RegulationPriority Review (US), Breakthrough Therapy (US), Fast Track (US), Orphan Drug (US), Priority Review (CN), Orphan Drug (KR), Orphan Drug (AU)

Structure/Sequence

Sequence Code 13505337

Source: *****

Clinical Trials associated with Avalglucosidase alfa

NCT06666413

/ Not yet recruitingPhase 4

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 Date27 Mar 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 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: Kiyono, Takashi ; Komaki, Hirofumi ; Mori-Yoshimura, Madoka ; Arimori, Akihiro ; Ikeda, Mitsunobu ; Kumada, Satoko ; Inoue, Kenji ; Ohki, Hirotaka ; Mashimo, Hideaki

Background:

The 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.

Methods:

Case 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.

Results:

Results 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.

Conclusions:

Although 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.

01 Feb 2025·CLINICAL PHARMACOLOGY & THERAPEUTICS

Quantitative Systems Pharmacology‐Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease

Article

Author: Bergeler, Silke ; Geerts, Hugo ; Ortemann‐Renon, Catherine ; Kaddi, Chanchala ; Foster, Meredith ; Zaher, Atef ; An Haack, Kristina ; Zaph, Susana ; Batista, Julie L. ; van der Graaf, Piet H. ; Tao, Mengdi ; George, Kelly

Pompe disease is a rare, progressive neuromuscular disease caused by deficient lysosomal glycogen degradation, and includes both late‐onset (LOPD) and severe infantile‐onset (IOPD) phenotypes. Due to very small patient numbers in IOPD and the high phenotypic heterogeneity observed in this population, a quantitative systems pharmacology (QSP)‐based “digital twin” approach was developed to perform an in silico comparison of the efficacy of avalglucosidase alfa vs. the standard of care, in a virtual population of IOPD patients. A QSP model was developed that represents key elements of Pompe disease pathophysiology, including tissue glycogen accumulation and the elevation of the biomarker urine Hex4 in both LOPD and IOPD patients. In this approach, the QSP model was used to generate digital twins of each IOPD patient enrolled in the avalglucosidase alfa clinical program, considering their respective disease burden, demographics, and treatment history. This virtual cohort supplemented clinical observations by simulating and comparing tissue glycogen and urine Hex4 following avalglucosidase alfa treatment vs. the standard of care. The digital twin analysis supports the interpretation that the enhanced reduction in urine Hex4 observed following avalglucosidase alfa treatment is attributable to greater tissue glycogen clearance. Overall, this study provides mechanism‐based insight into avalglucosidase alfa efficacy across the phenotypic spectrum of Pompe disease and demonstrates the value of applying a QSP‐based digital twin analysis to support rare disease drug development.

01 Feb 2025·NEUROMUSCULAR DISORDERS

Management of presymptomatic juvenile patients with late-onset Pompe disease (LOPD)

Article

Author: Rodolico, C ; Porcino, M ; Musumeci, O ; Toscano, A ; Pugliese, A ; Arena, I G ; Usbergo, C ; Schoser, B

Late-onset Pompe disease (LOPD) includes patients from 1 year of age to adulthood. The vast heterogeneity in clinical manifestations and disease progression is not fully explained; however, a short disease duration and a young age seem to be good predictors of a better response to treatment. For this purpose, we investigated and followed up a cohort of 13 juvenile patients with LOPD from the clinical and therapeutic point of view, mainly pointing out the transition from presymptomatic to symptomatic status. We retrospectively collected clinical, morphological, biochemical and molecular data from 13 juvenile LOPD patients. Motor and respiratory functional data, obtained during annual follow-up visits, were analyzed. The data included serial evaluations of the Medical Research Council (MRC) scale, the 6-Minute Walking Test (6MWT), the Gait, Stairs, Gower, and Chair (GSGC) score, and seated and supine Forced Vital Capacity (FVC). Muscle Magnetic Resonance Imaging (MRI) was also included, although it was not performed in all cases. Currently, patients mean age is 18 years. All patients but one were diagnosed because of an isolated hyperCKemia: the mean age at diagnosis was 6.8 years (range 1-18). The onset of symptoms occurred from 6 months to 12 years after the diagnosis. The mean clinical follow-up duration was 9 years (range 2-18). From the genetic point of view, the most shared mutation was c.32-13T>G, found in twelve patients as compound heterozygosis. Seven patients underwent muscle biopsy, which showed vacuolar myopathy with glycogen accumulation in four of them with unspecific changes in the other three cases. Five patients developed proximal muscle weakness during the follow-up with a mild waddling gait and a positive Gowers manoeuver. Muscle MRI revealed mild hypotrophy of the thighs at the development of symptoms in four out of five cases. Four patients started alglucosidase alfa, and one avalglucosidase alfa. These five patients on Enzyme Replacement Therapy (ERT) showed motor and respiratory stability in the following years. Timely identification of emerging clinical manifestations in presymptomatic LOPD patients, as a result of careful follow-up, is essential to start prompt treatment to modify the disease natural course.

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

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

25 Jul 2024

·www.fiercepharma.com

With capacity upgrades ready to roll, Sanofi stands firm on blockbuster sales goal for RSV drug in 2024

Beyfortus, which was approved last summer, delivered 547 million euros for all of 2023. Sanofi expects the drug to become a blockbuster in 2024. While manufacturing and supply hitches have troubled the rollout of Sanofi’s key respiratory syncytial virus (RSV) drug, the near-term expected approval of a pair of new filling lines is giving the French pharma assurance that its AstraZeneca-partnered antibody Beyfortus can breach the billion-dollar threshold before the year is out. “We’re confident that Beyfortus will be a blockbuster in 2024,” Thomas Triomphe, Sanofi’s executive vice president of vaccines, said on a call with analysts Thursday. With the first Beyfortus shipment in the Northern Hemisphere expected to occur in the third quarter, Sanofi figures the last three months of the year will represent the antibody’s biggest sales season, the company’s chief financial officer, François Roger, added on the call. That fourth-quarter sales boost is likely to come courtesy of both the seasonality of RSV and the expected approval of two new filling lines in September, Roger explained. Beyfortus, which was approved last summer, delivered 547 million euros for all of 2023. Despite the antibody’s popularity out of the gate, the immunization quickly ran up against supply constraints, with the Centers for Disease Control and Prevention in October urging doctors to prioritize 100-mg doses for infants at the highest risk of RSV disease. At the time, Sanofi said it was working with AstraZeneca to “accelerate additional supply” and explore “a number of actions” to beef up the partners’ manufacturing network. Now, Sanofi is “exactly on track” with its plan to activate more filling capacity around September, Triomphe said Thursday. “We believe that there’s reason to be confident about the accelerated approval process of those lines and the fact that we’ll be able to move forward with this update for the 2024 RSV season,” he said. The executives’ comments came as Sanofi reported a roughly 10% overall sales increase to 10.75 billion euros (about $11.6 billion) in the second quarter. Much of that growth was driven by immunology superstar Dupixent, which set a new quarterly record by growing sales 29% to 3.3 billion euros ($3.6 billion) over the second three months of the year. All told, Sanofi continues to expect its immunology stalwart to generate roughly 13 billion euros in 2024, according to an earnings release. Elsewhere, Sanofi's new pharma launches grew a whopping 80.4%, bringing home total sales of 689 million euros ($746.9 million). That group includes Altuviiio in hemophilia A, Nexviazyme in Pompe disease, Rezurock in graft-versus-host disease and Sarclisa in multiple myeloma. Meanwhile, Sanofi’s vaccines sales dipped 4.8%, though revenues were overall stable once the effects of 2023’s COVID-19 sales were removed from the equation, CEO Paul Hudson said on the company’s call. Thanks to its strong performance so far in 2024, Sanofi is upgrading its earnings per share (EPS) guidance, which it now expects to be stable at constant currencies. The company previously expected EPS to decrease by a low single-digit percentage.

Drug ApprovalVaccine

100 Deals associated with Avalglucosidase alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D11744	Avalglucosidase alfa	A16AB07	DB16099

R&D Status

10 top approved records.

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

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	dvrpiqdpma(kpaxyrqfce) = dmovdhpwsh ocrdiptchi (tclirwigil, 26.0) View more	Positive	09 Apr 2024
				Avalglucosidase Alfa 10 mg/kg/every other week	dvrpiqdpma(kpaxyrqfce) = tdfzyzgcvt ocrdiptchi (tclirwigil, 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)	hkrlcljsqr(ebxevchtam) = fyhmxpuefx jroxlukafg (zqkpcaikgm, msecihgich - wbilxwrhcm) View more	-	01 Mar 2024
				Avalglucosidase Alfa (Group 1: Avalglucosidase Alfa 10 mg/kg)	hkrlcljsqr(ebxevchtam) = kmnoavwmpk jroxlukafg (zqkpcaikgm, mezdksbcxk - nzmahwpdlv) View more
NCT03019406 (Mini-COMET, SSIEM2023)	Phase 2	Glycogen Storage Disease Type II CK \| Hex4	22	Avalglucosidase alfa 20mg/kg/qow	oqifjydvvk(ykxeoczfrj) = yjhmydwffz snssjpfoub (olvtqxgtzy )	-	30 Aug 2023
				Avalglucosidase alfa 40mg/kg/qow	oqifjydvvk(ykxeoczfrj) = yhtbnefyzx snssjpfoub (olvtqxgtzy )
NCT02782741 (COMET, Pubmed \| JAMA Neurol) Manual	Phase 3	Glycogen Storage Disease Type II First line	100	Avalglucosidase alfa	olzuyzheex(ishjidwgvz) = zixthgvnci faaukgmwhq (nlnuzykbau, 1.05) View more	Positive	10 Apr 2023
				Alglucosidase alfa (switched to avalglucosidase alfa after 49 weeks)	olzuyzheex(ishjidwgvz) = eeqnbegrna faaukgmwhq (nlnuzykbau, 1.12) View more
NCT02782741 (COMET, MDA2023) Manual	Phase 3	Glycogen Storage Disease Type II	100	Avalglucosidase alfa 20 mg/kg (AVAL-arm)	betsnpoqft(qseapfwnio) = cdrewtabpa vyefajloln (sjtqdgqbyc, 9.60) View more	Positive	19 Mar 2023
				Alglucosidase alfa 20 mg/kg (Switch-arm)	betsnpoqft(qseapfwnio) = obxszafotj vyefajloln (sjtqdgqbyc, 10.42) View more
NCT02782741 (COMET, EAN2021)	Phase 3	Glycogen Storage Disease Type II	-	Avalglucosidase alfa	bofkedexta(etbntssjmg) = tqdnheognw zshvzpwqhz (mjgwqtmwid, 9.93)	Positive	18 Jun 2021
				Alglucosidase alfa	bofkedexta(etbntssjmg) = mjlqzoeori zshvzpwqhz (mjgwqtmwid, 10.40)
NCT02782741 (COMET, CTgov)	Phase 3	Glycogen Storage Disease Type II	101	PAP+Avalglucosidase Alfa (PAP: Avalglucosidase Alfa)	trjfhlifvb(pmnduggkjb) = uovuffwpzq qeawgtquvj (aggodqzyqh, mmwwrqdqvp - bvzjfpbbpv) View more	-	08 Apr 2021
				PAP+Alglucosidase Alfa (PAP: Alglucosidase Alfa)	trjfhlifvb(pmnduggkjb) = mojtpvmhww qeawgtquvj (aggodqzyqh, xijfhrzpnv - jaiwibfniu) View more
NCT01898364 (Pubmed) Manual	Phase 1	Glycogen Storage Disease Type II	24	avalglucosidase alfa (Naïve patients)	qifxytgqab(exoyqsokye) = no deaths/life-threatening serious adverse events (SAEs). wgeakbgmid (zsnuzghepl ) 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	zrgwoygiha(swuidacmqr) = kwaxatbhrm wyurbjiohp (diphydxtqj )	-	05 Apr 2016
				neoGAA 10mg/kg	zrgwoygiha(swuidacmqr) = qhnaedyzof wyurbjiohp (diphydxtqj )

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