Home China Releases 15th Five-Year Plan for Pharmaceutical Industry; Ultragenyx's $3.95M Gene Therapy FAYUVI Receives Full FDA Approval

China Releases 15th Five-Year Plan for Pharmaceutical Industry; Ultragenyx's $3.95M Gene Therapy FAYUVI Receives Full FDA Approval

Sep 18, 2026 19:49 CST Updated 19:49
WuXi XDC

End-to-End CDMO Service Provider for Biologics Conjugation Drugs

Huadong Medicine

Large Comprehensive Pharmaceutical Product Developer

Gan&Lee Pharmaceuticals

Biological Synthetic Human Insulin Pharmaceutical R&D Manufacturer

Hengrui Pharma

Innovative and High-Quality Pharmaceutical Developer

NOVAST

Developer and Manufacturer of High-End Prescription Drugs

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Key developments in the pharmaceutical industry today include: The Ministry of Industry and Information Technology (MIIT) and nine other departments jointly released the “15th Five-Year Plan for the Development of the Pharmaceutical Industry”; WuXi XDC licensed its WuXiTecan-2 linker-payload platform to Ona Therapeutics; Huadong Medicine entered into an exclusive collaboration with Meibo Biotech for ECM collagen MB007; GenScript joined Eli Lilly’s AI drug discovery platform, Lilly TuneLab; MindRank AI released Intera, a foundational model system for life sciences; Baili Tianheng invested $5 million in US-based Connexus; NOVAST launched its IPO on the ChiNext board, aiming to raise RMB 950 million; Gan&Lee Pharmaceuticals’ GLR1075 received clinical trial approval; Hengrui Pharma’s SHR-1139 received clinical trial approval; Cube Pharmaceutical’s clinical trial application for celecoxib and tramadol hydrochloride tablets was accepted; Everest Medicines’ Vydura received priority review for domestic production transfer in China; Eli Lilly’s RET inhibitor selpercatinib new formulation was approved in China; Insilico Medicine published an open-source AI longevity toolkit in a Cell cover article; Orbis reached a collaboration worth up to $1.4 billion with Novo Nordisk on oral macrocyclic drugs; Ultragenyx’s gene therapy FAYUVI received full FDA approval ($3.95 million per dose), among others.
◍Market FlashPolicy · Industry Trends
9/18“15th Five-Year” Plan for the Pharmaceutical Industry Released: Targets Include First-in-Class Innovative Drugs Accounting for One-Quarter of the Global Share and No Fewer Than Five Blockbuster Drugs
On September 18, the Ministry of Industry and Information Technology (MIIT) and nine other departments jointly issued the "15th Five-Year Plan for the Development of the Pharmaceutical Industry," proposing a series of indicative targets to be achieved by 2030: operating revenue of pharmaceutical industrial enterprises above designated size to reach no less than RMB 3.5 trillion; first-in-class (FIC) innovative drugs to account for no less than 25% of the global share; annual growth rate of the innovative drug industry scale to be no less than 20%; number of pharmaceutical industrial enterprises with annual operating revenue exceeding RMB 10 billion to reach 50; number of products with global annual sales exceeding USD 1 billion to be no less than five; and cumulative market launches of innovative medical devices to reach no less than 200. The plan prioritizes "enhancing original innovation capability" as its top key task, clearly focusing on major infectious diseases, chronic diseases, rare diseases, and pediatric medications. It strengthens efforts in four major directions: antibodies and recombinant proteins, next-generation conjugate drugs, cell and gene therapies, and novel vaccines, while dedicating a specific chapter to precision diagnosis and treatment as well as AI-driven drug discovery. The implementation of the plan coincides with the 2026 Zhangjiang Pharma Valley Conference and Shanghai International Biopharma Week. During the conference, it was disclosed that China has approved 59 innovative drugs for market launch this year (including 13 with new mechanisms and new targets). From January to August, there were 113 biomedical business development (BD) transactions nationwide, with a total value nearing USD 120 billion; Shanghai ranked first in the country with 37 transactions.
Interpretation:For the first time, the “15th Five-Year Plan” quantifies national targets such as “first-in-class (FIC) drugs accounting for one-quarter of the global share” and “no fewer than five blockbuster drugs,” sending a clear signal: China’s pharmaceutical innovation is shifting from “scale advantages in fast-follow strategies” to “competition in systems of original innovation.” By listing antibody-drug conjugates (ADCs), cell and gene therapies, precision diagnosis and treatment, and AI-driven drug discovery as core growth engines, the plan indicates that resources over the next five years will be concentrated on first-in-class targets and disruptive technologies. Furthermore, the language regarding “global expansion” has been upgraded from simple licensing-out to “co-development, co-commercialization, and international multi-center clinical trials,” thereby compelling pharmaceutical companies to strengthen their capabilities in completing the global regulatory approval and commercialization loop.
9/17WuXi XDC Licenses WuXiTecan-2 Payload-Linker Platform to Ona: Another Strategic Move in Global Expansion of ADC Technology
On September 17, WuXi XDC (2268.HK) announced a licensing collaboration with Ona Therapeutics regarding its independently developed WuXiTecan-2 payload-linker technology, granting the latter exclusive global development rights for an antibody-drug conjugate (ADC) candidate targeting a single specific antigen. As a global biotechnology company focused on first-in-class ADCs, Ona Therapeutics will leverage its proprietary first-in-class (FIC) targets in conjunction with the WuXiTecan-2 platform to advance the development of next-generation ADCs. The transaction consideration includes an upfront payment, R&D/regulatory/commercial milestone payments, and tiered sales royalties post-commercialization. Additionally, WuXi XDC will provide CMC development and manufacturing services for the collaborative ADC. This marks the second collaboration between the two parties following their initial partnership in 2024. Leveraging the technical advantage of hydrophilic linkers that significantly enhance ADC stability, WuXiTecan-2 has completed two out-licensing deals within just six months.
Interpretation:WuXi XDC has consecutively completed two out-licensing deals for its WuXiTecan-2 platform within six months, validating that the “technology platforms” of Chinese CDMOs/CRDMOs are becoming hard assets capable of independent monetization, rather than merely relying on manufacturing orders. Amid the global surge in ADC R&D, the “pick-and-shovel” business of payload-linker conjugation offers both high gross margins and strong customer stickiness—the deeper the client engagement, the more stable the subsequent CMC manufacturing orders. This reflects a clear trajectory for the local bioconjugation industry chain, transitioning from “contract manufacturing” to “technology licensing plus integrated enablement.”
9/17Huadong Medicine Partners with Meibo Biologics for Exclusive Collaboration on MB007: Betting on the ECM Collagen Regenerative Medicine Sector
On September 17, Huadong Medicine announced that its wholly-owned subsidiary, Sinclair Aesthetics (Hangzhou) Medical Technology Co., Ltd., had signed a strategic cooperation agreement with Beijing Meibo Pharmaceutical Biotechnology Development Co., Ltd. and Hunan Meibo Biopharmaceutical Co., Ltd. The parties have reached an exclusive strategic partnership for the MB007 extracellular matrix (ECM) collagen product in the Chinese market. MB007 is a regenerative medicine product developed by Meibo Biopharma based on ECM-derived collagen. This collaboration will leverage Huadong Medicine’s commercialization network in the medical aesthetics and consumer healthcare sectors to accelerate the product’s market introduction in China.
Interpretation:Driven by the dual engines of “pharmaceutical manufacturing + medical aesthetics consumption,” Huadong Medicine continues to strengthen its regenerative medicine and collagen pipelines through external collaborations. ECM-derived collagen offers superior structural and biological activity advantages over traditional animal-derived or recombinant collagen, aligning well with the robust demand in medical aesthetics and tissue repair. This collaboration extends Huadong’s consistent strategy of “exchanging distribution channels for technology” and “expanding product portfolios through an asset-light approach.” However, the ultimate commercial scale-up of the product will still depend on clinical evidence and the progress of end-user education.
9/17GenScript Integrates with Eli Lilly’s AI Drug Discovery Platform, Lilly TuneLab: Wet-Lab Experiments Become the “Validation Foundation” for AI-Driven Drug Development
On September 17, GenScript Biotech (1548.HK) announced a collaboration with Lilly TuneLab, Eli Lilly and Company’s AI/machine learning-enabled drug discovery platform. Under the agreement, GenScript will provide wet-lab services to companies participating in Lilly TuneLab, facilitating the rapid generation of experimental data to translate AI predictions into biologically validated results and accelerate the evaluation of drug candidates. Following the announcement, GenScript’s stock price surged more than 14% on heavy trading volume.
Interpretation:As AI drug discovery competition shifts from “whose model is stronger” to “whose data is more authentic,” wet-lab capacity is becoming a scarce foundation for validation. By integrating its gene synthesis, protein, and cellular experimental capabilities into Eli Lilly’s global AI discovery platform, GenScript is essentially closing the AI prediction loop with “real-world validation data.” This underscores an industry consensus: upstream computing power, midstream large language models, and downstream high-quality wet-lab validation are jointly constituting the new infrastructure of AI-driven drug discovery. In this evolving division of labor, Chinese CROs/CDMOs with substantial experimental capacity are poised to capture a critical segment.
9/18MindRank Unveils Intera, a Foundational Model Suite for Life Sciences: AI Drug Discovery Moves from “Screening Tool” to “Structural Understanding”
On September 18, MindRank, a clinical-stage AI-driven drug discovery company, officially launched its foundational life science model suite, Intera (Intelligence Era), and released the technical report for InteraFold-Chem, the first model in the InteraFold series. Focusing on predicting binding conformations between proteins and small molecules, InteraFold-Chem achieved a success rate of 54.4% in binding conformation prediction benchmarks, outperforming control models such as Protenix-v2 (48.5%), Chai-1, and Boltz-2. With a median ligand RMSD of 1.426 Å and a complex lDDT of 0.924, it ranked top among all evaluated models across these three metrics. The company plans to launch online services for InteraFold-Chem in the fourth quarter of 2026, followed by the sequential release of models for predicting antibody–antigen (InteraFold-Ab) and peptide–protein (InteraFold-Peptide) complex structures. Over the past five years, MindRank has focused on targets such as GPCRs and therapeutic modalities including molecular glues, allosteric regulation, and dual-target agents, resulting in seven preclinical candidates (PCCs) and two clinical pipelines. Its internally developed oral small-molecule GLP-1RA, MDR-001, progressed from project initiation to Phase III trial launch in approximately 4.5 years.
Interpretation:As AI-driven drug discovery shifts from “predicting which molecule is better” to “understanding how molecules bind to their targets,” structure prediction is becoming the decisive factor in the capabilities of large models. MindRank leverages InteraFold-Chem to tackle protein–small molecule binding conformation prediction and plans to develop multimodal structural models for antibodies, peptides, and more, essentially turning “structural understanding” into a reusable R&D infrastructure. Notably, the value of such foundation models lies not only in academic metrics but also in their ability to form a feedback loop with real-world wet-lab experiments and clinical data. MindRank’s approach of using its own clinical pipeline to feed back into model iteration represents a critical leap for AI for Science (AI4S) from “aesthetically pleasing models” to “practically useful R&D tools.” As competitors like Xunming Bio race to develop biological foundation models, China’s AI drug discovery sector is evolving from isolated point solutions to a competition over comprehensive, systematized platforms.
◍Capital InformationFinancing · IPO · Equity Changes
9/18Baili Tianheng Invests $5 Million in US-based Connexus: A Forward-Looking Move into Anti-Tumor Small Molecules
On September 18, Baili Tianheng announced that its wholly-owned subsidiary, Pangu Capital, intends to subscribe for 3.881 million Series A preferred shares of the U.S. innovative pharmaceutical company Connexus Therapeutics for $5 million. Upon completion of the transaction, Pangu Capital will hold a 7.91% equity stake in Connexus, corresponding to a post-money valuation of $63.21 million. Connexus focuses on the research and development of small-molecule drugs in the oncology field, with all its current pipeline projects in the preclinical stage. This investment aims to expand Baili Tianheng’s early-stage layout in the field of cutting-edge anti-tumor drugs.
Interpretation:As core ADC pipelines such as BL-B01D1 (iza-bren) enter the global registration and commercialization phase, Baili Tianheng has strategically acquired minority equity stakes in overseas early-stage small-molecule anti-tumor assets. This move exemplifies a capital allocation strategy of “using commercialization cash flows to fund frontier exploration.” The 7.91% minority stake allows the company to maintain technological oversight and retain the initiative for potential in-licensing, without bearing the heavy risks associated with asset-intensive R&D. This approach aligns with the prevailing trend among leading Chinese pharmaceutical companies of pursuing dual tracks of “independent R&D” and “global equity investment.”
9/17NOVAST Launches ChiNext IPO with Plans to Raise RMB 950 Million: Leading Exporter of Oral Extended-Release and Controlled-Release Formulations Sprints Toward A-Share Listing
On September 17, Novast Laboratories, Limited disclosed its prospectus and related documents, initiating its initial public offering (IPO) process on the ChiNext Board of the Shenzhen Stock Exchange. China International Capital Corporation Limited serves as the sponsor. The company plans to publicly issue approximately 134 million shares (accounting for 15% of the total share capital post-issuance), with the preliminary inquiry date scheduled for September 22 and the subscription date for September 28. The proposed fundraising amount is RMB 950 million, to be used for the industrialization base, R&D center, drug development, and working capital replenishment. As of the end of March 2026, the company had 48 independently developed products approved by the U.S. FDA, ranking first in China for the number of FDA-approved oral extended-release and controlled-release formulations.
Interpretation:NOVAST has established a differentiated competitive moat in the high-barrier niche of oral sustained- and controlled-release formulations through its unique positioning of “batch ANDA approvals in the U.S. + industrialization in China.” The allocation of RMB 950 million in fundraising toward production capacity and R&D signifies the company’s upgrade from “overseas contract manufacturing/generics” to an “autonomous and controllable complex formulation platform.” Against the backdrop of import substitution for domestically produced complex formulations and the acceleration of dual filings in China and the United States, companies like NOVAST, backed by genuine overseas regulatory approvals and quality systems, are being reevaluated by the capital markets.
◍Pharmaceutical Industry UpdatesDomestic Pharmaceutical Companies · Clinical Trials and Regulatory Submissions
9/18Gan&Lee Pharmaceuticals’ GLR1075 Injection Receives Clinical Trial Approval: Targeting Hypoalbuminemia in Cirrhotic Ascites
On September 18, Gan&Lee Pharmaceuticals (603087.SH) announced that it had received the "Notice of Approval for Drug Clinical Trials" from the National Medical Products Administration regarding its investigational drug, GLR1075 injection. The approval permits the conduct of clinical trials for GLR1075 in the treatment of hypoalbuminemia associated with cirrhotic ascites. GLR1075 is an innovative biologic developed by Gan&Lee as part of its strategic portfolio in metabolism and liver diseases. The company will proceed with clinical trials in accordance with relevant drug registration regulations and, upon successful completion, will submit a production application through the statutory procedures.
Interpretation:Gan&Lee Pharmaceuticals, renowned for its expertise in insulin and analogs, marks its pipeline expansion into adjacent therapeutic areas such as liver disease and metabolic complications with the clinical approval of GLR1075. There is a significant unmet clinical need for treating hypoalbuminemia associated with cirrhotic ascites, given the limited existing symptomatic therapies. If GLR1075 can correct albumin metabolic imbalance, it will open up a second growth curve beyond the company’s core insulin business; however, as an early-stage asset, it remains far from commercialization, and value realization still requires validation through clinical data.
9/18Hengrui Pharma’s SHR-1139 Injection Receives Clinical Trial Approval: Ulcerative Colitis Welcomes a Potential First-in-Class Biologic
On September 18, Hengrui Pharma (600276.SH) announced that its subsidiary, Guangdong Hengrui Pharmaceutical Co., Ltd., had received the "Notice of Approval for Drug Clinical Trials" from the National Medical Products Administration (NMPA) for SHR-1139 Injection, approving the initiation of clinical trials for ulcerative colitis. SHR-1139 is a therapeutic biological product independently developed by Hengrui, expected to achieve synergistic efficacy through mechanisms such as inhibiting inflammatory responses and maintaining the epithelial barrier. A search indicates that no similar drugs have been approved for marketing either domestically or internationally. The company has incurred cumulative R&D investments of approximately RMB 71.28 million.
Interpretation:In the field of autoimmune and inflammatory diseases, Hengrui Pharma is rapidly strengthening its position through a dual-track strategy of small molecules and biologics. As a potential first-in-class biologic for ulcerative colitis, SHR-1139 targets a therapeutic area with a large patient population and insufficient response to existing therapies. If its scarcity label—“no similar products approved domestically or internationally”—can be translated into clinical superiority, it will enhance Hengrui’s differentiated competitiveness in this prime autoimmune segment and continue its pipeline momentum characterized by “high-intensity R&D investment leading to frequent clinical approvals.”
9/18Lifang Pharmaceutical's Clinical Trial Application for Celecoxib and Tramadol Hydrochloride Tablets Accepted: First Domestic Filing for a Combination Product for Acute Pain
On September 18, Lifang Pharmaceutical (003020.SZ) announced that it had received the "Notice of Acceptance" from the National Medical Products Administration (NMPA) for its clinical trial application for the drug Celecoxib and Tramadol Hydrochloride Tablets. The proposed indication is for the treatment of moderate to severe acute somatic pain in adults over a short term. The drug formulation contains 56 mg of celecoxib and 44 mg of tramadol hydrochloride. The original innovator drug was approved for marketing in the United States in 2021 but has not yet been launched in China. Lifang Pharmaceutical is the first company in China to file for clinical trials of this product.
Interpretation:Combination analgesia has become the mainstream approach to mitigate the risk of opioid abuse and enhance synergistic, multi-mechanism pain relief. The fixed-dose combination of celecoxib (an NSAID) and tramadol (a weak opioid) balances anti-inflammatory and central analgesic effects while reducing monotherapy doses and associated side effects. Although this formulation is already marketed in the United States, it remains unavailable in China. Lifang Pharmaceutical has taken the lead by initiating the first clinical trials, positioning itself to potentially secure a first-to-market generic or improved-new-drug advantage in the niche market for postoperative and acute pain management. However, the stringent requirements for clinical endpoint design and safety evaluation of combination drugs introduce uncertainty regarding the approval timeline.
9/17Everest Medicines’ Application for Domestic Production Transfer of Vosevi Accepted by NMPA: Accelerating Localization of High-End Formulations
On September 17, Everest Medicines announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) had accepted its marketing application for the domestic production transfer of VELSIPITY® (etrasimod arginine tablets), and included it in the priority review and approval program. VELSIPITY® is an oral small-molecule immunosuppressant. Localized production in China is expected to officially commence in 2027, which will significantly enhance the supply stability and accessibility of this product in the domestic market.
Interpretation:Driven by the dual imperatives of “supply chain security + cost optimization,” the localized production of imported innovative drugs is shifting from isolated cases to a new norm. Everstone’s decision to transition Vespera® to domestic manufacturing not only substantively safeguards drug accessibility for patients in China but also reflects that the License-in model has entered a deeper phase characterized by “introduction–localization–scale-up.” In this context, the true competitive moat lies no longer merely in acquisition rights, but in the capability to successfully execute the entire value chain—registration, production, and commercialization—of overseas assets within China.
9/18Eli Lilly’s RET Inhibitor Selperatinib: New Formulation Approved in China—Tablet Enhances Convenience of RET-Targeted Therapy
On September 18, the official website of the National Medical Products Administration (NMPA) announced the approval of Eli Lilly’s selpercatinib tablets for marketing. Selpercatinib is a potent, highly selective oral RET tyrosine kinase inhibitor. Its capsule formulation was first approved by the U.S. Food and Drug Administration (FDA) in May 2020, becoming the world’s first highly selective RET inhibitor. The capsule formulation was approved in China in October 2022 for the treatment of RET fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC), and RET fusion-positive thyroid cancer. The approval of the tablet formulation in China will provide a more convenient oral administration option for patients with tumors harboring RET gene alterations. According to the LIBRETTO-121 study, which evaluated selpercatinib in pediatric patients with advanced solid tumors or primary central nervous system (CNS) tumors carrying RET gene alterations, the objective response rate (ORR) was 36% among evaluable patients (n=36) after a median follow-up of 30 months. The duration of response (DOR) rate at 24 months was 100%, and the progression-free survival (PFS) rate was 86%.
Interpretation:Selpercatinib is the world’s first highly selective RET inhibitor. Eli Lilly’s formulation upgrade from capsules to tablets essentially leverages a superior administration experience to consolidate its first-mover advantage in the RET target space. Compared with capsules, tablets offer distinct advantages in swallowing compliance, dosing flexibility, and supply-chain resilience, which are particularly critical for the chronic disease management of cancer patients requiring long-term medication. In niche indications such as RET fusion-positive non-small cell lung cancer (NSCLC) and medullary thyroid cancer, highly selective RET inhibitors are gradually replacing multi-kinase inhibitors like cabozantinib and vandetanib as the standard of care. When benchmarked against competitors such as Blueprint Medicines’ pralsetinib (Gavreto), formulation differences are emerging as a new dimension of competitive differentiation. This also reflects the strategy of multinational pharmaceutical companies in China to intensively launch new formulations and new indications, while continuously deepening their engagement in rare and precise oncology targets.
9/18Insilico Medicine’s Cell Cover Paper Open-Sources AI Longevity Toolkit: From “Discovering Drugs” to “Defining Aging”
On September 18, Insilico Medicine (HKEX: 3696) announced that its collaborative research with Liquid AI, the Buck Institute for Research on Aging, Harvard Medical School, and Brigham and Women’s Hospital was selected as the cover story of the current issue of Cell (Impact Factor: 66.85). This study marks the first global open-source release of an AI toolkit for longevity research, comprising: LongevityBench (the first open benchmark for evaluating AI reasoning capabilities in aging biology, covering five major modalities: clinical, genetic, epigenetic, transcriptomic, and proteomic); Longevity-LLMs (five lightweight open-source large language models with 600 million to 9 billion parameters, fine-tuned on clinical and multi-omics aging data, whose performance matches or surpasses that of 16 leading general-purpose models from OpenAI, Google, and others, with L-Qwen3.5-9B achieving the highest comprehensive score); and Longevity Claw (an open-source agent platform capable of autonomously planning multi-step workflows, integrating specialized tools, and nominating potential targets, which has already identified 328 anti-aging candidate targets with a 5.6-fold enrichment compared to the reference set).
Interpretation:Following the publication in Nature Biotechnology in September of Phase IIa evidence demonstrating rentosertib’s ability to reverse “biological age,” Insilico Medicine has once again made headlines with a Cell cover story, pushing the boundaries of AI-driven drug discovery from merely “identifying candidate molecules” to “defining and quantifying aging itself.” Notably, lightweight specialized models have outperformed larger general-purpose large language models on LongevityBench, reaffirming that “domain-specific data plus deep optimization” can surpass “sheer scale.” Open-source benchmarks and agent-based platforms are shifting the competition in AI for Science (AI4S) from a race among closed-source models to a contest over scientific infrastructure that is “reproducible, evaluable, and co-developable.” Ultimately, the true watershed moment for such longevity-focused AI will be whether its target nominations can successfully complete the closed loop of wet-lab validation.
◍Global Pharma NewsGlobal Perspective · Cross-Border Dynamics
9/17Orbis and Novo Nordisk Strike $1.4 Billion Deal: Oral Macrocyclic Drugs to Transform Drug Delivery in the Metabolic Disease Arena
On September 17, Danish biotech company Orbis Medicines announced a strategic collaboration and licensing agreement with Novo Nordisk, valued at up to $1.4 billion, to jointly discover and develop next-generation oral macrocycle drugs for cardiometabolic diseases. Orbis will receive upfront payments, research and development milestones, commercial milestones, and tiered royalties on future product sales, totaling up to $1.4 billion; Novo Nordisk will also make a strategic investment in Orbis. The collaboration leverages Orbis’s proprietary nGen platform—which integrates generative AI with high-throughput synthesis—to design macrocyclic molecules with oral bioavailability, aiming to replace current injectable biologics with oral formulations.
Interpretation:In the global race to develop oral GLP-1 therapies, macrocyclic drugs are regarded as the key molecular modality bridging “biologic potency” with “small-molecule oral convenience.” Building on its existing portfolio of oral GLP-1 products, Novo Nordisk has further bet on Orbis’s AI-driven macrocycle discovery platform, aiming to comprehensively transition injectable metabolic therapies into oral formulations. The fact that Orbis has simultaneously secured investment from Eli Lilly (Novo’s primary competitor) while forming an alliance with Novo underscores how major pharmaceutical companies are casting a wide net to secure next-generation delivery technologies through minority equity investments and multi-platform collaborations. This deal further confirms that AI-enabled molecular discovery has moved beyond mere narrative to become a core component of frontline business development transactions.
9/17Ultragenyx’s Gene Therapy FAYUVI Receives Full FDA Approval: At $3.95 Million Per Dose, It Becomes the World’s Second Most Expensive Drug
On September 17, Ultragenyx Pharmaceutical announced that the U.S. FDA has granted standard full approval to FAYUVI™ (rebisufligene etisparvovec-hopf, development code UX111) for the treatment of pediatric patients with Mucopolysaccharidosis Type IIIA (MPS IIIA, Sanfilippo Syndrome Type A). FAYUVI is the first FDA-approved therapy for Sanfilippo Syndrome Type A and the second approved gene therapy from Ultragenyx. UX111 is an in vivo AAV9 gene therapy that delivers a functional SGSH gene to cells via a single intravenous infusion, replacing the deficient sulfamidase enzyme and reducing the accumulation of heparan sulfate (HS) in the central nervous system. The approval was based on the pivotal Transpher A trial and long-term follow-up data (up to nearly 8 years): In the modified intent-to-treat population (N=17), children aged 24–60 months showed a 23.5-point higher raw score on the Bayley-III Cognitive Scale compared to an external natural history cohort (N=27) (p<0.0001). Ultragenyx will price the therapy at $3.95 million per dose, making it the second most expensive drug globally after Lenmeldy ($4.25 million). J.P. Morgan estimates its global peak sales to be approximately $200–250 million.
Interpretation:One-Time Gene Therapies Are Pushing Pricing for “Ultra-Rare Diseases” to Unprecedented Heights—FAYUVI, at $3.95 million per dose, has become the second most expensive drug globally, underpinned by its strong pricing power as the “first approved therapy” for Sanfilippo syndrome type A, which has a commercially accessible population of only 3,000–5,000 patients. The transition of AAV9 in vivo gene therapy from RMAT/Fast Track designation to standard full approval also marks that this class of one-time curative therapies is moving from accelerated approval toward comprehensive regulatory recognition of confirmed clinical benefit. For the industry, the commercial logic for ultra-rare disease gene therapies has shifted from “selling drugs” to “one-time transactions priced based on lifetime value”; for payers, establishing innovative payment mechanisms such as installment plans and outcome-based payments for these sky-high-priced one-time therapies will become the true bottleneck to scaling up access.