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Last update 28 Feb 2026

Valoctocogene roxaparvovec

Last update 28 Feb 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

AAV5-HFVIII-SQ, DNA (synthetic adeno-associated virus 5 vector BMN 270 human blood-coagulation factor VIII SQ variant-specifying), Factor-VIII-gene-therapy-BioMarin

+ [4]

Target

Action

stimulants

Mechanism

F8 stimulants(Coagulation factor VIII stimulants)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia A

Inactive Indication-

Originator Organization

BioMarin Pharmaceutical, Inc.

Active Organization

BioMarin Pharmaceutical, Inc.

Shanghai Vitalgen Biopharma Co., Ltd.

Inactive Organization

BioMarin International Ltd.

License Organization-

Drug Highest PhasePhase 3

First Approval Date

European Union (24 Aug 2022),

Hemophilia A

RegulationOrphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Orphan Drug (Australia), Regenerative Medicine Advanced Therapy (United States), Conditional marketing approval (European Union), Accelerated assessment (European Union), Priority Review (United States), Breakthrough Therapy (United States)

Structure/Sequence

Sequence Code 29923082

Source: *****

Clinical Trials associated with Valoctocogene roxaparvovec

NCT06224907

/ Active, not recruitingPhase 3

GENEr8-JPN: A Phase 3 Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Japanese Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.

Start Date25 Dec 2023

Sponsor / Collaborator

BioMarin Pharmaceutical, Inc.

NCT04684940

/ Active, not recruitingPhase 1/2

A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.

Start Date10 Dec 2020

Sponsor / Collaborator

BioMarin Pharmaceutical, Inc.

NCT04323098

/ CompletedPhase 3

A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients

This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.

Start Date08 Dec 2020

Sponsor / Collaborator

BioMarin Pharmaceutical, Inc.

100 Clinical Results associated with Valoctocogene roxaparvovec

100 Translational Medicine associated with Valoctocogene roxaparvovec

100 Patents (Medical) associated with Valoctocogene roxaparvovec

Literatures (Medical) associated with Valoctocogene roxaparvovec

01 Jan 2026·IUBMB LIFE

Balancing Promise and Peril: Hemophilia Gene Therapy Insights

Review

Author: Follenzi, Antonia ; Cottonaro, Alessia ; Akula, Saicharan ; Borroni, Ester ; Merlin, Simone

ABSTRACT:

Hemophilia is an inherited disorder characterized by impaired blood clotting caused by mutations in the genes responsible for producing coagulation factor (F) VIII (hemophilia A, HA) or FIX (hemophilia B, HB). Current treatment primarily relies on replacement therapy, involving frequent and costly infusions of FVIII or FIX concentrates. While effective, these treatments come with the risk of developing neutralizing antibodies (inhibitors) against the infused factor. In recent years, non‐factor replacement therapies have emerged as innovative treatment options, offering enhanced efficacy especially for patients with inhibitors. Despite their advantages, these approaches still fall short of providing a definitive, long‐term cure. Since hemophilia is a monogenic disease, it presents an excellent opportunity for cell and gene therapy approaches aimed at achieving durable treatment and potentially a cure. Over the past three decades, remarkable advancements have been made in hemophilia gene therapy, culminating in the approval of Valoctocogene roxaparvovec (ROCTAVIAN, AAV‐FVIII) and Etranacogene dezaparvovec (HEMGENIX, AAV‐FIX) for patients with severe HA and HB, respectively. Nevertheless, gene therapy poses questions regarding its long‐term efficacy and safety. This review synthesizes findings from clinical trials, addresses persistent challenges in hemophilia gene therapy, and underscores the biological constraints and limitations inherent to viral vector‐based approaches.

05 Dec 2025·Hematology-American Society of Hematology Education Program

Deconstructing gene therapy in hemophilia for the clinician

Review

Author: Young, Guy

Abstract:

After decades of research, gene therapy for hemophilia is now commercially available for both hemophilia A and B. Currently, two products, valoctocogene roxaparvovec (for hemophilia A) and etranacogene dezaparvovec (for hemophilia B), have been licensed following approval in the United States and several other countries. Therefore, clinicians must familiarize themselves with these novel treatment options just as they do with other newly available products in order to provide their patients the opportunity to consider which treatment may suit them best. Undoubtedly, gene therapy is a novel platform for treating human disease, and unlike other hemophilia treatments, its biology, mechanisms, and administration logistics are quite complex. Furthermore, additional products using different approaches have entered clinical trials, with more in the preclinical stages of development. This review's aims are (1) to deconstruct gene therapy in hemophilia and provide a basic framework for understanding its components and processes, including the transgene, the vector, and the delivery systems, in order to help clinicians present gene therapy as a treatment option in a shared decision-making model, better understand the clinical data, and explain gene therapy to their patients; (2) to gain knowledge of the currently approved gene therapies for hemophilia A and B, including their eligibility and exclusion criteria and the range of expected outcomes; and (3) to comprehend the shared decision-making process for these therapies and their implementation in clinical practice. In addition, a brief review of the currently approved products and those in clinical trials is presented, followed by a discussion of practical considerations for implementing gene therapy in practice.

01 Mar 2026·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Overview of gene therapy for hemophilia: questions and answers to navigate the innovation

Review

Author: La Mura, Vincenzo ; Miesbach, Wolfgang ; Hoffmann, Pascal ; Bitto, Niccolò

Hemophilia is a rare inherited bleeding disorder affecting approximately 1.1 million individuals worldwide, characterized by deficiency in clotting factor (F)VIII (hemophilia A) or FIX (hemophilia B). Despite advances in prophylactic factor replacement therapy and nonfactor treatments, persons with hemophilia continue to experience breakthrough bleeding, joint deterioration, and treatment burden, highlighting the need for transformative therapeutic approaches. This educational review provides a comprehensive overview of gene therapy developments for hemophilia, focusing on adenoassociated virus (AAV)-based approaches, their clinical implementation, and multidisciplinary care requirements. Gene therapy represents a paradigm shift in hemophilia treatment by addressing the underlying genetic cause through a potentially 1-time intervention. AAV-based gene therapies have demonstrated sustained factor expression in clinical trials, with persons with hemophilia, achieving factor levels in the normal or mild hemophilia range and significant reductions in bleeding rates. Recent phase III trials of valoctocogene roxaparvovec (hemophilia A) and etranacogene dezaparvovec (hemophilia B) have shown durable factor expression for up to 5 years, with the FIX-Padua variant enhancing treatment efficacy 5- to 8-fold. However, approximately 20% to 90% of persons with hemophilia experience transaminase elevation postinfusion, requiring immunosuppressive management and hepatologic monitoring. Gene therapy for hemophilia has evolved from experimental concept to clinical reality, offering sustained hemostatic protection with reduced treatment burden. While challenges remain regarding long-term durability, immune responses, and standardized care pathways, current AAV-based therapies represent a transformative advance. Future developments including alternative vectors, enhanced factor variants, and gene editing technologies promise to further improve treatment outcomes and expand patient access.

227

News (Medical) associated with Valoctocogene roxaparvovec

28 Feb 2026

·endpoints.news

GLP-1 news galore; Top 100 venture investors; Doug Ingram to step down; and more

Happy Saturday, and welcome back to Endpoints Weekly. Our senior biopharma journalist Elizabeth Cairns covered a flurry of readouts this week from Eli Lilly and Novo Nordisk. We’ve summarized the data below, as well as Elizabeth’s thoughts on how Novo Nordisk might navigate what Jefferies managing director Michael Leuchten called a “strategic conundrum.” Keep reading for John Carroll’s breakdown on the top 100 venture investors in biotech, plus more details on Doug Ingram’s decision to step down from Sarepta and the end of Roctavian’s commercial saga. Endpoints’ Max Bayer and Zachary Brennan also reported this week that the FDA is using an outside investigator to look into complaints against CBER Director Vinay Prasad. And don’t forget to submit your nominations to Endpoints’ fifth annual LGBTQ+ Leaders in Biopharma special report. Nominations are due by March 25. — Nicole DeFeudis It was a busy week for Eli Lilly and Novo Nordisk. Things kicked off with news about CagriSema, Novo’s GLP-1 plus amylin therapy. The Danish pharma company announced that in a head-to-head trial testing CagriSema against Lilly’s Zepbound in obesity, Zepbound came out on top . Patients who were taking CagriSema lost 20.2% of their weight after 84 weeks, while those taking Lilly’s drug lost 23.6%. Then, Novo followed up with results about UBT251, its triple-G agonist going after the GLP-1, GIP and glucagon receptors. In a Phase 2 study, the data looked slightly better than Lilly’s more advanced triple-G, retatrutide, in its own similar trial. (These drugs were not pitted head-to-head.) Novo’s study was conducted by its partner United Therapeutics in China and enrolled about 200 patients. Despite the positive results for Novo, UBT251 will likely reach the market after retatrutide. And in a third trial, looking at oral GLP-1 drugs, Lilly’s orforglipron again came out on top over Novo’s Rybelsus pill. These data came from a trial conducted by Lilly in type 2 diabetes patients that isn’t intended to support approval, but could encourage doctors to prescribe orforglipron off-label. Patients given 12 mg or 36 mg of orforglipron for a year had their blood sugar levels cut by 1.71% and 1.91%, respectively. Those given Novo’s drug at 7 mg and 14 mg doses saw reductions in blood sugar of 1.23% and 1.47%, respectively. So what does this all mean? Senior biopharma journalist Elizabeth Cairns attempted to answer that question , and how Novo Nordisk and new CEO Maziar Mike Doustdar are navigating a “strategic conundrum.” Despite reaching heights that began to reshape Denmark’s economy in 2023, Novo’s stock price is now approaching a five-year low. Read more here. Endpoints founding editor John Carroll was surprised to see OrbiMed jump to number one on our most recent annual list of top venture investors in the life sciences sector. OrbiMed, which finished fourth in 2024, more than doubled the number of rounds it participated in last year, and total funding in those rounds rose from $4.5 billion in 2024 to $5.9 billion in 2025. RA Capital, which was in the top slot for 2024 and 2023, pulled back in 2025, taking second place. It participated in 37 rounds and invested $4.2 billion, down from 49 deals and $5.6 billion the year prior. There were other notable changes, including ARCH’s slide out of the top 10. The company landed at 11th with 26 deals in 2025, down from 33 the year before. The list highlights the growing role of big rounds in biotech. There were 103 venture rounds over $100 million in biopharma in 2025, compared to 93 in 2024 and 69 in 2023. That boost came as M&A sped up last year and the industry geared up for another round of IPOs. Click here to see John’s full breakdown of the top 100. In a surprising move, Sarepta CEO Doug Ingram said he would step down from his position at the end of the year, unless a replacement is found before then. Ingram said he is retiring for family reasons, as two of his immediate family members were diagnosed with myotonic dystrophy type 1, a progressive genetic muscle disease. Ingram’s decision ends a brash tenure in which he brought several Duchenne muscular dystrophy products to market and later presided over a spiraling share price amid intense safety and regulatory questions. The announcement came as a shock during the company’s earnings call, with no official press release or mention in the company’s earnings report earlier that afternoon. He became a contentious figure in the rare disease field. Sarepta won three new drug approvals using limited and often murky data, generating billions in revenue. Last year, the company faced intense scrutiny over patient deaths on its gene therapies, and ultimately pulled its Duchenne gene therapy Elevidys for older boys. Read more from reporter Lei Lei Wu, as she takes a bird’s-eye view of Ingram’s tenure — and what’s next for Sarepta. The FDA has been using an outside investigator in a probe of workplace complaints against CBER Director Vinay Prasad, Endpoints’ Max Bayer and Zachary Brennan reported this week. The Wall Street Journal previously reported the existence of some workplace behavior complaints against Prasad. Max and Zach reported that among the complaints are that Prasad created a toxic workplace, verbally berated staff and retaliated against staff pushback. The details come as FDA leadership continues to publicly defend Prasad against criticism. Commissioner Marty Makary called Prasad “a genius” in a recent CNBC interview. A spokesperson for HHS said in a statement to Endpoints that “this story is part of a PR campaign against Dr. Prasad.” Read more here. For all its blockbuster hopes and high-profile status in the gene therapy field, Roctavian’s commercial saga has ended, senior biopharma correspondent Andrew Dunn writes . After a few months of seeking to divest Roctavian, BioMarin said this week it failed to find “a qualified buyer” for the gene therapy and will voluntarily withdraw it from the market. The commercial failure is a reminder of the gap between promising science and a viable drug. Roctavian was never able to find a foothold in the market for severe hemophilia A, a genetic blood-clotting disorder. The drug brought in $36 million in 2025 revenue, up from $26 million the year before — but far less than the company and Wall Street initially hoped. DON’T MISS

Clinical ResultPhase 2IPO

25 Feb 2026

·firstwordpharma.com

Biopharma bites: BioMarin's search for Roctavian buyer a bust, plus GSK's Frontier deal, and FDA updates from Immedica, MacroGenics

With no buyer, BioMarin pulls gene therapy from shelvesGSK inks $1B kidney-focused siRNA pactFDA approves Immedica's ultra-rare metabolic disease therapyPatient death triggers partial hold of MacroGenics' mid-stage cancer studyWith no buyer, BioMarin pulls gene therapy from shelvesHaving failed to find a buyer for its haemophilia A gene therapy, BioMarin Pharmaceutical said Monday that it will withdraw Roctavian (valoctocogene roxaparvovec-rvox) from the market. The company announced plans to divest the programme in October, after the therapy posted third-quarter sales of just $3 million. According to BioMarin, it "undertook a comprehensive effort to identify a potential buyer."In connection with its decision to pull Roctavian from shelves, BioMarin said it will take a $240 million charge, including a $119-million inventory write-off. -Elizabeth EatonGSK inks $1B kidney-focused siRNA pact GSK has agreed to shell out $40 million upfront for global rights to two siRNA programmes from China's Frontier Biotechnologies in a deal that could net the Chinese drugmaker up to $963 million in milestones, plus tiered royalties (see – Vital Signs: Charting China's course westward into 2026).In return, the UK drugmaker obtains exclusive worldwide rights to the two assets; one candidate is at the IND stage, while the second remains preclinical. Frontier will advance one programme through a Phase I study in China and complete IND-enabling work on the other before GSK assumes responsibility for global clinical development, regulatory filings and commercialisation."This agreement further strengthens our immunology pipeline, adding two potential first-in-class oligonucleotide therapies with significant opportunity to improve patient outcomes across multiple kidney diseases," said Kaivan Khavandi, global head for respiratory, immunology and inflammation R&D at GSK.The Frontier pact follows GSK's licensing deal with Empirico last October for rights to a Phase I siRNA programme, EMP-012, in chronic obstructive pulmonary disease. Empirico received $85 million upfront and is eligible for up to $660 million in milestones.-Anna BratulicFDA approves Immedica's ultra-rare metabolic disease therapyThe FDA on Monday awarded Immedica Pharma's arginine specific enzyme therapy, Loargys (pegzilarginase-nbln), with an accelerated approval to treat hyperargininaemia in patients 2 years and older with arginase 1 deficiency (ARG1-D) in conjunction with dietary protein restriction. The treatment is expected to be available in the US starting in April. The clearance was based on findings from the Phase III PEACE trial, in which Loargys significantly reduced plasma arginine from baseline compared with placebo at 24 weeks. According to Immedica, the therapy is the first and only treatment to address persistently elevated levels of plasma arginine, the disease's main driver. ARG1-D is an ultra-rare progressive metabolic disease that affects an estimated 250 people living in the US.-Elizabeth EatonPatient death triggers partial hold of MacroGenics' mid-stage cancer studyMacroGenics said Monday that the FDA has placed a partial clinical hold on the Phase II LINNET study of its experimental cancer drug lorigerlimab following a patient death, pausing patient enrolment. The company reported safety events that occurred across four patients, including two cases of grade 4 thrombocytopenia, one report of grade 4 myocarditis, and one patient with grade 4 neutropenia and concurrent septic shock, which resulted in their death. MacroGenics has dosed 41 patients with platinum-resistant ovarian cancer or clear cell gynaecologic cancer with lorigerlimab, a bispecific DART molecule that targets PD-1 and CTLA-4. Though enrolment is paused, study participants can continue to receive lorigerlimab."MacroGenics is fully committed to working closely with the FDA to resolve the partial clinical hold and we intend to resume study enrollment as soon as possible," said CEO Eric Risser.-Elizabeth Eaton

Phase 3Phase 2License out/inDrug ApprovalClinical Result

24 Feb 2026

·www.fiercepharma.com

BioMarin pulls hemophilia gene therapy Roctavian, taking $240M hit after divestiture efforts flounder

BioMarin's decision to withdraw Roctavian from the market resulted in charges of about $240 million over the fourth quarter of 2025, including a $119 million inventory write-off. Just three months after further scaling back its support for the struggling hemophilia A gene therapy Roctavian, the company is walking away altogether by pulling the treatment from the market. The move follows a “comprehensive effort” to identify a potential buyer for the therapy, BioMarin explained Monday in its fourth-quarter earnings press release. Back in October, the company had decided to divest Roctavian, with CEO Alexander Hardy citing the approach as “consistent with BioMarin's portfolio strategy” and offering “the most promising opportunity for ensuring continued patient access to Roctavian,” he said at the time. The writing had long been on the wall for BioMarin to part with Roctavian, which was first approved in 2023 as the first one-time gene therapy for hemophilia A. Since mid-2024, the company has left its gene therapy manufacturing plant in an “idle state” as it dwindled its Roctavian operations to solely the U.S., Germany and Italy, three markets where the drug has secured reimbursement policies. At the time, the goal was to cut Roctavian’s direct annual expenses to $60 million starting in 2025 and make the product profitable by the end of 2026.Now, after BioMarin was unsuccessful in finding a “qualified buyer” to take on the product, the company will officially withdraw the med from the market, it said. This decision resulted in charges of about $240 million over the fourth quarter of 2025, including a $119 million inventory write-off and $118 million in asset value impairments.Roctavian sales came out to $36 million in 2025, up from 2024’s $26 million. The drug was still seeing some commercial use in its end of days, with a man in Michigan becoming the state’s first patient to use the therapy outside of clinical trials upon being infused in January, the Detroit Free Press reported earlier this month.Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and Novo Nordisk’s Alhemo. Common gene therapy hurdles, such as eligibility limitations and reimbursement struggles, also pulled Roctavian back. Upon taking the helm in 2023, Hardy had outlined three potential paths for the drug. While the focus at the time was on “establishing the opportunity” for Roctavian, a divestment was listed as Hardy’s third option. BioMarin's post-Roctavian future As BioMarin leaves Roctavian behind, the company is looking to a new chapter after inking its largest-ever transaction late last year with a $4.8 billion acquisition of Amicus Therapeutics. The deal gives BioMarin two potential blockbusters in Fabry disease drug Galafold and Pompe disease combination treatment Pombiliti-Opfolda. The addition of Amicus’ portfolio would “accelerate BioMarin’s revenue growth immediately upon close,” Hardy said at the time. The deal is expected to wrap up in the second quarter of this year. Any contribution from the Amicus products is excluded from BioMarin’s 2026 guidance, which forecasts between $3.3 billion to $3.4 billion in full-year sales. In 2025, the company collected revenue of $3.2 billion, up 13% from 2024. BioMarin attributed much of its yearly growth to market gains for its collection of enzyme therapies, as well as achondroplasia treatment Voxzogo. With $927 million in 2025 sales, Voxzogo is the company’s biggest revenue driver. But William Blair analysts argued in a Tuesday note that BioMarin will ultimately require “additional business development” to fully offset the anticipated Voxzogo erosion to come from new market entrants over the next two years. That competition could come from BridgeBio and its oral candidate infigratinib, which recently showed a statistically significant improvement in annualized height velocity from baseline in a phase 3 trial of children with achondroplasia. Mizuho analysts predicted that BridgeBio’s option could “very well end up being the agent of choice for this indication,” pointing to the wide age group trialed and the convenience of an oral offering. Voxzogo, meanwhile, is administered as a daily subcutaneous injection. However, BioMarin has another achondroplasia option up its sleeve in its early-stage BMN 333, which could be a “potentially superior version of Voxzogo,” an Evercore ISI analyst pointed out after the company revealed positive phase 1 results in August.

AcquisitionPhase 1Phase 3Clinical ResultDrug Approval

100 Deals associated with Valoctocogene roxaparvovec

External Link

KEGG	Wiki	ATC	Drug Bank
-	Valoctocogene roxaparvovec	-	DB15561

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hemophilia A	European Union	BioMarin International Ltd.	24 Aug 2022
Hemophilia A	Iceland	BioMarin International Ltd.	24 Aug 2022
Hemophilia A	Liechtenstein	BioMarin International Ltd.	24 Aug 2022
Hemophilia A	Norway	BioMarin International Ltd.	24 Aug 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03370913 \| NCT02576795 (GENEr8-1, Pubmed \| Adv Ther)	-	Hemophilia A	141	Valoctocogene roxaparvovec (Severe Hemophilia A)	vwlgpbbzfs(hlsnmvrujo) = oebsdjzdmu sipyvndcmp (kaxjxinisi ) View more	Positive	23 Sep 2025
NCT03520712 (GENEr8-AAV5+, CTgov)	Phase 1/2	Hemophilia A	3	BMN	hsqazibojx = ehmrhbjcuc rmrizmmwbo (njuxaoposo, wihjkfyurf - vkjhntxbcp) View more	-	22 Aug 2025
NCT02576795 (CTgov)	Phase 1/2	Hemophilia A	15	6E12 (BMN 270 6E12 vg/kg)	aaiizwctah = akzbkdwuyd teumkrhwus (jlieaxfgij, asbebocctd - wjiqaqpgnd) View more	-	10 Apr 2025
NCT02576795 (CTgov)	Phase 1/2	Hemophilia A	15	BMN (BMN 270 2E13 vg/kg)	aaiizwctah = ldqqzwnmlh teumkrhwus (jlieaxfgij, icnttoyrse - nesauualek) View more	-	10 Apr 2025
NCT03370913 (GENEr8-1, ASH2024)	Phase 3	Hemophilia A FVIII levels	-	Valoctocogene roxaparvovec	ejavxtyfjw(xbhgqsoulm) = iimojbgxof tysldeeuta (krlzoqwsnw )	Positive	09 Dec 2024
NCT03370913 (GENEr8-1, Pubmed)	Phase 3	Hemophilia A	112	Valoctocogene Roxaparvovec	dqhaxtoiwz(urpeqovcsj) = zhlusbomdx ozixvggfjb (wkqcjyplwj )	Positive	15 Apr 2024
NCT03370913 (GENEr8-1, Pubmed)	Phase 3	Hemophilia A	112	Prophylactic Factor VIII Replacement	dqhaxtoiwz(urpeqovcsj) = fnbcxvqtmo ozixvggfjb (wkqcjyplwj )	Positive	15 Apr 2024
NCT04323098 (GENEr8-3, CTgov)	Phase 3	Hemophilia A	22	BMN 270+roxaparvovec (BMN 270 (Valoctocogene roxaparvovec))	jdeoovrkmz(fqprwkaplq) = ovxuspbxcp lbpvfkwyzm (kprnlahbis, 22.38)	-	04 Apr 2024
NCT04323098 (GENEr8-3, CTgov)	Phase 3	Hemophilia A	22	BMN 270+Roxaparvovec (BMN 270 (Valoctocogene Roxaparvovec))	uopleinjog(nayggwudat) = qephgaucra xfobsnunoi (rwhtxeshvw, 3208.50) View more	-	04 Apr 2024
NCT04323098 (GENEr8-3, EAHAD2024) Manual	Phase 3	Hemophilia A	22	valoctocogene roxaparvovec+corticosteroids	fhjactnshq(zdcsvmvwdi) = rpelsolmqj lnkxrbkela (ujxijjbwee, 22.4) View more	Negative	06 Feb 2024
NCT04684940 (GENEr8-INH, EAHAD2024) Manual	Phase 1/2	Hemophilia A	-	valoctocogene roxaparvovec	pmdwgijjcq(itryoodczd) = non-serious ALT elevations (PAP1, PBP1 and PBP2) and grade 1 non-serious AEs related to CS use (moon face, acne, and weight gain). No serious or severe AEs were reported, including malignancy, FVIII inhibitor recurrence in part B, or thromboembolism. xfxfrmtmpo (nhlmlkolwy ) View more	Positive	06 Feb 2024
NCT03370913 (GENEr8-1 \| 270-301 \| BMN 270-301, CTgov)	Phase 3	Hemophilia A	144	BMN 270+Roxaparvovec	ddlcgmfzdw(zuzbrtiyxg) = mxctsnyvyq zfxsbyxvho (xxegaudarf, 6.93) View more	-	28 Nov 2023
FDA Manual	Phase 3	Hemophilia A	134	ROCTAVIAN	xsaldiikcb(kdymetusjq) = dzuzjyvihv rvchphhyct (sfymjhjsqu, 6.2) View more	Positive	29 Jun 2023

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