This is a double-blind, crossover food challenge study using pork with and without α-gal in patients with a clinical diagnosis of gastrointestinal (GI)- α-gal allergy, and to investigate the pathophysiology underlying their symptoms.

Start Date17 Oct 2023

Sponsor / Collaborator

The University of North Carolina at Chapel Hill

[+1]

NCT05067868 / Active, not recruitingPhase 4

A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase Alfa (r-DNA Origin) (Replagal™) in Indian Children and Adults With Fabry Disease

The main aim of this study is to learn more about the safety profile of Replagal.
Participants will receive Replagal every 2 weeks at the clinic for about 1 year.

Start Date01 Nov 2022

Sponsor / Collaborator

Shire Plc

NCT04974749 / CompletedPhase 3

An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects With Fabry Disease

The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks. Participants will visit their study clinic many times throughout the study.

Start Date06 May 2022

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

100 Clinical Results associated with Agalsidase alfa

100 Translational Medicine associated with Agalsidase alfa

100 Patents (Medical) associated with Agalsidase alfa

1,170

Literatures (Medical) associated with Agalsidase alfa

Orphanet journal of rare diseases

Reducing agalsidase beta infusion time in Fabry patients: low incidence of antibody formation and infusion-associated reactions in an Italian multicenter study

Article

Author: Pennacchiotti, Fabio ; Pieruzzi, Federico ; Tartaglia, Luciano ; Cianci, Vittoria ; Battaglia, Yuri ; Lanzillo, Chiara ; Mignani, Renzo ; Americo, Claudio ; Sapuppo, Annamaria ; Aucella, Filippo ; Marchi, Giacomo

Abstract:

Background:

Fabry disease is a rare progressive X-linked lysosomal storage disease caused by mutations in the GLA gene that encodes α-galactosidase A. Agalsidase beta is a recombinant enzyme replacement therapy authorized in Europe at a standard dose of 1.0 mg/kg intravenously every other week at an initial infusion rate of ≤ 0.25 mg/min until patient tolerance is established, after which the infusion rate may be increased gradually. However, specific practical guidance regarding the progressive reduction in infusion time is lacking. This study investigated a new and specific protocol for reducing agalsidase beta infusion time in which a stable dosage of 15 mg/h is infused for the first four months, and the infusion rate is increased progressively from 15 to 35 mg/h for the subsequent four infusions. The shortest infusion time is reached after six months and maintained thereafter. The incidence of infusion-associated reactions (IARs) and the development of anti-drug antibodies were analyzed, and the disease burden and the clinical evolution of the disease at 12 months were evaluated.

Results:

Twenty-five of the 31 patients were naïve to enzyme or chaperone treatment at baseline and six patients had been switched from agalsidase alfa. The reduced infusion time protocol was well tolerated. Only one patient exhibited an IAR, with mild symptoms that resolved with low-dose steroids. Six patients globally seroconverted during treatment (4 with a classic phenotype and 2 with late-onset disease). All but three patients were seronegative at month 12. All patients were stable at the study’s end (FAbry STabilization indEX value < 20%); reducing infusion time did not negatively impact clinical outcomes in any patient. The perceived medical assessment showed that the quality of life of all patients improved.

Conclusions:

The study demonstrates that reducing agalsidase beta infusion time is possible and safe from both an immunogenic and clinical point of view. The use of a low infusion rate in the first months when the probability of onset of the development of antibodies is higher contributed to very limited seroconversion to antibody-positive status.

24 Jan 2024·Journal of the American Chemical Society

Linkage-Editing Pseudo-Glycans: A Reductive α-Fluorovinyl-C-Glycosylation Strategy to Create Glycan Analogs with Altered Biological Activities

Article

Author: Nagatake, Takahiro ; Saika, Azusa ; Nakahashi, Rintaro ; Kunisawa, Jun ; Moritsuka, Natsuho ; Kudo, Noyuri ; Moriyama, Takahiro ; Usui, Kazuteru ; Yoritate, Makoto ; Kusuhara, Wakana ; Chiba, Suzuka ; Takahashi, Shunji ; Ono, Shunsuke ; Tomooka, Katsuhiko ; Hirai, Go ; Tanabe, Riko ; Igawa, Kazunobu ; Kato, Naoki ; Koshino, Hiroyuki ; Matoba, Hiroaki ; Hidaka, Yu ; Kiya, Noriaki ; Ishikawa, Eri ; Yamasaki, Sho

The acetal (O-glycoside) bonds of glycans and glycoconjugates are chemically and biologically vulnerable, and therefore C-glycosides are of interest as more stable analogs. We hypothesized that, if the O-glycoside linkage plays a vital role in glycan function, the biological activities of C-glycoside analogs would vary depending on their substituents. Based on this idea, we adopted a "linkage-editing strategy" for the creation of glycan analogs (pseudo-glycans). We designed three types of pseudo-glycans with CH₂ and CHF linkages, which resemble the O-glycoside linkage in terms of bond lengths, angles, and bulkiness, and synthesized them efficiently by means of fluorovinyl C-glycosylation and selective hydrogenation reactions. Application of this strategy to isomaltose (IM), an inducer of amylase expression, and α-GalCer, which activates iNKT cells, resulted in the discovery of CH₂-IM, which shows increased amylase production ability, and CHF-α-GalCer, which shows activity opposite that of native α-GalCer, serving as an antagonist of iNKT cells.

02 Jan 2024·Angewandte Chemie (International ed. in English)

Diversification of a Novel α‐Galactosyl Ceramide Hotspot Boosts the Adjuvant Properties in Parenteral and Mucosal Vaccines

Article

Author: Davari, Mehdi D ; Vasco, Aldrin V ; Méndez, Yanira ; Wessjohann, Ludger A ; Guzmán, Carlos A ; Schulze, Kai ; Rivera, Daniel G ; Ebensen, Thomas ; Westermann, Bernhard ; Yousefi, Mohammad

Abstract:

The development of potent adjuvants is an important step for improving the performance of subunit vaccines. CD1d agonists, such as the prototypical α‐galactosyl ceramide (α‐GalCer), are of special interest due to their ability to activate iNKT cells and trigger rapid dendritic cell maturation and B‐cell activation. Herein, we introduce a novel derivatization hotspot at the α‐GalCer skeleton, namely the N‐substituent at the amide bond. The multicomponent diversification of this previously unexplored glycolipid chemotype space permitted the introduction of a variety of extra functionalities that can either potentiate the adjuvant properties or serve as handles for further conjugation to antigens toward the development of self‐adjuvanting vaccines. This strategy led to the discovery of compounds eliciting enhanced antigen‐specific T cell stimulation and a higher antibody response when delivered by either the parenteral or the mucosal route, as compared to a known potent CD1d agonist. Notably, various functionalized α‐GalCer analogues showed a more potent adjuvant effect after intranasal immunization than a PEGylated α‐GalCer analogue previously optimized for this purpose. Ultimately, this work could open multiple avenues of opportunity for the use of mucosal vaccines against microbial infections.

News (Medical) associated with Agalsidase alfa

20 Jun 2024

·www.biospace.com

Prostate Drugs May Find New Use in Reducing Risk of Dementia: Study

Pictured: Concept art showing an elderly woman overlaid with an illustration of a brain/iStock, boonstudio Drugs commonly used for the treatment of enlarged prostate may also help lower the risk of dementia with Lewy bodies, according to a new observational study from the University of Iowa Health Care. Drawing from Merative MarketScan, one of the largest proprietary U.S. healthcare claims databases, University of Iowa researchers identified more than 643,000 men who were starting treatment for benign prostatic hyperplasia with one of six common therapies. Three of these drugs fell under the class of α-1 adrenergic receptor antagonists, better known as α-blockers, which work by inhibiting the contraction of smooth muscles. The researchers focused on terazosin, which is sold by Abbott under the brand name Hytrin, doxazosin, which is marketed by Pfizer as Cardura, and alfuzosin, which is branded as Uroxatral by Sanofi. Alpha-blocker drugs are known to have a unique side effect: they activate a key enzyme involved in the production of ATP, which is the molecular form of cellular energy. In the brain, these drugs can help boost the production of energy and fight neurodegeneration, the University of Iowa scientists hypothesized. As active comparators, the researchers also followed men in Merative MarketScan who were being treated with tamsulosin or with the 5α-reductase inhibitors (5ARI) finasteride and dutasteride, all of which are not known to affect energy production in the brain. The men included in the study had no history of dementia with Lewy bodies (DLB). Participants were then followed until they exited the database or until they developed DLB. After an average follow-up of around three years, the study found that men who were initiated on the three alpha-blocker drugs were 40% less likely to develop DLB than comparators who were on tamsulosin. Compared with men whose enlarged prostates were being treated with 5ARI agents, the risk of DLBA was 27% lower in those taking alpha-blocker drugs. Both effects were statistically significant. By contrast, DLB had a statistically similar likelihood of developing in men being managed with tamsulosin and 5ARIs. These results were robust to several sensitivity analyses, according to researchers. The University of Iowa scientists published their findings Wednesday in Neurology, the medical journal of the American Academy of Neurology. “If terazosin and these similar medications can help slow this progression—if not outright preventing the disease—this would be important to preserving cognitive function and quality of life in people with DLB,” Jacob Simmering, assistant professor of internal medicine at the University of Iowa and lead study author, said in a statement. However, the researchers caution that the study is simply observational in design and only shows that there is a statistical correlation between the use of alpha-blocker drugs and the reduced risk of DLB. It does not establish causation nor does it show any directionality of association, they said. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Clinical Result

24 Apr 2024

·www.fiercepharma.com

Chiesi survey shows how Fabry drugs are failing patients, providing road map for ousting incumbents

Survey results speak to why Chiesi sees a need for Elfabrio. A Chiesi survey has found widespread unmet needs among Fabry disease patients taking drugs such as Sanofi’s Fabrazyme, pointing to how the Italian drugmaker can muscle in on the market. Chiesi won FDA approval for the Fabry disease drug Elfabrio last year. Patients already had access to Sanofi’s old stager Fabrazyme and Amicus Therapeutics' more recently approved oral challenger Galafold. But Chiesi identified a persistent unmet need, leading it to pay Protalix BioTherapeutics $50 million upfront plus $45 million to cover development costs to secure the rights to a Fabry prospect. Seeking to understand the unmet need, Chiesi and its collaborators surveyed 280 adults in the U.S. and Canada about their experience of living with Fabry. The researchers recently published the findings in the Orphanet Journal of Rare Diseases. Most, 84%, of the respondents were taking an enzyme replacement therapy (ERT) such as Fabrazyme or Takeda’s Replagal. Eleven percent of respondents were taking chaperone therapy, namely Galafold. Most of the people had unmet needs despite being on one of the approved treatments. The most common symptoms were low energy, tingling and pain in the hands or feet. Between 60% and 72% of respondents had those symptoms. More than half of the Fabry patients had ringing in their ears or hearing loss, general body pains or stomach pains. For 51% of respondents, the symptoms were hard to control or bothersome. Symptoms temporarily worsened between infusions in around half of respondents. However, only 48% of those patients reported the worsening to their physicians. In most cases, the doctor neither changed the patient’s treatment nor prescribed them a medicine to manage the symptoms. The survey results speak to why Chiesi sees a need for Elfabrio. Chiesi’s ERT performed similarly to Fabrazyme on the primary endpoint in a head-to-head phase 3 clinical trial. Elfabrio has a longer half-life, though, suggesting symptoms may be less likely to worsen between infusions, and its PEGylated design and other elements could improve tolerability and lower the rate of infusion-related reactions. Chiesi posted sales of 3 billion euros ($3.2 billion) in 2023, supported by 65% growth at the rare disease unit that sells Elfabrio. The statement lacks details of Elfabrio sales. Protalix generated $17.5 million from sales of Elfabrio to Chiesi last year.

Phase 3Clinical ResultDrug Approval

02 Oct 2023

·www.prnewswire.com

Global Enzyme Replacement Therapy (ERT) Markets, 2017-2022, 2022-2027F, 2032F: Lamzede's FDA Approval Paves the Way for a Brighter Future in Enzyme Replacement Therapies

DUBLIN, Oct. 2, 2023 /PRNewswire/ -- The "Enzyme Replacement Therapy Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering. The global enzyme replacement therapy market is expected to grow from $10.03 billion in 2022 to $10.85 billion in 2023 at a compound annual growth rate (CAGR) of 8.1%. The market is expected to reach $14.79 billion in 2027 at a CAGR of 8.1%. In the forefront of medical advancement, the enzyme replacement therapy (ERT) market stands tall, offering vital treatment avenues for individuals grappling with chronic disorders arising from enzyme deficiencies. The research report delves deep into the nuances of the ERT sector, highlighting market statistics, regional shares, and key players commanding significant market share. ERT's pivotal role in normalizing urinary GAG levels and promoting liver and spleen health is becoming increasingly recognized. Primary enzymes like agalsidase alfa - a critical tool in the combat against Fabry's disease - are reshaping the therapeutic landscape. Our comprehensive report also categorizes other prominent enzymes used, shedding light on their applications in conditions such as Gaucher disease, Pompe disease, and more. A spotlight trend in the ERT realm is the surge in product approvals. For instance, the recent FDA nod to Lamzede (velmanase alfa) in February 2023 marked a significant milestone. As the inaugural enzyme replacement therapy greenlit in the U.S for tackling the non-central neurological manifestations of alpha-mannosidosis, Lamzede offers a promising beacon of hope. Moreover, with the escalating prevalence of rare, chronic, and genetic disorders, the ERT market trajectory looks set for robust growth. The U.S National Library of Medicine projects a near-doubling of individuals aged 50 and above with a chronic ailment by 2050, further emphasizing the critical need for effective enzyme replacement therapies. The report also touches upon the market revenues, detailing contributions from key treatments like Sebelipase alfa, Elosulfase alfa, and more. Our exhaustive coverage ensures businesses and stakeholders are well-armed with insights to navigate the dynamic ERT ecosystem. In a geographical context, while North America reigned supreme in the ERT domain in 2022, the forecasted period paints a promising picture for the Asia-Pacific region. Our study covers a broad spectrum of nations from Australia to the USA, offering a global perspective on this burgeoning market. Harness the power of data-driven insights, industry trends, and strategic opportunities with our comprehensive report, and be poised at the cusp of ERT innovations and advancements. Major players in the enzyme replacement therapy market are Biomarin Pharmaceutical Inc. Leadiant Biosciences Inc. Pfizer Inc. Sanofi S.A. AbbVie Inc. Takeda Pharmaceutical Company Limited Protalix BioTherapeutics Inc. Amicus Therapeutics Inc. Johnson & Johnson Services Inc. Digestive Care Inc. Nestle Health Science Alexion Pharmaceuticals Inc. Allergan Plc. Actelion Pharmaceuticals Ltd. CANbridge Life Sciences Ltd. Chiesi Farmaceutici S.P.A. Astrazeneca Plc. Genzyme Corporation JCR Pharmaceutical Co. Ltd. TEIJIN LIMITED Horizon Therapeutics Plc. Perseo Pharma AG SmartPharm Therapeutics Inc. Key Topics Covered: 1. Executive Summary 2. Enzyme Replacement Therapy Market Characteristics 3. Enzyme Replacement Therapy Market Trends And Strategies 4. Enzyme Replacement Therapy Market - Macro Economic Scenario 4.1. COVID-19 Impact On Enzyme Replacement Therapy Market 4.2. Ukraine-Russia War Impact On Enzyme Replacement Therapy Market 4.3. Impact Of High Inflation On Enzyme Replacement Therapy Market 5. Enzyme Replacement Therapy Market Size And Growth 5.1. Global Enzyme Replacement Therapy Historic Market, 2017-2022, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Enzyme Replacement Therapy Forecast Market, 2022-2027F, 2032F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market 6. Enzyme Replacement Therapy Market Segmentation 6.1. Global Enzyme Replacement Therapy Market, Segmentation By Enzyme Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Agalsidase Alfa Agalsidase Beta Galsulfase Other Enzyme Types 6.2. Global Enzyme Replacement Therapy Market, Segmentation By Application, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Gaucher Disease Pompe Disease Fabry Disease Other Applications 6.3. Global Enzyme Replacement Therapy Market, Segmentation By Route Of Administration, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Oral Parenteral 6.4. Global Enzyme Replacement Therapy Market, Segmentation By End-Users, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Hospitals Infusion Centers Other End-Users 7. Enzyme Replacement Therapy Market Regional And Country Analysis 7.1. Global Enzyme Replacement Therapy Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion 7.2. Global Enzyme Replacement Therapy Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1904 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

100 Deals associated with Agalsidase alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D02784	Agalsidase alfa	A16AB03	DB15874

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Fabry Disease	EU	Takeda Pharmaceuticals International AG	03 Aug 2001
Fabry Disease	IS	Takeda Pharmaceuticals International AG	03 Aug 2001
Fabry Disease	LI	Takeda Pharmaceuticals International AG	03 Aug 2001
Fabry Disease	NO	Takeda Pharmaceuticals International AG	03 Aug 2001

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04840667 (CTgov)	Phase 3	Fabry Disease	17	REPLAGAL	eekeuahktb(bfqopuydtu) = pjygwozdjm lebacopyqe (rusqqkmzxp, fusbihtfgw - ndgedvdnpt) View more	-	17 Jun 2024
NCT01298141 (Pubmed \| Drugs R D) Manual	Not Applicable	Fabry Disease	167	Agalsidase alfa	zeinwhefwz(rtpbureluy) = zzroevwgfr zntncwevow (vdfrdcnrvb ) View more	Positive	01 Dec 2021
SSIEM2019	Not Applicable	Fabry Disease	138	agalsidase alfa	dfontmufbm(yntebjhdsm) = eeuonuwdsd gyzqelomwn (tqsvxjveqq ) View more	-	25 Aug 2019
SSIEM2019	Not Applicable	Fabry Disease	138	agalsidase alfa	dfontmufbm(yntebjhdsm) = fxzjvkzrfx gyzqelomwn (tqsvxjveqq ) View more	-	25 Aug 2019
SSIEM2019	Not Applicable	Fabry Disease	493	Agalsidase alfa	nhnfvzkwxx(amfoutwrfp) = oozrxjvbzw pqdydvnlcf (llwdgqfkqm ) View more	Positive	25 Aug 2019
NCT01298141 (CTgov)	Phase 3	Fabry Disease	171	Replagal (Agalsidase Alfa)	gkjpwnrybn(ogqcmfntxm) = qemgxetowf rvozjfyyqz (nrdwzyntls, osozejsjyj - xdbjmmfxtf) View more	-	15 Feb 2019
ESC2017	Not Applicable	Fabry Disease Î±-galactosidase A activity \| lyso-gb3 in leucocytes	6	Migalastat therapy	hkhyvnaohy(rsjkqlpjyf) = uteouycdse masfksvltv (kkudxrjlmf ) View more	-	27 Aug 2017
NCT01363492 (HGT-REP-084, Pubmed \| Drug Des Devel Ther) Manual	Phase 2	Fabry Disease	14	agalsidase alfa	oiqryaipps(nwzngxazyz) = xnjtgnfywq dubleidqnc (vcnmfdlhaj ) View more	Positive	25 May 2016
NCT01124643 (CTgov)	Phase 3	Fabry Disease	35	Replagal	mxwawkpgmz(rztajltoob) = zkowmrfmsw hwvbtdodeb (ljoledophs, iokmvqdobg - mjwqovzlld) View more	-	22 Aug 2014
NCT01363492 (CTgov)	Phase 2	Fabry Disease	15	Replagal (Replagal (0.2 mg/kg))	bvebmoktfq(nehndqvxww) = gllyasvebx zzjczjbsba (ffdebsaaeu, xkuczdrugy - trmsbrzxdt) View more	-	20 May 2014
NCT01363492 (CTgov)	Phase 2	Fabry Disease	15	Replagal (Replagal 0.2 mg/kg)	zksaipbwvw(mkrnsxwgsc) = hlfvqjfqdy hhsuohuqar (bgbpbgeqak, dikceqyotf - uvnimruokd) View more	-	20 May 2014
NCT01304277 (CTgov)	Phase 2	Fabry Disease	17	agalsidase alfa	adijemeedo(bckobmghbv) = pqfnrrpkev lobyogfgxb (bcvyzkacmm, pmmhdimxss - ucrhdmykmn) View more	-	25 Apr 2014

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