A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.
Study details include:
* The study duration: total study duration is approximately 64 weeks.
* Screening period of up to 8 weeks
* Treatment period of 52 weeks
* Follow-up period of 4 weeks.
* The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.

Start Date08 May 2025

Sponsor / Collaborator

Genzyme Corp.

NCT06121011

/ RecruitingNot Applicable

A Global Prospective Observational Registry of Patients With Pompe Disease

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate.
The objectives of the registry are:
* To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients
* To evaluate the long-term real-world effectiveness of Pompe disease treatments
* To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs)
* To describe the natural history of untreated Pompe disease

Start Date16 Feb 2024

Sponsor / Collaborator

Amicus Therapeutics, Inc.

NCT05734521

/ RecruitingNot Applicable

A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting

This is a worldwide, descriptive safety study collecting data on women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation, to assess the risks of avalglucsodiase alfa on pregnancy and maternal complications and adverse effects in the developing fetus, neonate, and infant.
* Outcomes in exposed infants, including growth and development, will be assessed through at least the first year of life.
* Data will be collected for approximately 10 years.

Start Date26 Oct 2022

Sponsor / Collaborator

Sanofi

100 Clinical Results associated with Avalglucosidase alfa

100 Translational Medicine associated with Avalglucosidase alfa

100 Patents (Medical) associated with Avalglucosidase alfa

Literatures (Medical) associated with Avalglucosidase alfa

01 Jun 2025·Molecular Genetics and Metabolism Reports

Successful desensitization protocol to alglucosidase and avalglucosidase alfa in a patient with infantile-onset Pompe disease

Article

Author: Sordo, Beatriz ; Segurola, Aritza ; Gendive, Miriam ; Delgado, Teresa C ; Unceta, María ; De Las Heras, Javier ; Arza, Arantza

Infantile-onset Pompe disease is a lysosomal disease characterized by cardiomyopathy and muscle weakness that, without specific treatment, is fatal within the first two years of life. We present the case of an infant who developed anaphylaxia to enzyme replacement therapy with alglucosidase-alfa. We provide a desensitization protocol to alglucosidase-alfa and, for the first time, a desensitization protocol to avalglucosidase-alfa, both delivered in a reasonable time of <6 h, and without any further reactions in the patient.

01 May 2025·Genetics in Medicine

Efficacy of transitioning from alglucosidase alfa to avalglucosidase alfa in infantile-onset Pompe disease: A single-center cohort analysis

Article

Author: Chen, Hui-An ; Lee, Ni-Chung ; Fang, Ching-Ya ; Chien, Yin-Hsiu ; Chien, Yin-Hsuan ; Yeh, Chien-Hua ; Hwu, Wuh-Liang ; Hsu, Rai-Hseng

PURPOSEAlthough alglucosidase alfa (AGL) is the standard treatment for Pompe disease, its efficacy is limited, partially because of its low mannose-6-phosphate content. Avalglucosidase alfa (AVA), a glycoengineered recombinant human acid α-glucosidase, has shown improved receptor-mediated uptake compared with AGL. Herein, we report the long-term efficacy and safety of AVA in patients with infantile-onset Pompe disease (IOPD) previously treated with AGL.METHODSThis retrospective cohort study included 9 patients with IOPD who transitioned from AGL to AVA; these patients were diagnosed and treated after being detected with IOPD via newborn screening. We analyzed the clinical status, biomarker levels (serum creatine kinase and urine glucose tetrasaccharide), and functional assessments before and after AVA treatment of these patients. Statistical analyses were performed using the Wilcoxon matched-pair signed-rank test.RESULTSDue to inadequate responses, all 9 patients received AGL at dosages exceeding the label recommendations, including one who also had tried cipaglucosidase alfa plus miglustat before transitioning to AVA. After transitioning to AVA at a dosage of 40 mg/kg every other week for a median duration of 4.9 years, the patients experienced significant reductions in biomarker levels (serum creatine kinase levels decreased by 63% and urine glucose tetrasaccharide levels decreased by 69%). Functional assessments, including pulmonary function and 6-minute walk tests, showed improvement in young patients but remained stable in older patients. Safety analyses revealed manageable infusion-associated reactions. Immune modulation therapy for antidrug antibodies was administered to 1 IOPD patient.CONCLUSIONThe transition from a high dose of AGL to AVA demonstrated sustained improvements in biomarker levels and motor function in patients with IOPD. Early initiation of AVA is crucial for patients with IOPD.

01 Mar 2025·Molecular Genetics and Metabolism Reports

Efficacy and safety of avalglucosidase alfa in Japanese patients with late-onset and infantile-onset Pompe diseases: A case series from clinical trials

Article

Author: Arimori, Akihiro ; Inoue, Kenji ; Kiyono, Takashi ; Ohki, Hirotaka ; Mori-Yoshimura, Madoka ; Ikeda, Mitsunobu ; Kumada, Satoko ; Mashimo, Hideaki ; Komaki, Hirofumi

BackgroundThe efficacy and safety of avalglucosidase alfa for Pompe disease (PD) have been demonstrated in a global Phase 3 trial (COMET) in patients with late-onset PD (LOPD) and a global Phase 2 trial (Mini-COMET) in patients with infantile-onset PD (IOPD). This case series examines the individual results of three Japanese patients enrolled in these trials.MethodsCase reports were assembled from data collected in the COMET and Mini-COMET trials. Detailed methods have been reported previously. The primary endpoint of COMET was change from baseline to week 49 in upright forced vital capacity percent (FVC %) predicted. The primary endpoint of Mini-COMET was safety and tolerability of avalglucosidase alfa. In both trials, key secondary endpoints included motor function tests and other qualitative measures of improvement. Changes in biomarkers and anti-drug antibodies were also assessed in both trials.ResultsResults for Japanese patients were representative of those from the overall populations in the COMET and Mini-COMET trials. We detail results for one Japanese patient with LOPD enrolled in the COMET trial and two Japanese patients with IOPD enrolled in the Mini-COMET trial. Importantly, avalglucosidase alfa was well tolerated at doses of both 20 mg/kg and 40 mg/kg in Japanese patients with LOPD and IOPD, respectively.ConclusionsAlthough the number of patients was small, avalglucosidase alfa provides an efficacy and safety profile in Japanese patients representative of the overall populations from key global clinical trials.

News (Medical) associated with Avalglucosidase alfa

28 Oct 2024

·www.echemi.com

Merck's RSV Antibody Faces Major Challenges vs. Sanofi's Beyfortus Exhibiting 83% Efficacy in Reducing Hospitalization

While Merck & Co. 's infant respiratory syncytial virus (RSV) antibody clesrovimab is nearing approval, sanofi also won approval last year for its RSV prevention drug beyforts in partnership with astrazeneca. This news undoubtedly brings new hope to the global healthcare industry, especially in the field of RSV vaccines. "We would very much welcome having more participants on board because it increases the scale of the competition," Thomas Triomphe, Sanofi's executive vice president for vaccines, said of the upcoming RSV competition during an analyst call on Friday. He stressed that more players mean more innovation and better healthcare solutions, which is good for the industry as a whole and for patients. Triomphe noted that Merck still has more data on clesrovimab after announcing phase 2b/3 clinical results last week. In the meantime, Sanofi remains "very confident" in the Beyfortus numbers it has released so far, he said. He mentioned that the protection data provided by Beyfortus in six months is very encouraging, as the effect has not diminished. "After six months, we were still seeing 83 percent hospitalization efficacy," he said, adding that he believed "the duration of protection from Beyfortus was significantly more favorable compared to clesrovimab." This data suggests that Beyfortus has a significant advantage in preventing RSV infection. In Merck's infant RSV study, clesrovimab successfully reduced RSV-related hospitalizations and RSV-related lower respiratory tract infection hospitalizations by 84 percent and 91 percent, respectively, over five months compared to placebo. This result is impressive and shows the great potential of clesrovimab in preventing RSV infection. At the trial's primary endpoint, clesrovimab reduced the incidence of RSV-related medical lower respiratory tract infections by 60 percent compared to placebo. This data provides further evidence of the effectiveness of clesrovimab in preventing RSV infection. Sanofi Chief Financial Officer franois-xavier Roger noted in a conference call on Friday that the inherent uncertainty of cross-trial comparisons suggested that existing Beyfortus data showed a reduction of about 75 percent in the incidence of lower respiratory tract infections with clesrovimab compared to 60 percent with Clesrovimab. This data further reinforces Beyfortus' advantage in preventing RSV infection. And, whether or not Merck brings clesrovimab into the 2025-2026 RSV season as planned, "2025 will be a year of growth for Beyfortus," the CFO stressed. He is confident that Beyfortus' sales will grow significantly as it gradually gains market share. Since it was approved last July, Beyfortus has gradually built up its sales profile. In the third quarter of 2024, sales of this preventive antibody reached 645 million euros ($699), an increase of 382% compared to the same period in 2023. Overall, sales of the drug have reached 845 million euros since the beginning of the year, which is close to the best-selling threshold. Sanofi said at the time that Beyfortus quickly fell into a supply crunch shortly after going public last year due to "higher than expected demand." To counter this, Sanofi and its partner Astrazeneca have moved quickly to ensure there is no repeat of the supply crunch in 2024. Recently they added a new filling line and shipped antibody doses ahead of the 2024 RSV season. "There are 20 markets and we don't see any capacity constraints," Triumph said on Sanofi's conference call. "We will increase supply next year and the year after to make sure there are no supply constraints." This initiative demonstrates Sanofi's confidence in the future of Beyfortus and its commitment to meeting the needs of the global market. The comments by Sanofi executives came as the French pharmaceutical company reported a 15.7 per cent rise in third-quarter sales. This growth was mainly driven by the excellent performance of its immunology product Dupixent, which brought in sales of nearly 3.5 billion euros (about $3.8 billion), an increase of about 24 percent over the third quarter of 2023. Sanofi confirmed DuPont's full-year sales target of about 13 billion euros. Elsewhere, newly launched drugs rose 67.1 percent to 727 million euros ($787), led by hemophilia drug Altuviiio, Pompeii disease treatment Nexviazyme and Rezurock for graft-versus-host disease. The strong performance of these new drugs further strengthens Sanofi's position in the field of innovative medicines. Meanwhile, Sanofi's consumer health division, Opella, saw revenue rise 7.9 percent. This growth shows that Sanofi is also making significant progress in consumer health. Earlier this week, Sanofi confirmed it had entered exclusive talks with U.S. private equity firm Clayton, Dubilier & Rice to sell a controlling 50 percent stake in its consumer healthcare business, which is valued at around 16 billion euros. This move will further strengthen Sanofi's financial strength and provide additional financial support for its future expansion.

Drug ApprovalClinical ResultVaccine

25 Oct 2024

·www.fiercepharma.com

Sanofi shrugs off potential RSV competition from Merck as Beyfortus grows sales 382%

Regardless of whether Merck enters the 2025-26 respiratory syncytial virus season with clesrovimab as planned, “2025 will be a year of growth for Beyfortus,” Sanofi's chief financial officer said Friday. Despite Merck & Co. homing in on approval for its infant respiratory syncytial virus (RSV) antibody clesrovimab, Sanofi—which last year won its own nod for its AstraZeneca-partnered RSV preventive Beyfortus—isn’t breaking a sweat.“We really welcome that there are more players to come because it increases the size of the playing field,” Thomas Triomphe, Sanofi’s executive vice president for vaccines, said of the looming RSV competition on an analyst call Friday.Triomphe noted that Merck still has more data to show on clesrovimab after unwrapping phase 2b/3 results last week. In the meantime, Sanofi remains “very confident” in the Beyfortus readouts it’s published thus far, he said.Speaking to the data on protection afforded by Beyfortus through six months, “what’s very nice about these data is that there is no waning efficacy,” Triomphe pointed out.“After six months, we still see 83% efficacy against hospitalization," he said, adding that he believes “duration of protection is significantly in favor of Beyfortus compared to clesrovimab.” In Merck’s infant RSV study, clesrovimab managed to cut RSV-related hospitalizations and RSV-related lower respiratory infection hospitalizations by 84% and 91%, respectively, versus placebo through five months.On the trial’s primary endpoint, clesrovimab reduced the incidence of RSV-associated medically attended lower respiratory infections by 60% versus placebo.Caveating against the inherent uncertainties of cross-trial comparisons, Sanofi Chief Financial Officer François-Xavier Roger pointed out on Friday’s call that the available Beyfortus data have shown higher efficacy of about 75% reduction in incidence of medically attended lower respiratory tract infection versus clesrovimab’s 60%.And, regardless of whether Merck enters the 2025-2026 RSV season with clesrovimab as planned, “2025 will be a year of growth for Beyfortus,” the CFO stressed.Since its approval last July, Beyfortus has gradually built up its sales profile.In 2024’s third quarter, the preventive antibody logged sales of 645 million euros ($699), marking a staggering 382% increase over the same period in 2023. Overall, the drug is nearing the blockbuster threshold with 845 million euros in sales since the start of the year.Shortly after last year’s launch, Beyfortus quickly ran into supply constraints thanks to “higher than anticipated demand,” Sanofi said at the time.Sanofi and its partner AZ have moved quickly to ensure there isn’t a supply crunch repeat in 2024, recently adding a new filling line and shipping doses of the antibody early to get ahead of 2024’s RSV season.“With 20 markets, we don’t see any capacity limitation,” Triomphe said on Sanofi’s call. “We’ll grow that supply for next year and the year after to make sure that there will be, again, no supply constraints.”The Sanofi execs’ Beyfortus comments came as the French pharma reported 15.7% sales growth in the third quarter.The company’s immunology stalwart Dupixent continued to deliver, bringing home sales of nearly 3.5 billion euros (around $3.8 billion), marking a roughly 24% increase over 2023’s third quarter. Sanofi confirmed its full-year sales target for Dupixent of roughly 13 billion euros.Elsewhere, new pharma launches grew 67.1% to 727 million euros ($787), with hemophilia med Altuviiio, Pompe disease treatment Nexviazyme and Rezurock for graft-versus-host disease leading the charge. Meanwhile, Sanofi saw revenues for its consumer health arm, Opella, grow 7.9%.Earlier this week, Sanofi confirmed it had entered exclusive negotiations with U.S. private equity firm Clayton, Dubilier & Rice to sell a 50% controlling stake in its consumer health business, which is valued at around 16 billion euros.

Drug ApprovalIPO

12 Sep 2024

·endpts.com

FDA revisits new structure for drug review documents, aiming to better explain its decisions

Back in 2019, as part of a program focused on improving documentation and communications for drug approvals, the FDA made a change to how it explains its decisions. At the time, academics and industry criticized the shift because it could potentially reduce the quantity and quality of the documents the agency was releasing about its drug reviews. “We are writing to express our concern with FDA’s proposal to replace individual original reviews with a new integrated review. There is a scientific publishing norm that authorship should be voluntary, and by institutionalizing group authorship, all authors of integrated reviews may not have a chance to voluntarily approve of the text in a document that bears their name,” Fiona Godlee, former editor-in-chief of the BMJ , and Clare Stone, former editor-in-chief of PLOS Medicine , wrote to the FDA in 2019. On Thursday, the FDA sought additional comment on the integrated reviews documentation, which has already been implemented for new molecular entities, original biologic applications, and select efficacy supplements, and is coming soon for other products. The agency is again seeking responses to questions like: “Comparing the integrated review to previous review documentation, is there any information you are having difficulty locating?” The agency acknowledged that the new system is an abbreviated version of what it did before. “This new integrated review document replaces the previous documentation, which included a separate review document authored by each discipline. It also replaces the multidisciplinary review (i.e., Unireview) in which each discipline provided a separate review section but within a single review document,” the FDA explained . But the regulator believes the new format will still provide “sufficient detail” concerning efficacy and its assessment of risk and risk management “as well as a clearer description of FDA’s analysis of the scientific issues raised by the application and the scientific reasoning supporting the benefit-risk determination.” By implementing the new streamlined structure for its documents, the FDA said the internal process is more coordinated, and it’s easier to reach a more diverse audience, including the public, drug sponsors, researchers and others seeking to understand the basis for FDA’s decisions. The FDA released three examples of what the new format looks like, including three integrated review documents for Sanofi’s Nexviazyme , GSK’s Rukobia and Madrigal’s Rezdiffra .

Accelerated Approval

100 Deals associated with Avalglucosidase alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D11744	Avalglucosidase alfa	A16AB07	DB16099

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Glycogen Storage Disease Type II	United States	Genzyme Corp.	06 Aug 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Glycogen Storage Disease Type II	Phase 3	Russia	Genzyme Corp.	02 Nov 2016
Glycogen Storage Disease Type II	Phase 3	Brazil	Genzyme Corp.	02 Nov 2016
Glycogen Storage Disease Type II	Phase 3	Switzerland	Genzyme Corp.	02 Nov 2016
Glycogen Storage Disease Type II	Phase 3	Argentina	Genzyme Corp.	02 Nov 2016
Glycogen Storage Disease Type II	Phase 3	Mexico	Genzyme Corp.	02 Nov 2016
Glycogen Storage Disease Type II	Phase 3	Turkey	Genzyme Corp.	02 Nov 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02032524 \| NCT01898364 (NEO1/NEO-EXT Studies, AAN2024)	Phase 1/2	Glycogen Storage Disease Type II	24	Avalglucosidase Alfa 5 mg/kg/every other week	sbpvvinhxw(lrszxusbxk) = bctegiiqra foipzpndqh (sqpevalbdw, 26.0) View more	Positive	09 Apr 2024
				Avalglucosidase Alfa 10 mg/kg/every other week	sbpvvinhxw(lrszxusbxk) = saejvbdhkk foipzpndqh (sqpevalbdw, 19.3) View more
NCT02032524 (NEO1 and NEO-EXT \| NEO-EXT, CTgov)	Phase 2	Glycogen Storage Disease Type II	19	Avalglucosidase Alfa (Group 1: Avalglucosidase Alfa 5 mg/kg)	zyqltvcwcj(ydnidlikzn) = cqnttbhxka ragdjzashg (ysmeemloqo, zphfqgipsk - vehhrktzcp) View more	-	01 Mar 2024
				Avalglucosidase Alfa (Group 1: Avalglucosidase Alfa 10 mg/kg)	zyqltvcwcj(ydnidlikzn) = ogqzpwjovs ragdjzashg (ysmeemloqo, tnbqkuizzh - vslketsgmw) View more
NCT02782741 (COMET, Pubmed \| JAMA Neurol) Manual	Phase 3	Glycogen Storage Disease Type II First line	100	Avalglucosidase alfa	(udpzwcsvjz) = gwffhgsdon zvwrmlngyl (kxehmutqfh, 1.05) View more	Positive	10 Apr 2023
				Alglucosidase alfa (switched to avalglucosidase alfa after 49 weeks)	(udpzwcsvjz) = friuoiivlf zvwrmlngyl (kxehmutqfh, 1.12) View more
NCT02782741 (COMET, MDA2023) Manual	Phase 3	Glycogen Storage Disease Type II	100	Avalglucosidase alfa 20 mg/kg (AVAL-arm)	dsarmoqvwr(nedogbblbm) = toogrcrkol qxukauftxf (cpdjyspeka, 9.60) View more	Positive	19 Mar 2023
				Alglucosidase alfa 20 mg/kg (Switch-arm)	dsarmoqvwr(nedogbblbm) = ivpjojizhi qxukauftxf (cpdjyspeka, 10.42) View more
NCT02782741 (COMET, EAN2021)	Phase 3	Glycogen Storage Disease Type II	-	Avalglucosidase alfa	(tsrxmebdpq) = nsztevwknf qtaqcpdkry (vpjbipkxdi, 9.93)	Positive	18 Jun 2021
				Alglucosidase alfa	(tsrxmebdpq) = oztqqfrdkq qtaqcpdkry (vpjbipkxdi, 10.40)
NCT02782741 (COMET, CTgov)	Phase 3	Glycogen Storage Disease Type II	101	PAP+Avalglucosidase Alfa (PAP: Avalglucosidase Alfa)	pywzykbxdi(qajbatvtgb) = vqoboauupj envaltrymv (nmsdlvnztx, wuzonxdnfe - gugfnvssxu) View more	-	08 Apr 2021
				PAP+Alglucosidase Alfa (PAP: Alglucosidase Alfa)	pywzykbxdi(qajbatvtgb) = pccyjtqhel envaltrymv (nmsdlvnztx, dlchnndmaw - iyvocjbhxq) View more
NCT01898364 (Pubmed) Manual	Phase 1	Glycogen Storage Disease Type II	24	avalglucosidase alfa (Naïve patients)	(gpwnzyjyfh) = no deaths/life-threatening serious adverse events (SAEs). ardvdwbapz (ofnlohnjww ) View more	Positive	01 Mar 2019
				Alglucosidase Alfa (Switch patients)
NCT01898364 (AAN2016)	Phase 1	Glycogen Storage Disease Type II acid α-glucosidase enzyme deficiency	-	neoGAA 5mg/kg	(dldjsvhovs) = ylowjozqry vaandfxtdo (urhvtoepth )	-	05 Apr 2016
				neoGAA 10mg/kg	(dldjsvhovs) = rkgfcnuyvj vaandfxtdo (urhvtoepth )
NCT01898364 (I4.011, AAN2016)	Phase 1	Glycogen Storage Disease Type II	24	NeoGAA 5mg/kg	ludjpxgsrk(rtfdmohnuk) = minimal changes boxhcfgqlz (aywvtfcmzl ) View more	-	05 Apr 2016
				NeoGAA 10mg/kg

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