Akebia Therapeutics(US:AKBA)2026-02-26 12:35Manufacturing and Supply Agreements - Vafseo relies on STA Pharmaceutical and Patheon for the manufacture of drug substance and drug product for commercial use[93]. - Auryxia's drug substance is supplied by Siegfried Evionnaz SA, with pricing structured on a per-kilogram basis, while the drug product is supplied by Patheon on a tiered pricing basis[98]. - The company has established CMO relationships for Auryxia to ensure sufficient material for ongoing commercial sales and clinical trials[98]. - The company controls all clinical and commercial manufacturing of praliciguat, with royalties on net sales subject to reduction upon patent expiration or generic product launch[130]. Licensing and Royalties - The company received an upfront payment of $10.0 million under the Medice License Agreement for Vafseo, with potential commercial milestone payments up to $100.0 million[100][103]. - The Medice License Agreement includes tiered royalties ranging from 10% to 30% of annual net sales of Vafseo in the Medice Territory[103]. - The TPC Agreement allows for potential payments of up to $225.0 million based on development and sales milestones, with tiered royalties of 13% to 20% on annual net sales in the TPC Territory[109]. - The Vifor License Agreement included an upfront payment of $25.0 million and a profit-sharing arrangement where the company receives approximately 66% of the profits[112]. - The company will pay CSL Vifor tiered royalty payments ranging from a high single-digit percentage of net sales of Vafseo up to $450.0 million[114]. - The Averoa License Agreement allows for tiered royalties from mid-single digit to low double-digit percentages of annual net sales, with minimum royalty amounts in certain years[118]. - Under the Q32 Purchase Agreement, the company made an upfront payment of $7.0 million and will make additional payments totaling up to $94.5 million based on development milestones for AKB-097[133]. - The royalty payments for AKB-097 will range from low single digits to mid-teen percentages, expiring on a country-by-country basis after the last patent claim or ten years from the first commercial sale[133]. - The company recorded $5.7 million in license revenue from royalties on net sales of Riona in Japan during the year ended December 31, 2025[122]. - The company recorded $11.3 million in royalties due to Panion related to sales of Auryxia in the U.S. and Riona in Japan during the year ended December 31, 2025[126]. - The Cyclerion Agreement includes a $3.0 million upfront payment and potential milestone payments up to $197.5 million based on product development[129]. Patent and Intellectual Property - The company holds 19 issued patents for vadadustat, with expected expiration dates ranging from 2027 to 2039, plus any extensions or adjustments[139]. - Auryxia's patent portfolio includes 3 issued patents with expected expiration dates between 2026 and 2030[142]. - The company has filed patent term extension applications for several patents covering Vafseo due to delays caused by FDA regulatory review[157]. - The company expects to apply for patent term extensions on issued patents for future product candidates upon receiving regulatory approval[161]. - The expected expiration dates for patents related to ferric citrate in Japan are between 2026 and 2028, with no contested proceedings reported to date[143]. - The company has entered into settlement agreements with six ANDA filers, allowing them to market a generic version of Auryxia starting March 20, 2025, subject to FDA approval[145]. Competition and Market Landscape - The pharmaceutical and biotechnology industries are highly competitive, with increasing demand for better anemia management solutions driving competition[162]. - The company faces competition from both large pharmaceutical companies and smaller biotechnology firms, as well as academic and research institutions[163]. - Vafseo competes with several established drugs including Epogen® and Aranesp®, both marketed by Amgen, and Procrit® by Johnson & Johnson[165]. - In Europe, roxadustat is approved for treating anemia in CKD patients, posing competition to Vafseo[166]. - Auryxia competes in the hyperphosphatemia market against FDA-approved phosphate binders like Renagel® and Renvela®[171]. - Auryxia also faces competition in the iron deficiency anemia market from various oral and intravenous iron formulations[172]. - The company is developing AKB-097 for potential treatment of IgAN, LN, and C3G, amidst competition from other complement inhibitors[179]. - In November 2025, the FDA approved VOYXACT® for reducing proteinuria in adults with primary IgAN, indicating increasing competition in this space[180]. Regulatory Environment - The company is subject to extensive regulations regarding drug development and approval processes, requiring significant time and financial resources[184]. - The FDA requires a 30-day waiting period after the submission of each IND before clinical trials may begin[190]. - Sponsors must prepare and submit an NDA requesting marketing for one or more proposed indications to the FDA[191]. - Clinical trials are conducted in four phases, with Phase 3 trials being pivotal for regulatory approval[204]. - The FDA has issued eight notices of non-compliance as of December 31, 2025, signaling enforcement of clinical trial information submission[198]. - Sponsors are required to develop and submit a diversity action plan for each Phase 3 clinical trial as mandated by the Food and Drug Omnibus Reform Act[205]. - The FDA's final guidance on GCPs aims to modernize clinical trial design and conduct, facilitating the development of medical products[209]. - Progress reports detailing clinical trial results must be submitted annually within 60 days of the IND anniversary[211]. - The Right to Try Act allows certain patients to access investigational drugs that have completed Phase I trials without enrolling in clinical trials[202]. - Expanded access regulations improve access to investigational drugs for patients with serious or life-threatening conditions[199]. - The FDA may impose a clinical hold on trials if there are concerns about patient safety or protocol compliance[193]. - The FDA's application fee for NDA submission in fiscal year 2026 is approximately $4.7 million, with an annual program fee of $442,213 per eligible prescription product[218]. - The FDA aims to complete a preliminary review of all applications within 60 days of receipt, informing sponsors whether the application is sufficiently complete for substantive review[219]. - Most NDA applications are intended to be reviewed within ten months, while priority review products are targeted for six months[220]. - The FDA may extend the review process by three additional months to consider new information provided by the sponsor[221]. - Before approving an NDA, the FDA typically inspects manufacturing facilities to ensure compliance with cGMP requirements[222]. - The FDA may require sponsors to develop a Risk Evaluation and Mitigation Strategy (REMS) as a condition of approval to minimize risks associated with the product[225]. - The FDA can designate products for expedited review if they address unmet medical needs for serious or life-threatening conditions[227]. - The FDA may withdraw fast track designation if it believes the designation is no longer supported by emerging clinical trial data[228]. - Accelerated approval may be granted based on surrogate endpoints that predict clinical benefit, with post-marketing trials required as a condition of approval[233]. - New legislation allows the FDA to require confirmatory clinical trials to be underway before granting accelerated approval and mandates progress reports every six months[234]. - The FDA may approve a new drug based on one adequate and well-controlled clinical investigation if confirmatory evidence is provided, as indicated in recent guidance[237]. - A Complete Response Letter (CRL) outlines deficiencies in a submission and may require substantial additional testing or information for reconsideration[239]. - An approval letter authorizes commercial marketing of a product with specific prescribing information, which may include post-approval trials to assess safety[240]. - The FDA's action packages summarizing decisions to approve new drugs must be published within 30 days of approval[241]. - Post-approval requirements may include additional studies and monitoring programs to ensure ongoing safety and effectiveness of the drug[242]. Data Privacy and Compliance - Compliance with evolving federal and state data privacy laws, such as the California Consumer Privacy Act (CCPA), may increase regulatory risks for the company[268]. - At least eighteen states have enacted comprehensive privacy laws similar to the CCPA, which may impact the company's business activities and data handling practices[270]. International Regulatory Considerations - The approval process for marketing products outside the U.S. varies by country and may involve additional testing and administrative review periods[273]. - The EU applies harmonized regulatory rules for medicinal products, but pricing and reimbursement processes differ significantly across member states[274]. - The Clinical Trials Regulation (CTR) No 536/2014 became effective on January 31, 2022, simplifying the approval process for clinical trials in the EU[277]. - The centralized procedure for marketing authorization allows for a single authorization valid across all EU member states, with a maximum evaluation timeframe of 210 days[284]. - For products of major public health interest, accelerated evaluation may be granted, reducing the assessment period to 150 days[284]. - Marketing authorizations are valid for five years and can be renewed based on a reevaluation of the risk-benefit balance[288]. - The Pediatric Investigation Plan (PIP) is mandatory for marketing authorization, covering all pediatric population subsets unless waivers are granted[282]. - The PRIME scheme facilitates the development of innovative products for unmet medical needs, providing early regulatory dialogue and accelerated assessment[281]. - A marketing authorization may only be granted to sponsors established in the EU, with orphan-designated products receiving a 12-year market exclusivity[287]. - The decentralized procedure allows for marketing approval in multiple EU member states based on a single assessment by a reference member state[285]. - MA holders must comply with EU pharmacovigilance legislation, including establishing a pharmacovigilance system and reporting serious adverse reactions[289]. - New marketing authorization applications must include a risk management plan detailing measures to minimize associated risks[290].