RegulationAccelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China), Fast Track (United States)

Structure/Sequence

Sequence Code 4726027

Source: *****

Clinical Trials associated with Idursulfase

NCT06031259

/ Active, not recruitingPhase 2/3

An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive Impairment

The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome and cognitive impairment. Participants will receive the same treatment as in the previous studies.

Start Date05 Mar 2024

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

NCT05494593

/ WithdrawnPhase 4

An Open-label, Multicenter, Phase 4 Study to Assess the Effects of a Prophylactic Immune Tolerizing Regimen in MPS II Treatment-Naïve Patients Planned to Receive ELAPRASE Who Are at Risk of Developing Persistent Neutralizing Antibodies

The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome.
In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR.
Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously [IV], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle).
Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR.
Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).

Start Date28 Feb 2023

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

[+1]

NCT05371613

/ RecruitingPhase 2/3

A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II).
Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

Start Date21 Jul 2022

Sponsor / Collaborator

Denali Therapeutics, Inc.

100 Clinical Results associated with Idursulfase

100 Translational Medicine associated with Idursulfase

100 Patents (Medical) associated with Idursulfase

144

Literatures (Medical) associated with Idursulfase

01 Mar 2026·MOLECULAR GENETICS AND METABOLISM

Safety profile of idursulfase administered at home in patients with mucopolysaccharidosis II enrolled in the Hunter Outcome Survey

Article

Author: Guffon, Nathalie ; Audi, Jennifer ; Giugliani, Roberto ; Roberts, Jane ; Whiteman, David A H ; van der Ploeg, Ans T ; Burton, Barbara K ; Botha, Jaco ; Jones, Simon A ; Muenzer, Joseph

The Hunter Outcome Survey (HOS) collected global, real-world data on the natural history of mucopolysaccharidosis II and its treatment with intravenous idursulfase. For eligible patients, home therapy offers a convenient alternative to in-clinic therapy. Using data in HOS as of January 2023, we provide an updated assessment of the safety/tolerability profile of home therapy with idursulfase. The analysis population comprised 333 patients who had received at least one home infusion and 708 patients who had never received home therapy. Median (10th percentile [P10], 90th percentile [P90]) age at home therapy start was 8.9 (2.9, 21.1) years. Median (P10, P90) ages at latest visit were 15.5 (7.7, 29.3) years in the home therapy group and 13.9 (5.2, 29.0) years in the no home therapy group. Patients received a median (P10, P90) of 6.0 (0.8, 12.0) years of home infusions after 1.8 (0.3, 8.7) years of in-clinic therapy, and these timings varied by geographic region. The infusion-related reaction (IRR) rate was 0.11/patient-year during home therapy, 0.13/patient-year for the in-clinic period for the same patients (first 6 months excluded), and 0.05/patient-year for patients who never received home therapy (first 6 months excluded). More than 60% of IRRs were categorized as mild. The adverse event (AE) rate was lower during home therapy (0.59 AEs/patient-year) than during the in-clinic period for the same patients (0.86 AEs/patient-year). Among patients who never received home therapy, the rate was 0.60 AEs/patient-year. Deaths occurred at a rate of 2.17 deaths/100 patient-years of home therapy and 3.60 deaths/100 patient-years among patients never treated with home therapy. No deaths were deemed related to treatment. The rate of missed infusions was 0.52/year during the home therapy period compared with 1.42/year during the in-clinic period for the same patients and 0.57/year for patients who never received home therapy. Our data indicate a similar safety/tolerability profile for intravenous idursulfase administered at home and in clinic in patients with mucopolysaccharidosis II.

01 Jan 2026·JIMD reports

Female Patients With Mucopolysaccharidosis II ( MPS II ): Insights From the Hunter Outcome Survey

Article

Author: Audi, Jennifer ; Jain, Siddarth ; Scarpa, Maurizio ; Raiman, Julian ; Burton, Barbara K. ; Kampmann, Christoph ; Botha, Jaco ; Tylki‐Szymańska, Anna ; Amartino, Hernan ; Giugliani, Roberto ; Muenzer, Joseph

ABSTRACT:

Mucopolysaccharidosis II is a rare, X‐linked disease, with very few reports of affected female patients. Natural history data describe a predominantly male population, and appropriate disease characterization in female patients is lacking. This analysis explores the somatic disease burden and clinical progression of female patients with MPS II enrolled in the Hunter Outcome Survey (HOS; NCT03292887), a global disease registry. In total, 15 female patients were identified, representing 1.1% of the total patients in HOS. The median ages at first symptom onset and diagnosis were 1.8 and 3.1 years, respectively. A total of 8/14 (57.1%) of patients had cognitive impairment at the latest visit. X‐chromosome abnormalities were reported in two patients. Most patients (11/15, 73.3%) had received at least one dose of idursulfase, which was generally well tolerated; no serious adverse events during follow‐up were considered treatment‐related. Musculoskeletal and ear symptoms were present in all 14 patients with data recorded. Almost all females also experienced neurological, abdominal/gastrointestinal, and pulmonary disease, similar to the symptomatology reported in males. Most patients underwent surgery (41 procedures in 12 patients). Two participants had a male sibling with MPS II who was also enrolled in HOS. Both sibling sets had missense variants and demonstrated several differences in signs/symptoms between the male and female siblings. Notably, only the female siblings displayed cognitive impairment. This report illustrates the disease burden in female patients with MPS II, helping to inform clinicians about the likely prognosis for this extremely rare subgroup of patients.

01 Dec 2025·MOLECULAR GENETICS AND METABOLISM

Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey

Article

Author: Scarpa, Maurizio ; Kampmann, Christoph ; Link, Bianca ; Muenzer, Joseph ; Burton, Barbara K ; Jain, Siddharth ; Molter, David ; Harmatz, Paul ; Tylki-Szymańska, Anna ; Botha, Jaco ; Whiteman, David A H ; Lin, Shuan-Pei ; Raiman, Julian ; Giugliani, Roberto ; Amartino, Hernan

BACKGROUND:

Mucopolysaccharidosis II (MPS II) is a rare, progressive, X-linked lysosomal storage disease. Enzyme replacement therapy (ERT) with intravenous (IV) idursulfase has been approved for the treatment of patients with MPS II since 2005. The Hunter Outcome Survey (HOS; NCT03292887) was established as a condition of approval to monitor the long-term safety and effectiveness of idursulfase. Here, we report the final results from HOS.

METHODS:

HOS was a multicenter, long-term, observational registry that enrolled patients with a biochemically and/or genetically confirmed diagnosis of MPS II who were untreated or treated with idursulfase and/or bone marrow transplant. Patients were enrolled prospectively (alive at enrollment) and retrospectively (deceased at enrollment). For prospectively enrolled patients, it was requested that data from routine examinations were recorded after each follow-up visit and/or a minimum of every 6 months. The safety population (SP) included patients who received at least one dose of idursulfase and were alive at HOS entry. The treatment outcomes population (TOP) included patients who received at least one dose of idursulfase and were alive at HOS entry, excluding patients who received a bone marrow transplant, patients for whom an informed consent form could not be generated by the center, and patients with a missing date of birth. Safety and effectiveness endpoints were analyzed with descriptive statistics.

RESULTS:

In total, 1332 patients were enrolled in HOS. For patients in the SP (N = 1014), the median (10th percentile, 90th percentile) age at initiation of ERT with idursulfase was 5.7 (1.6, 18.1) years, ranging from 0.0 to 65.5 years. In the TOP (N = 989), a consistent and sustained decline in urinary glycosaminoglycan levels, trends of sustained improvements in walking capacity and left ventricular mass index, and reductions in liver and spleen size were observed. Treated patients also demonstrated a median increase in survival time of approximately 10 years and a 57.9 % lower risk of death compared with an unmatched cohort of untreated patients. In the SP, 691 patients (68.1 %) experienced at least one adverse event and 269 (26.5 %) experienced at least one infusion-related reaction (IRR); most were mild or moderate in severity. There was no relationship observed between anti-drug antibody status and IRR rates.

CONCLUSION:

Data from HOS, collected for over 18 years, represent the largest dataset of patients with MPS II to date. The effectiveness and safety profile of idursulfase support its use for the long-term treatment of patients with MPS II.

News (Medical) associated with Idursulfase

26 Mar 2026

·www.biopharmadive.com

FDA clears Denali drug in ‘clear step’ for rare disease biotechs

Dive Brief:Denali Therapeutics on Wednesday won Food and Drug Administration approval for the first biologic designed to cross into the brain to treat neurological symptoms of Hunter syndrome.Hunter syndrome is a rare genetic condition in which certain sugar molecules build up and damage to the skeleton as well as multiple organs including the heart and brain. The potentially life-threatening disease almost always strikes boys, and symptoms usually begin between the ages of two and four. The FDA estimates it affects about 500 Americans.People suffering from Hunter syndrome dont have enough of an enzyme that helps break down the harmful sugar molecules. Denalis Avlayah is a weekly infusion designed to deliver the enzyme to tissues and the central nervous system and reduce levels of those molecules.Dive Insight:Analysts hailed the approval as a positive sign at an agency that has given wildly different signals to rare disease drugmakers under the second Trump administration.FDA Commissioner Martin Makary has repeatedly touted initiatives designed to give the agency more flexibility in speeding potentially transformative medicines to market. But under his leadership, the FDA has blindsided investors with surprise rejections, regulatory reversals and an unusual public spat over the fate of a UniQure gene therapy for Huntingtons disease.The trend has drawn criticism from newspaper opinion pages and lawmakers, much of it aimed at Vinay Prasad, the controversial head of the office that regulates vaccines and gene therapies. Prasad is now stepping down, but the pressure on Makary remains. Makary installed a team that treated the patient-focused flexibility built into rare-disease regulation as a flaw rather a necessity, the Wall Street Journal wrote earlier this month.In the case of Avlayah, the FDA opted for an accelerated approval based on a Phase 1/2 study that showed the medicine could reduce levels of a molecule known as heparan sulfate in cerebrospinal fluid. To stay on the market, the agency can demand research showing that reduction translates into a real clinical benefit for patients. Denali already has a confirmatory trial under way to do that.Makary called the approval a milestone and appeared to be addressing critics in the FDAs statement. The FDA is capable of doing two things: one, exercising regulatory flexibility; and two, complying with our obligation under the law to approve drugs based on substantial evidence of effectiveness, he said. We will continue to do everything we can to accelerate treatments for rare diseases.The FDA cleared Avlayah for pediatric patients who weigh at least 5 kilograms, or 11 pounds, and dont yet have advanced neurological impairment. Pricing will be based on a patients weight, going up to about $811,000 a year for someone who weighs 30 kilograms, a meaningful premium over Takedas enzyme replacement therapy Elaprase, which sells for about $500,000, Stifel analyst Paul Matteis wrote in a note to clients.The FDA had originally planned to give Denali an answer on its application in January but delayed the decision date to review more data submitted by the company. Meanwhile, the agency recently rejected a gene therapy for Hunter syndrome from Regenxbio, raising some concern about whether Denali would ultimately succeed with its plan to seek approval based on the heparan sulfate biomarker.After all the setbacks for drugmakers who thought they were in step with the FDA, this is a clear step in the right direction for rare disease sponsors, Matteis wrote. This at least draws a line in the sand and shows that this FDA is still willing to be somewhat flexible.The approval is the first for Denali, a well-funded company focused on neuroscience that has seen setbacks in other research, including for ALS. As part of the approval, Denali received a priority review voucher that it can sell to another drugmaker; they regularly fetch $150 million or more. '

Priority ReviewDrug ApprovalGene Therapy

26 Mar 2026

·www.biospace.com

Denali Delivers ‘Welcome Positive’ for Rare Disease Space With FDA’s Hunter Syndrome Nod

iStock, Shutter2U Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research. The FDA has granted accelerated approval to Denali Therapeutics’ enzyme replacement therapy for Hunter syndrome, handing a much-needed win to the rare disease market. The drug will carry the brand name Avlayah. Wednesday’s approval for Avlayah delivers several firsts for the industry. The therapy is the first new medicine for Hunter syndrome in nearly 20 years, Denali said in its press announcement on Wednesday , adding that Avlayah is the first FDA-approved biotherapeutic that uses the transferrin receptor to cross the blood-brain barrier. Avlayah is also “the first product approved to address neurologic complications of Hunter Syndrome,” according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research. Full approval of Avlayah will hinge on the findings of a confirmatory trial designed to verify its clinical benefits. Denali closed Wednesday’s trading session at $22.47 apiece, up 13% from its previous closing price. The approval is also a “welcome positive” for the broader rare disease space, Stifel analysts said in a Wednesday note. Indeed, the past few months have brought a string of bad news for rare disease drugmakers, driven by inconsistencies and perceived inflexibilities at the highest levels of the FDA. One recent example is that of uniQure, which in November 2025 announced that the regulator changed its tune regarding the development of the company’s Huntington’s disease gene therapy. The agency at that time told uniQure it “no longer agrees” that data from the company’s Phase 1/2 program would be enough for a filing, despite allegedly previously signing off on the study design. The about-face triggered months of back -and- forth , playing out in the public sphere. FDA Rare Disease Leaders Call for Regulatory Consistency After Chaotic Year As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a consistent approval process that will support and further catalyze momentum. December 8, 2025 · 8 min read · Heather McKenzie Read more Around 500 patients in the U.S. have Hunter syndrome, a rare, genetic disease that predominantly affects boys and manifests as cognitive delays, behavioral issues and hearing loss. The disease is caused by mutations to the IDS gene resulting in a faulty enzyme that, in turn, leads to accumulation of toxic byproducts across multiple organs and tissues, including the brain. Avlayah works by supplying patients with enough IDS enzyme. Importantly, the drug makes use of Denali’s proprietary TransportVehicle platform, which relies on the transferrin receptor to cross the blood-brain barrier into the central nervous system. This sets it apart from Takeda’s Elaprase , another enzyme replacement therapy for Hunter syndrome, initially approved in 2006. Phase 1/2 data for Avlayah, which served as the basis for Wednesday’s approval, showed that the drug led to a 91% reduction in levels of heparan sulfate in the cerebrospinal fluid, a key disease marker that is “reasonably likely to predict clinical benefit,” according to Denali’s news release. At 24 weeks, 93% of patients treated with Avlayah had heparan sulfate levels comparable to people without Hunter syndrome. For many analysts, Avlayah’s approval derisks Denali’s technology. The FDA’s greenlight “provides a key validation for the company’s differentiated TransportVehicle chemistry for CNS delivery,” which could have promising readthrough to the rest of its pipeline, analysts at William Blair told investors on Wednesday. The firm forecasts peak U.S. Avlayah sales of over $250 million in the late 2030s, with worldwide revenue approaching $1 billion. Stifel agreed with this assessment, calling the nod a “key milestone” for Denali. On Feb. 10, the FDA rejected REGENXBIO’s gene therapy for Hunter syndrome—a decision that put Denali in a good spot —citing concerns over the company’s use of natural history controls to gauge the efficacy of its products, a design feature that REGENXBIO said the FDA had previously agreed to. The FDA also pointed to problems with REGENXBIO’s eligibility criteria for patient enrollment and the validity of the biomarker it used. REGENXBIO, which had thought it had addressed the FDA’s concerns prior to receiving the complete response letter last month, has stated its plans to request a Type A meeting with the FDA to discuss the rejection and a possible resubmission. Rare diseases Denali’s Hunter Syndrome Candidate in the Spotlight After REGENXBIO Rejection The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month. March 16, 2026 · 6 min read · Ben Hargreaves Read more

Drug ApprovalGene TherapyAccelerated Approval

25 Mar 2026

·allsci.com

Denali wins first product approval for Avlayah in Hunter syndrome

The US FDA has granted accelerated approval to Avlayah (tividenofusp alfa-eknm), an enzyme replacement therapy developed by Denali Therapeutics for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II, or MPS II). The decision marks the first FDA-approved biologic designed to cross the blood-brain barrier and the first new treatment option for MPS II in nearly 20 years. It is also Denali’s first approved product, validating the company’s proprietary TransportVehicle platform, which engineers therapeutic proteins to traverse the blood-brain barrier via transferrin receptor-mediated transcytosis. The FDA also awarded Denali a Rare Pediatric Disease Priority Review Voucher in connection with the approval. Indication specifics Avlayah is indicated for the treatment of neurologic symptoms in pediatric patients weighing at least 5 kg with Hunter syndrome, provided treatment is initiated prior to advanced neurologic impairment. The drug carries Breakthrough Therapy, Fast Track, and Orphan Drug designations. Because the approval was granted under the accelerated pathway, it was based on a surrogate endpoint — reduction of cerebrospinal fluid heparan sulfate (CSF HS) — considered reasonably likely to predict clinical benefit. Continued approval may depend on verification of clinical benefit in a confirmatory trial. AVLAYAH is not recommended for use in combination with other enzyme replacement therapies. The label includes a boxed warning for hypersensitivity reactions, including anaphylaxis. The approval rests on data from a Phase I/II international, multi-center, open-label trial enrolling 47 pediatric patients aged 0.3 to 13 years, including both enzyme replacement therapy-naïve and previously treated individuals. By week 24, Avlayah demonstrated a 91% reduction in CSF HS from baseline (95% CI: 89%, 92%), and 93% of treated patients (41 of 44) achieved CSF HS levels within the range observed in individuals without Hunter syndrome. The most common adverse reaction was infusion-related reactions. Results were published in The New England Journal of Medicine in January 2026. Denali is conducting the ongoing Phase II/III COMPASS study, a randomized trial comparing Avlayah to idursulfase, to generate confirmatory evidence and support global regulatory submissions. Disease context Hunter syndrome affects approximately 500 individuals in the United States and 2,000 worldwide. It is caused by deficiency of the iduronate 2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in tissues including the brain. The neurologic manifestations — cognitive decline, behavioral changes, loss of speech and mobility — affect nearly all patients and have represented the central unmet need in MPS II for decades. Since 2006, the only FDA-approved therapy has been idursulfase (Elaprase), marketed by Takeda, which addresses somatic symptoms but does not cross the blood-brain barrier. Avlayah, composed of the IDS enzyme fused to Denali’s TransportVehicle platform, is engineered to bind the transferrin receptor on brain endothelial cells, enabling delivery to both peripheral tissues and the central nervous system. It joins a small but growing field of blood-brain barrier-crossing biologics; pabinafusp alfa (Izcargo), developed by JCR Pharmaceuticals using a different transferrin receptor-targeting approach, was approved in Japan in 2021 but has not received FDA or EMA approval. Gene therapy candidates, including RGX-121 from REGENXBIO, remain in clinical development. For Denali, the approval provides a commercial foundation and a proof of concept for the TransportVehicle platform, which the company said has five additional programs in clinical development across neurodegenerative diseases and lysosomal storage disorders. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Drug ApprovalFast TrackOrphan DrugClinical StudyBreakthrough Therapy

100 Deals associated with Idursulfase

External Link

KEGG	Wiki	ATC	Drug Bank
D04499	Idursulfase	A16AB09	DB01271

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Mucopolysaccharidosis II	United States	Takeda Pharmaceuticals U.S.A., Inc.	24 Jul 2006

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cognitive Dysfunction	Phase 3	United States	Shire Plc	24 Mar 2014
Cognitive Dysfunction	Phase 3	Australia	Shire Plc	24 Mar 2014
Cognitive Dysfunction	Phase 3	Mexico	Shire Plc	24 Mar 2014
Cognitive Dysfunction	Phase 3	Spain	Shire Plc	24 Mar 2014
Cognitive Dysfunction	Phase 3	United Kingdom	Shire Plc	24 Mar 2014

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01506141 (CTgov)	Phase 1/2	Cognitive Dysfunction \| Mucopolysaccharidosis II	15	Idursulfase-IT (Idursulfase-IT 10 mg)	rnmpsizjdu = nygfvnmoml fngjuufwss (eestpuokdl, cebvafhkac - qequeafove) View more	-	06 Aug 2025
				Idursulfase-IT (Idursulfase-IT 30 mg)	rnmpsizjdu = sgiadphnym fngjuufwss (eestpuokdl, qqgzxtlucz - rmnjogjfvb) View more
NCT02412787 (HGT-HIT-094, CTgov)	Phase 2/3	Cognitive Dysfunction \| Mucopolysaccharidosis II	56	Idursulfase-IT	jyiqgqhjpe = fcrrzsastc taxrgwlmfh (aooiqizoux, trjuyzyazb - fvzmzfpthm) View more	-	19 Jun 2025
NCT05058391 (CTgov)	Phase 4	Mucopolysaccharidosis II	5	Elaprase	kfkqeemtir = kkabkollvi yspvwegyjv (gywojdxftd, atdaqiizhd - qkkbgoypsv) View more	-	23 Jan 2025
NCT02055118 (Pubmed)	Phase 2/3	Mucopolysaccharidosis II	49	Idursulfase-IT 10 mg	rdnafqcszf(cecsdvpokg): treatment difference = 3.0 (95% CI, -7.3 to 13.3), P-Value = 0.5669	Negative	02 Aug 2022
				No idursulfase-IT treatment
NCT02412787 \| NCT02055118 (HGT-HIT-094, Pubmed)	Phase 2/3	Mucopolysaccharidosis II	56	Idursulfase-IT 10 mg	yvxyjphnbb(eygjdrqwdr) = Idursulfase-IT was generally well tolerated over the cumulative treatment period of up to 36 months. Overall, 25.0% of patients had at least one adverse event (AE) related to idursulfase-IT; most treatment-emergent AEs were mild in severity. Of serious AEs (reported by 76.8% patients), none were considered related to idursulfase-IT treatment. There were no deaths or discontinuations owing to AEs zxbxqzpeat (qozxzikhfw ) View more	-	02 Aug 2022
				IV idursulfase (0.5 mg/kg)
NCT02055118 (AIM-IT, CTgov)	Phase 2/3	Cognitive Dysfunction \| Mucopolysaccharidosis II	58	Elaprase (No IT Treatment)	gjomcjweee(ndwiekcmkf) = egzdakgnzj tifowlsdnw (wmkenxrjpr, 4.22) View more	-	13 Dec 2018
				Idursulfase (IT Treatment)	gjomcjweee(ndwiekcmkf) = adshgggtrs tifowlsdnw (wmkenxrjpr, 3.14) View more
NCT02055118 (PipelineReview) Manual	Phase 2/3	Cognitive Dysfunction \| Mucopolysaccharidosis II	48	SHP609	cpfrpkrltq(ksesxpjdgw) = not meet rwmskuwkrj (cjbwhdzzgj ) View more	Negative	19 Dec 2017
NCT00920647 (Pubmed \| Genet Med) Manual	Phase 1/2	Mucopolysaccharidosis II	16	idursulfase-IT (no treatment or 10-mg, 30-mg)	tfugcaxsul(dibwpfgluo) = No serious adverse events related to idursulfase-IT were observed. vjunjrbxhe (ywtpknxany )	Positive	01 Jan 2016
				idursulfase-IT (1-mg)
NCT00920647 (CTgov)	Phase 1/2	Cognitive Dysfunction \| Mucopolysaccharidosis II	16	Control	uejurvejqj = ycquxfthgl eruvuotdhz (bcszzrcxuq, swlspkgjnx - bcwxgddwpr) View more	-	16 May 2014
NCT00630747 (CTgov)	Phase 2/3	Mucopolysaccharidosis II	94	Idursulfase	nczafnbqap(fhwqjlgltv) = bonpglmyrv mpvbndpics (bffwzdbmjc, 1.059) View more	-	17 Mar 2014

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