penalties, including fines and reputational harm[269]. Clinical Trials in the EU - The Clinical Trials Regulation (EU) No 536/2014 aims to simplify and streamline the authorization and conduct of clinical trials in the EU, requiring only a single application for trials conducted in multiple Member States[272]. - The maximum timeframe for the evaluation of a Marketing Authorization Application (MAA) under the centralized procedure is 210 days, with accelerated evaluation possible in exceptional public health cases, reducing the timeframe to 150 days[281]. - A marketing authorization is valid for five years and can be renewed based on a reevaluation of the risk-benefit balance, with the possibility of unlimited validity upon renewal[287]. - The EMA's PRIME scheme facilitates the development of product candidates for unmet medical needs, providing benefits such as early regulatory dialogue and accelerated assessment[278]. - Non-clinical studies must comply with GLP principles, ensuring the safety of new chemical or biological substances[271]. - The centralized procedure is compulsory for specific products, including orphan medicinal products and advanced therapy products, ensuring a single marketing authorization valid across all EU member states[280]. - The Pediatric Investigation Plan (PIP) is required for marketing authorization in the EU, covering all subsets of the pediatric population unless waivers are granted[279]. - The MA holder must establish a pharmacovigilance system to monitor the safety of medicinal products, including expedited reporting of serious adverse reactions[288]. - The manufacturing process for medicinal products in the EU is highly regulated, requiring compliance with EU GMP standards[290]. - Non-compliance with EU regulations may result in severe penalties, including delays in trial authorization and marketing approvals, product withdrawals, and criminal penalties[293].