The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

Start Date13 Jul 2022

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT05139810

/ CompletedPhase 3

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).

Start Date03 Dec 2021

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT04549922

/ CompletedPhase 2IIT

Antisense Therapy to Block the Kallikrein-kinin Pathway in COVID-19: A Phase II Randomized Controlled Trial

Up to 1/3 of all patients infected with COVID-19 can develop complications that require hospitalization. Severe pneumonia associated with acute respiratory distress syndrome (ARDS) is the most threatening and feared complication of COVID-19 infection, with mortality rates close to 50% in some groups.
Autopsies between these severe cases reveal severe capillary involvement, with signs of intense inflammatory changes, microvascular thrombosis, endothelial injury and abnormal tissue repair. The available evidence suggests that abnormal activation or imbalance in the counter-regulation of the kallikrein-kinin system may play a central role in a positive feedback cycle, leading to consequent diffuse microangiopathy. Blockade of the kallikrein-kinin system can therefore prevent deterioration of lung function by reducing inflammation, edema and microthrombosis.
The objective of this phase IIb study is to assess the preliminary effects on the oxygenation parameters of an antisense oligonucleotide that inhibits pre-kallikrein synthesis in patients with moderate to severe COVID-19.

Start Date19 Oct 2020

Sponsor / Collaborator

Associacao Beneficente Siria

[+2]

100 Clinical Results associated with Donidalorsen

100 Translational Medicine associated with Donidalorsen

100 Patents (Medical) associated with Donidalorsen

Literatures (Medical) associated with Donidalorsen

01 Aug 2025·ALLERGY

Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema

Article

Author: Treadwell, Sabrina ; Yarlas, Aaron ; Cohn, Danny M. ; Riedl, Marc A. ; Wang, Sophie ; Bordone, Laura ; Newman, Kenneth B.

ABSTRACT:

Background:

Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, frequently severe swelling that negatively impacts patients' quality of life (QoL). In the phase III OASIS‐HAE study (NCT05139810), donidalorsen reduced HAE attack rate, increased disease control, and improved QoL. Here, we report further analysis of donidalorsen's impact on QoL and other patient‐reported outcomes (PROs).

Methods:

This double‐blind, placebo‐controlled study randomized patients with HAE to donidalorsen 80 mg or placebo once every 4 (Q4W) or 8 weeks (Q8W) over 24 weeks. PROs included Angioedema (AE)‐QoL Questionnaire, Angioedema Control Test (AECT), Patient Global Impression of Severity (PGIS), and Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questions (WPAI+CIQ).

Results:

Ninety patients received donidalorsen Q4W (n = 45), donidalorsen Q8W (n = 23), or placebo (n = 22). A larger percentage of the donidalorsen Q4W group (88%) achieved clinically meaningful improvement (≥ 6‐point reduction) in AE‐QoL total score vs. placebo (45%). Both donidalorsen groups reported larger least‐squares mean (LSM) changes from baseline to week 24 vs. placebo in AE‐QoL functioning (difference: Q4W, −24.5; Q8W, −16.1), fears/shame (Q4W, −23.9; Q8W, −20.1), and nutrition (Q4W, −15.7; Q8W, −10.7) domains. Donidalorsen improved AECT scores vs. placebo (difference: Q4W, 6.0; Q8W, 4.1). A greater proportion of the donidalorsen Q4W group reported decreased disease severity vs. the placebo group (PGIS; 82% vs. 44%). Donidalorsen Q4W showed benefits vs. placebo in the presenteeism, overall work/school impairment, and activity impairment domains of the WPAI+CIQ.

Conclusions:

Donidalorsen significantly improved QoL and other PROs vs. placebo in patients with HAE.

01 Jul 2025·Journal of Allergy and Clinical Immunology-In Practice

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis

Article

Author: Lin, Tao ; Manning, Michael E ; Perego, Francesca ; Craig, Timothy ; Treadwell, Sabrina ; Newman, Kenneth B ; Jacobs, Joshua S ; Bordone, Laura ; Lumry, William R ; Yarlas, Aaron ; Riedl, Marc A ; Bernstein, Jonathan A ; Cohn, Danny M ; Banerji, Aleena ; Wedner, H James ; Gierer, Selina

BACKGROUND:

Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by episodes of tissue swelling. Donidalorsen, an investigational ligand-conjugated antisense oligonucleotide, reduces plasma prekallikrein production.

OBJECTIVE:

We report an interim analysis on safety, efficacy, quality of life (QoL), and treatment preference and satisfaction from an ongoing open-label phase 3 study (OASISplus Switch cohort, NCT05392114).

METHODS:

Patients with HAE receiving stable doses (≥12 weeks) of lanadelumab, complement protein 1 inhibitor, or berotralstat switched to donidalorsen 80 mg subcutaneously every 4 weeks, using a predefined algorithm. The primary end point was the incidence and severity of treatment-emergent adverse events. Other end points included change in HAE attack rate, angioedema-QoL score, disease control (≥10 points on the Angioedema Control Test), and treatment preference and satisfaction at week 16, compared with baseline on prior treatment.

RESULTS:

A total of 65 patients were enrolled; 32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat. At cutoff, 58 were ongoing in the study (89%). Forty-five patients (70%) reported treatment-emergent adverse events; 62% were unrelated to donidalorsen. At week 16, total HAE attack rates had decreased by 62%. Hereditary angioedema attack rates decreased by 65%, 41%, and 73%, and mean angioedema-QoL scores improved by 8.4, 9.6, and 17.1 points for patients switching from lanadelumab, C1INH, and berotralstat, respectively. More patients reported well-controlled disease (93% vs 67%), and most patients preferred donidalorsen over their prior treatment, with improved treatment satisfaction.

CONCLUSIONS:

Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred donidalorsen over their prior treatment. Further analyses are planned at week 52.

01 Dec 2024·JOURNAL OF CRITICAL CARE

Antisense therapy to block the Kallikrein-kinin pathway in COVID-19: The ASKCOV randomized controlled trial

Article

Author: Buchele, Gustavo ; Gomes, Samara P C ; Santos, Maria Adelaide Dos ; Negrelli, Karina L ; Dal-Pizzol, Felipe ; Falavigna, Maicon ; de Freitas, Flávio Geraldo ; Miranda, Tamiris A ; Germano, Almir ; Gebara, Otávio ; Rosa, Regis G ; Santucci, Eliana Vieira ; Santos, Renato H N ; Zampieri, Fernando G ; Gomes, Jackeline O ; Valeis, Nanci ; Ishihara, Luciana M ; Azevedo, Luciano C P ; Cavalcanti, Alexandre B ; Berti, Isabele Ribeiro ; Machado, Flávia R ; Schettini, Daniel Almeida ; Westphal, Glauco Adrieno ; Janiszewski, Mariano ; Cohn, Danny M ; de Matos Soeiro, Alexandre ; Laranjeira, Ligia N ; de Souza Dantas, Vicente Cés ; Veiga, Viviane C ; Damiani, Lucas P

PURPOSE:

To assess the effect of antisense therapy to block kallikrein-kinin pathway in COVID-19 patients.

MATERIAL AND METHODS:

Randomized, placebo-controlled, double blind, controlled trial enrolling hospitalized COVID-19 patients that required supplementary oxygen to sustain peripheral oxygen saturation. Key exclusion criteria included use of mechanical ventilation or vasopressors, and patients with more than 10 days since symptom onset or more than 48 h of oxygen use. Patients were randomized to either one subcutaneous dose of ISIS721744, an antisense that blocks prekallikrein, or placebo. The primary outcome was the number of days alive and free of oxygen support up to 15 days (DAFOR15). Secondary endpoints included organ failure score, need and duration of mechanical ventilation up to 15 days, and all-cause mortality at 30 days. Exploratory endpoints included physiological parameters, biomarkers, and quality of life.

RESULTS:

From October 10, 2020, to December 09, 2020, 111 patients were randomized at thirteen sites in Brazil (56 to treatment and 55 to control group). Average age was 57.5 years, and most patients were male (68.5%). There were no significant differences in DAFOR15 between groups (5.9 ± 5.2 days for the intervention arm and 7.7 ± 5.1 for the control group; mean difference - 0.65, 95% confidence intervals from -2.95 to 1.36, p = 0.520).

CONCLUSION:

Antisense therapy designed to block the kallikrein-kinin pathway did not demonstrate clinical benefits in increasing days-alive without respiratory support at 15 days in patients with COVID-19 during the first wave in 2020.

CLINICALTRIALS:

GOV IDENTIFIER:

NCT04549922.

News (Medical) associated with Donidalorsen

25 Aug 2025

·www.prnewswire.com

Orsini Selected as Exclusive Specialty Pharmacy Partner for Ionis' DAWNZERA™ (donidalorsen)

DAWNZERA is an FDA-approved prophylactic medicine for the treatment of hereditary angioedema (HAE), self-administered via subcutaneous auto-injector ELK GROVE VILLAGE, Ill., Aug. 25, 2025 /PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, announced today that it is the exclusive specialty pharmacy partner for Ionis Pharmaceuticals' DAWNZERA™ (donidalorsen), an FDA-approved prophylactic medicine to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, self-administered via subcutaneous auto-injector. Read the full Prescribing Information here. HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect more than 7,000 people in the U.S. While minor trauma or stress could trigger an attack, swelling often occurs without a known trigger. DAWNZERA, which is joining Orsini's extensive portfolio of HAE therapies, is designed to reduce patients' production of prekallikrein, which plays a key role in acute attacks of HAE. "We are honored to be trusted by Ionis as the exclusive specialty pharmacy partner for DAWNZERA and eager to demonstrate the meaningful impact that Orsini's exceptional care has on HAE patients," Darin DeCarlo, Orsini's Chief Commercial Officer, said. "For more than 14 years, Orsini has been a leader in HAE care. This collaboration underscores our mutual commitment to the HAE community and putting patients first." About Orsini Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™. Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, visit . SOURCE Orsini WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

22 Aug 2025

·pipelinereview.com

DAWNZERA™ (donidalorsen) approved in the U.S. as first and only RNA-targeted prophylactic treatment for hereditary angioedema

– DAWNZERA demonstrated significant and sustained HAE attack rate reduction and long-term disease control – – Offers longest dosing option for HAE, with dosing every 4 or 8 weeks – – Compelling profile supported by recently published switch data – – Ionis’ second independent launch in just nine months, with potential for two additional launches next year – CARLSBAD, CA, USA I August 21, 2025 I Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE. DAWNZERA 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W). HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the U.S. “DAWNZERA represents a significant advance for people living with HAE who need improved treatment options. With strong and durable efficacy, convenient administration and the longest dosing option available, we believe DAWNZERA will be the prophylactic treatment of choice for many people living with HAE. Importantly, the recently published switch data empowers patients and physicians with a roadmap for switching to DAWNZERA from other prophylactic therapies,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “At Ionis, we are dedicated to turning groundbreaking science into life-changing medicines. With the early success of our first independent launch of TRYNGOLZA® for familial chylomicronemia syndrome (FCS), and now with DAWNZERA, our second independent medicine approved in less than nine months, we are proudly delivering on that vision. To the patients, families, advocacy partners and investigators who helped make this moment a reality, we express our deepest gratitude.” The approval of DAWNZERA was based on positive results from the Phase 3 global, multicenter, randomized, double-blind, placebo-controlled OASIS-HAE study in patients with HAE. The study met its primary endpoint, with DAWNZERA Q4W significantly reducing monthly HAE attack rate by 81% compared to placebo over 24 weeks. Mean attack rate reduction increased to 87% when measured from the second dose, a key secondary endpoint. Additionally, DAWNZERA Q4W reduced moderate-to-severe HAE attacks by ~90% over 24 weeks when measured from the second dose. These results are bolstered by the ongoing OASISplus open-label extension (OLE) study, in which DAWNZERA Q8W had a similar effect as Q4W over time. DAWNZERA demonstrated 94% total mean attack rate reduction from baseline across both dosing groups after one year in the OLE. The OASISplus study also includes a switch cohort evaluating DAWNZERA Q4W in patients previously treated with lanadelumab, C1-esterase inhibitor or berotralstat for at least 12 weeks. Switching to DAWNZERA reduced mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch. A total of 84% of patients surveyed preferred DAWNZERA over their prior prophylactic treatment, citing better disease control, less time to administer and less injection site pain or reactions. Across clinical studies, DAWNZERA demonstrated a favorable safety and tolerability profile. The most common adverse reactions (incidence ≥ 5%) were injection site reactions, upper respiratory tract infection, urinary tract infection and abdominal discomfort. “As the first FDA-approved RNA-targeted therapy for HAE, DAWNZERA represents a welcome advance in therapeutic options for preventing attacks. Today’s approval gives people living with HAE and their physicians another important choice for aligning treatment with individual needs,” said Anthony J. Castaldo, CEO & chairman of the board, U.S. Hereditary Angioedema Association (HAEA) and Hereditary Angioedema International (HAEi). “People living with HAE manage this condition for all their lives, and many continue to face unpredictable, painful and dangerous breakthrough attacks even with current treatments. Durable efficacy is essential in maintaining long-term disease control,” said Marc Riedl, M.D., M.S., clinical director, U.S. HAEA Angioedema Center; University of California, San Diego; OASIS-HAE and OASISplus trial investigator. “DAWNZERA is positioned to help meet patient needs, providing substantial and sustained reduction of HAE attacks, continued improvement over time and reduced burden of treatment.” DAWNZERA will be available in the U.S. in the coming days. Ionis is committed to helping people access the medicines they are prescribed and will offer a suite of services designed to meet the unique needs of the HAE community through Ionis Every Step™. As part of Ionis Every Step, patients and healthcare providers will have access to a wide range of support and resources including dedicated support from a Patient Education Manager, assistance with the insurance approval process, information on affordability programs, access to the DAWNZERA Direct digital companion and other ongoing services and resources to help patients stay on track. Visit DAWNZERA.com for more information. Webcast Ionis will hold a webcast today at 12:15pm ET to discuss the FDA approval. Interested parties may access the webcast here . A webcast replay will be available for a limited time. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions occur, discontinue DAWNZERA and institute appropriate therapy. ADVERSE REACTIONS Most common adverse reactions (incidence ≥ 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort. Please see full Prescribing Information for DAWNZERA. About DAWNZERA™ (donidalorsen) DAWNZERA™ (donidalorsen) is approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is an RNA-targeted medicine designed to target plasma prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of HAE. For more information about DAWNZERA, visit DAWNZERA.com . About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis has marketed medicines and a leading pipeline in neurology, cardiology and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) , LinkedIn and Instagram . SOURCE: Ionis Pharmaceuticals

Clinical ResultDrug ApprovalPhase 3siRNA

22 Aug 2025

·www.pharmaceutical-technology.com

FDA approves Ionis’ donidalorsen for HAE attack prevention

The 80mg dose of Dawnzera is self-administered through a subcutaneous autoinjector. Credit: Ionis Pharmaceuticals, Inc./Business Wire. The US Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ RNA-targeted prophylactic treatment, Dawnzera (donidalorsen), for use in preventing hereditary angioedema (HAE) attacks in those aged 12 years and above. Dawnzera is designed to inhibit plasma prekallikrein, a significant activator of the inflammatory mediators that cause acute HAE attacks. This 80mg dose of the medicine is self-administered through a subcutaneous autoinjector, with patients having the option to choose between dosing every four or eight weeks. The agency’s approval was influenced by the positive outcomes from the Phase III OASIS-HAE trial. The placebo-controlled, randomised, global, multicentre, double-blind trial’s primary endpoint was achieved, with an 81% reduction in the monthly rate of HAE attacks for those treated with Dawnzera every four weeks, as compared to placebo, over 24 weeks. This reduction rose to 87% from the second dose, which was a secondary endpoint. The frequency of moderate-to-severe HAE attacks was also reduced by 90% from the second dose onwards during the same timeframe. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Further evidence of Dawnzera’s performance comes from the OASISplus open-label extension trial, where the eight-week dosing schedule demonstrated a similar impact to the four-week regimen over time. After one year, a 94% decrease in the total mean attack rate from baseline was observed across both dosing groups. HAE is an uncommon genetic condition, characterised by recurrent, severe swelling in various body parts. Ionis Pharmaceuticals CEO Brett Monia stated: “Dawnzera represents a significant advance for people living with HAE who need improved treatment options. “With strong and durable efficacy, convenient administration and the longest dosing option available, we believe Dawnzera will be the prophylactic treatment of choice for many people living with HAE.” In March 2025, Ionis entered a licensing agreement with Sobi. This agreement provided Sobi with the exclusive rights to commercialise olezarsen for the treatment of familial chylomicronaemia syndrome and severely elevated triglycerides. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

Clinical ResultPhase 3License out/inDrug Approval

100 Deals associated with Donidalorsen

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10 top approved records.

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Indication	Country/Location	Organization	Date
Hereditary Angioedema	United States	Ionis Pharmaceuticals, Inc.	21 Aug 2025

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05392114 (OASISplus, Pubmed \| Company_Website) Manual	Phase 3	Hereditary Angioedema	65	Donidalorsen (32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat.)	ijbemzghwy(hvpqeivtso) = twqwixdayi fplmctosjp (wrptoijcwj ) View more	Positive	17 Jul 2025
NCT05139810 (OASIS-HAE, CTgov)	Phase 3	Hereditary Angioedema	91	Donidalorsen (Pooled Placebo)	qsxdecowqr(zozigtbfql) = yioefigdri mrflwfxkqq (ekbwtfrehe, bmgoxbpwmx - cmruwpgyir) View more	-	06 Mar 2025
				Donidalorsen (Cohort A: Donidalorsen 80 mg)	qsxdecowqr(zozigtbfql) = ddnmfjahdt mrflwfxkqq (ekbwtfrehe, fkjlbmaows - zircvnyrgw) View more
NCT04307381 (Corporate Publications) Manual	Phase 2	Hereditary Angioedema	17	DonidalorsenDonidalorsen 80 mg SC Q4W	ggvlptcjkp(mgjevnracl) = buznemfste weumrelqkr (repkwkgzax, 7.81)	Positive	30 Oct 2024
				Donidalorsen 80 mg SC Q8WDonidalorsen 80 mg SC Q8W	qrobcmgeaf(cinbivbilq) = cqycuknouc cxmmxayams (kikjrejxph ) View more
NCT05392114 (OASISplus, ACAAI2024)	Phase 3	Hereditary Angioedema kallikrein-kinin system dysregulation	64	Donidalorsen 80 mg every-4-weeks	bizvgjbgyi(uaoddhisbr) = The percentage of patients with well-controlled symptoms (AECT ≥10) increased from 67% (baseline) to 93% (Week 17) tmgybnirfu (gacgbwjlwz ) View more	Positive	24 Oct 2024
NCT04307381 (Phase 2 OPEN-LABEL EXTENSION OF DONIDALORSEN, ACAAI2024)	Phase 2	Hereditary Angioedema plasma prekallikrein	17	Donidalorsen 80 mg Q4W	zebagufhks(rhjkotgwnf) = uqjhjijssy dzsqkxnfvk (swwumvuzht, 0.02 - 0.09) View more	Positive	24 Oct 2024
				Donidalorsen 80 mg Q8W	ebpqupokex(xdubflvrrb) = taahckzidc nwnapbjhaj (oauyswlchh )
NCT05392114 (OASISplus, ACAAI2024)	Phase 3	Hereditary Angioedema kallikrein-kinin system dysregulation	83	Donidalorsen 80 mg Q4W	spoaljvltc(xozajkdmrv) = Patients previously on placebo also reported improvements in AE-QoL total scores (24 points from OASIS-HAE baseline) sowmwqwytk (cntytfship ) View more	Positive	24 Oct 2024
				Donidalorsen 80 mg Q8W
NCT05139810 (OASIS-HAE, Pubmed \| N Engl J Med) Manual	Phase 3	Hereditary Angioedema	90	Donidalorsen 80 mg every 4 weeks	htduqtokiz(ixgeelssjq) = pxercaeptj boqcbbaxpg (nwtnhtesji, 0.27 - 0.73) View more	Positive	04 Jul 2024
				Donidalorsen 80 mg every 8 weeks	htduqtokiz(ixgeelssjq) = vrdptsbzlm boqcbbaxpg (nwtnhtesji, 0.65 - 1.59) View more
NCT05139810 (OASIS-HAE, Company_Website) Manual	Phase 3	Hereditary Angioedema	90	donidalorsen 80 mg Q4W	wtowcuyevm(oamueukrnd) = rvstikbybr dwmswxbosp (xjqdfijpog ) Met	Positive	31 May 2024
				donidalorsen 80 mg Q8W	wtowcuyevm(oamueukrnd) = aqlozrlxjm dwmswxbosp (xjqdfijpog ) Met
NCT04307381 (OASISplus, Company_Website) Manual	Phase 2	Hereditary Angioedema	83	Donidalorsen every four weeks (Open-Label Extension Cohort)	vbvmuzpvoo(sfnhmdmgfw) = None zmukulcpvb (lquwvvglhz )	Positive	31 May 2024
				Donidalorsen every eight weeks (Open-Label Extension Cohort)
NCT04307381 (ISIS 721744-CS3, Pubmed \| Allergy)	Phase 2	Hereditary Angioedema	17	Donidalorsen 80 mg Q4W	unelhbwetd(amscbawnmj) = Mean D-dimer concentrations decreased dwglxmguap (ktwxfckdce )	Positive	01 Mar 2024
				Donidalorsen 80 mg Q8W

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