A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age

The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).

Start Date25 Oct 2022

Sponsor / Collaborator

BioCryst Pharmaceuticals, Inc.

NCT04933721

/ Active, not recruitingPhase 3

An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

Start Date23 Jul 2021

Sponsor / Collaborator

BioCryst Pharmaceuticals, Inc.

NCT03873116

/ CompletedPhase 3

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema

This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in Japan.

Start Date27 Dec 2018

Sponsor / Collaborator

BioCryst Pharmaceuticals, Inc.

100 Clinical Results associated with Berotralstat Hydrochloride

100 Translational Medicine associated with Berotralstat Hydrochloride

100 Patents (Medical) associated with Berotralstat Hydrochloride

Literatures (Medical) associated with Berotralstat Hydrochloride

01 Oct 2025·CLINICAL IMMUNOLOGY

A national survey of four decades of hereditary angioedema prophylaxis: Efficacy and safety of old and new drugs

Article

Author: Czaller, Ibolya ; Visy, Beáta ; Kőhalmi, Kinga Viktória ; Balla, Zsuzsanna ; Andrási, Noémi ; Farkas, Henriette ; Horváth, Hanga Réka ; Zotter, Zsuzsanna

BACKGROUND:

Hereditary angioedema (HAE) imposes a significant burden on patients due to the unpredictability of attacks. Long-term prophylaxis (LTP) aims to prevent these episodes and improve quality of life. Over the past five decades, LTP options have evolved from non-specific treatments (fresh frozen plasma, attenuated androgens, antifibrinolytics) to more targeted therapies (intravenous or subcutaneous C1 inhibitor (C1INH), lanadelumab, berotralstat).

OBJECTIVE:

To assess the use, efficacy, and safety of various LTP therapies among Hungarian HAE patients.

METHODS:

Data from the Hungarian HAE Registry (1979-2023) were analysed, including LTP usage, attack rates, side effects, and laboratory parameters.

RESULTS:

Danazol and tranexamic acid use increased over the first 30 years but has declined in the last decade. Conversely, the use of modern LTP therapies and the proportion of patients relying solely on on-demand medication have significantly increased over the past decade. Danazol and tranexamic acid reduced attack rates in 60 % of patients without significant changes in the laboratory parameters at the lowest effective doses. Furthermore, 100 % of patients on subcutaneous C1INH, lanadelumab, and berotralstat experienced fewer attacks and improved quality of life compared to pre-treatment periods, with no serious side effects or significant laboratory abnormalities.

CONCLUSION:

Modern prophylaxis usage is increasing among Hungarian HAE-C1INH patients, following the global trends. Both modern and traditional LTP options proved safe and effective in our patient population when used with appropriate monitoring.

23 Jun 2025·EXPERT OPINION ON PHARMACOTHERAPY

Hereditary angioedema treatment beyond biologics: current state of preventive and on-demand approaches and new perspectives.

Review

Author: Baroni, Irene ; Zanichelli, Andrea ; Caravella, Giuseppe ; Mansi, Marta ; Caruso, Rosario ; De Angeli, Giada

INTRODUCTION:

Hereditary angioedema (HAE) is a genetic rare condition characterized by recurrent attacks of swelling that might be potentially life-threatening. Recurrence and severity of attacks may impact psychological life, expectations and productivity. We aim to review the state-of-art of HAE preventive and on-demand treatment of non-biologics drugs, providing a perspective of their personalized use and development.

AREAS COVERED:

This literature analysis integrates international guidelines and clinical trial data on on-demand therapies and short-/long-term prophylaxis. Modern medications should be considered and personalized for HAE patients to provide benefits compatible with patients' lifestyles, preferences, and experiences. Accordingly, a new era toward oral formulations has begun starting from berotralstat, with a consistent number of drugs under development.

EXPERT OPINION:

All HAE patients should have an effective on-demand treatment available in case of attacks. Long-term prophylaxis (LTP) should be considered and individualized for all patients at every visit, following a shared decision-making approach to optimize disease control while limiting side effects. Parenteral administration of LTP is associated with treatment complexities and barriers. Oral treatment could address practical needs for HAE patients both in preventive and on-demand setting, avoiding injection-related side effects, reducing treatment burden, and improving quality of life. In the next future, significant advances in HAE therapeutics could result from gene therapy.

01 Jun 2025·Journal of Managed Care & Specialty Pharmacy

Reductions in medical visits and hospitalizations following berotralstat initiation in patients with hereditary angioedema in the United States.

Article

Author: Laliberté, François ; Johnston, Douglas T ; Christiansen, Sandra C ; Boudreau, Julien ; MacKnight, Sean D ; Spencer, Colleen ; Gillard, Patrick ; Zuraw, Bruce L ; Nestler-Parr, Sandra ; Lopez-Gonzalez, Lorena

BACKGROUND:

Hereditary angioedema (HAE) is a rare disease characterized by unpredictable recurrent, debilitating, and potentially fatal attacks of subcutaneous and submucosal tissue swelling.

OBJECTIVE:

To evaluate all-cause, angioedema-related, and HAE attack-related medical visits and hospitalizations before and after initiation of berotralstat long-term prophylaxis (LTP) for patients with HAE in the United States.

METHODS:

This retrospective pre-post analysis used Komodo's Healthcare Map claims data to identify patients who initiated berotralstat (December 2020 to December 2022). The first entry for berotralstat dispensing was defined as the index date. Inclusion criteria comprised patients aged at least 12 years at index with at least 6 months of continuous insurance eligibility pre-index and evidence consistent with HAE pre-index (International Classification of Diseases, Tenth Revision, Clinical Modification diagnosis codes D84.1, D68.2, or T78.3x; medication use [on-demand or LTP]; or presence of diagnostic HAE laboratory tests). Rates of all-cause, angioedema-related, and HAE attack-related medical visits per person-year were compared post-index vs pre-index using rate ratios with 95% CIs and P values from generalized estimating equation Poisson regression models with robust SEs. Study limitations included the inability to distinguish HAE types and the uncertainty of whether a dispensed medication was consumed or taken as prescribed.

RESULTS:

The study population included 260 patients treated with berotralstat (mean age = 39.7 years; 74.2% female). After berotralstat initiation, there were significant decreases in the rates of all-cause health care resource utilization (HRU): all-cause inpatient (IP) visits decreased by 34% (P = 0.037) and all-cause outpatient/emergency department (OP/ED) visits decreased by 14% (P = 0.005). There were also significant decreases in rates of angioedema-related HRU (IP visits: 52%, P = 0.001; OP/ED visits: 44%, P < 0.001) as well as HAE attack-related HRU (IP visits: 60%, P < 0.001; OP/ED visits: 50%, P < 0.001). Use of on-demand medications decreased significantly after berotralstat initiation (32%, P = 0.002). Results were similar among subgroups of patients defined by HAE treatment history, including patients who were LTP-experienced (n = 126) and LTP-naive but on-demand treatment-experienced (n = 67).

CONCLUSIONS:

Prophylactic treatment of HAE with berotralstat was associated with significant reductions in all-cause HRU, including decreases to angioedema-related and HAE attack-related medical visits, hospitalizations, and administration of on-demand treatment.

230

News (Medical) associated with Berotralstat Hydrochloride

29 Jun 2025

·www.globenewswire.com

BioCryst Announces Sale of European ORLADEYO® (berotralstat) Business to Neopharmed Gentili for up to $264 Million

—Neopharmed Gentili will pay BioCryst $250 million upfront for the European assets and rights related to ORLADEYO, and up to $14 million in future milestones— —BioCryst plans to use proceeds to retire all remaining term debt, which will eliminate approximately $70 million of future interest payments over remaining life of the loan— —With this transaction, BioCryst expects to end 2027 with approximately $700 million in cash and no term debt (an increase of $400 million from prior 2027 net cash guidance)— June 27, 2025 -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that it has entered into a definitive agreement to sell its European ORLADEYO® (berotralstat) business to Neopharmed Gentili for up to $264 million. BioCryst plans to use the proceeds from the transaction to retire all remaining term debt ($249 million) from Pharmakon, which will eliminate approximately $70 million of future interest payments. The company now expects to end 2027 with approximately $700 million in cash and no term debt. This represents an increase of $400 million to prior 2027 net cash guidance. “This transaction is consistent with our strategy of making ORLADEYO available to patients in Europe through the team we built, and it now provides the capital to retire our remaining term debt, leaving us with a significantly higher margin business. Our increased profitability puts us in an even stronger position to create value well into the next decade through sustainable ORLADEYO growth, our pipeline, and potential external opportunities,” said Jon Stonehouse, president and chief executive officer of BioCryst. “This transaction marks the beginning of a new path for Neopharmed Gentili, projecting the company onto the international stage and rare disease field, leveraging its deep Italian roots and welcoming a successful and experienced European team. This acquisition confirms and further strengthens our long-term growth trajectory, consolidating our position as a leading European specialty pharmaceutical company,” said Alessandro Del Bono, president and chief executive officer of Neopharmed Gentili. Upon closing, which is expected by early October, Neopharmed Gentili will pay BioCryst $250 million upfront for the European assets and rights related to ORLADEYO, and up to $14 million in future milestones associated with sales in Central and Eastern Europe. Neopharmed Gentili is acquiring BioCryst’s European organization, which will enable commercial continuity for ORLADEYO and result in at least $50 million in expected annual operating expense savings to BioCryst. Neopharmed Gentili will be responsible for all royalties related to European sales in the territory. European ORLADEYO revenues will still be credited to BioCryst in determining global royalty tiers (no royalty due on global revenues >$550 million) and count towards the cap on the OMERS royalty. BofA Securities, Inc. and TD Cowen are serving as financial advisors and Skadden, Arps, Slate, Meagher & Flom LLP is serving as legal advisor to BioCryst. Centerview Partners UK LLP is acting as exclusive financial advisor and White & Case LLP is acting as legal advisor to Neopharmed Gentili. BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. Neopharmed Gentili is a pharmaceutical company founded in Italy with historic roots in its territory, which is rapidly expanding beyond national borders thanks to a consolidated and reliable experience in M&A and licensing transactions, especially with multinational partners. Research, responsibility, and care are the pillars that guide Neopharmed Gentili’s mission to improve the health and quality of life of patients, caregivers, and healthcare personnel. Neopharmed Gentili’s ambition is to help people live long and healthy lives, acting with ethical value and scientific precision, and offering solutions with high therapeutic value. The content above comes from the network. if any infringement, please contact us to modify.

AcquisitionLicense out/in

27 Jun 2025

·www.biopharmadive.com

Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules

Today, a brief rundown of news involving Biocryst and Peter Marks, as well as updates from UCB and Altimmune that you may have missed.Biocryst said Friday it is selling the European business of its hereditary angioedema drug Orladeyoto Italys Neopharmed Gentili for $250 million upfront. The deal, which transfers BioCrysts European sales organization to Neopharmed, promises $14 million in additional milestone payments associated with sales in central and eastern Europe. Biocryst said it will use the proceeds to pay off about $249 million in term debt owed to Pharmakon Advisors, which it said will save $70 million in interest. Sale of the European organization will save an additional $50 million in annual operating costs. The company now expects to end 2027 with $700 million in cash, an increase of $400 million from previous guidance. Jonathan GardnerThe Food and Drug Administration is overstepping its authority over vaccination practices with new rules that will limit who can receive annual COVID-19 shots, according to a New England Journal of Medicine commentary authored by Peter Marks, the former head of the FDA division that reviews vaccines. Marks left the FDAs vaccine review division following disagreements with health secretary Robert F. Kennedy Jr.The FDAs new rules require any new COVID-19 booster for healthy, non-elderly adults and children must be supported by a large, randomized controlled trial evaluating death or hospitalization before approval, a policy that could make it difficult for any new vaccines to gain wide approval. Marks wrote that the FDA has taken on aspects of policymaking that have previously been in the domain of the Centers for Disease Control and Prevention. Moreover, it did so without a public discussion of the changes or consulting with outside public health experts who advise the FDA and CDC. In addition, he said the changes ignore the existing safety data for the COVID-19 shots and the continued hospitalizations and deaths caused by the virus. Jonathan GardnerExperimental liver disease drug pemvidutide met one of the two main goals of a Phase 2 trial in MASH, resolving the inflammatory disorder and stabilizing fibrosis in significantly more participants than did placebo after 24 weeks of treatment, its developer, Altimmune, said Thursday. In the trial, 59% of people who got a 1.2 milligram weekly shot of pemvidutide saw MASH resolution and no increase in fibrosis score, as did 52% of people who got a 1.8 milligram shot, the company said. Of those who received a placebo, 19% saw MASH resolution and no fibrosis increase. The drug missed on a second goal, fibrosis improvement with no worsening of MASH. Altimmune shares fell by more than half following the news. Jonathan GardnerBelgian drugmaker UCB plans to submit fenfluramine, one of the two drugs that made up the scandal-ridden fen-phen diet regimen, for approval in a new setting, now that it has positive results from a late-stage study. The company didnt release any detailed data, but said the drug, which was tested as an add-on therapy for patients with a rare genetic disorder that gravely impairs brain development, hit the studys main goal for effectiveness. It was also generally well tolerated, according to UCB, which wants to file it with regulatory agencies as soon as possible. An oral solution of fenfluramine is already cleared in the U.S. to treat Dravet syndrome and Lennox-Gastaut syndrome, two other uncommon, severe diseases characterized by persistent seizures. Jacob Bell '

VaccineClinical ResultPhase 2

26 Jun 2025

·www.businesswire.com

Pint Pharma Announces INVIMA’s Approval of ORLADEYO® (berotralstat), the First Oral Therapy for Preventing Hereditary Angioedema (HAE) Attacks in Patients Aged 12 and Older in Colombia

BOGOTA, Colombia--(BUSINESS WIRE)--Pint Pharma is pleased to announce that the Colombian National Food and Drug Surveillance Institute (INVIMA) has approved ORLADEYO®, indicated for the prophylaxis of hereditary angioedema (HAE) attacks in patients 12 years of age and older. This milestone represents a significant advancement in expanding access to innovative treatment options for individuals living with HAE across Latin America. Beyond its direct clinical benefits, the availability of ORLADEYO® in Colombia reflects our ongoing commitment to advancing research and innovation in rare disease treatment, David Muñoz, CEO of Pint Pharma. “Today’s approval marks another important step in our mission to support patients living with rare diseases throughout Latin America,” said David Muñoz, CEO of Pint Pharma. “Beyond its direct clinical benefits, the availability of ORLADEYO® in Colombia reflects our ongoing commitment to advancing research and innovation in rare disease treatment.” “The approval of ORLADEYO® by INVIMA is excellent news for the hereditary angioedema patient community in Colombia,” added Dr. Valnei Canutti, Hematologist and Chief of Scientific Affairs at Pint Pharma. “This innovative oral therapy represents a major breakthrough, offering an effective and convenient long-term prophylactic option for preventing HAE attacks.” Mauricio Botero, General Manager of Pint Pharma in Colombia, concluded: “This approval is a significant achievement for patients and highlights the strong collaboration between the medical community, the regulatory authorities and the pharmaceutical industry in Colombia to improve treatment options for rare conditions like HAE.” Hereditary angioedema is a rare genetic disorder characterized by recurrent episodes of painful and potentially life-threatening swelling of the skin, mucous membranes and internal organs. Until recently, treatment options for HAE were limited, significantly impacting patients’ quality of life and exposing them to the constant risk of unpredictable attacks. About ORLADEYO® (berotralstat) ORLADEYO® is the first and only oral therapy specifically developed to prevent hereditary angioedema (HAE) attacks in adult and pediatric patients aged 12 and older. Taken once daily, ORLADEYO® works by reducing plasma kallikrein activity, thereby preventing HAE attacks. ORLADEYO® is a product of BioCryst Pharmaceuticals, Inc., with registration, commercialization and distribution in Latin America exclusively managed by Pint Pharma. About Pint Pharma Pint Pharma is a specialty pharmaceutical company headquartered in Vienna, Austria, focused on acquiring, licensing and commercializing pharmaceutical products across Latin America. We are a community-driven organization committed to delivering innovative treatments and healthcare solutions through strategic partnerships with global leaders. Our mission is to ensure access, quality and excellence in care for patients across the region.

Drug Approval

100 Deals associated with Berotralstat Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D11673 D11674	-	B06AC	DB15982

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hereditary Angioedema	United States	BioCryst Pharmaceuticals, Inc.	03 Dec 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hereditary Angioedema Types I and II	Phase 3	Australia	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	Denmark	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	Germany	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	Israel	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	Italy	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	Spain	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	Switzerland	BioCryst Pharmaceuticals, Inc.	16 Feb 2018
Hereditary Angioedema Types I and II	Phase 3	United Kingdom	BioCryst Pharmaceuticals, Inc.	16 Feb 2018

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


GlobeNewswire Manual	Not Applicable	Hereditary Angioedema	56	ORLADEYO	ihwtjxtuol(zcgakqmgxk) = xyooonqggc bnmsedztdm (hdvallvnqe ) View more	Positive	16 May 2025
GlobeNewswire Manual	Not Applicable	Hereditary Angioedema	99	ORLADEYO (aged 12-17)	etdegshxoq(qiczqbhidi) = dhnlplcunl qqzhdlwang (eydurqnfxy ) View more	Positive	16 May 2025
ACAAI2024 Manual	Not Applicable	Hereditary Angioedema	257	Berotralstat	ehslpbvfsh(utyuqbrjru) = lvffjdolrx gaummmszvi (jvkfbujosz ) View more	Positive	24 Oct 2024
ACAAI2024 Manual	Not Applicable	Hereditary Angioedema	257	Lanadelumab	ehslpbvfsh(utyuqbrjru) = nzrdfjtjcs gaummmszvi (jvkfbujosz ) View more	Positive	24 Oct 2024
ACAAI2024 Manual	Not Applicable	Hereditary Angioedema	466	Berotralstat (patients with C1-inhibitor deficiency)	somagbltxh(xsfzdpuzvn) = nbvaqqzfzd xogcjivfpo (qletioqrhi ) View more	Positive	24 Oct 2024
ACAAI2024 Manual	Not Applicable	Hereditary Angioedema	353	Berotralstat (HAE-nl-C1-INH)	oufhbvfwur(pqdxbbmtie) = sjqbmosuut yygqfftiat (mejrjxpauh ) View more	Positive	24 Oct 2024
GlobeNewswire Manual	Not Applicable	Hereditary Angioedema	260	ORLADEYO® (berotralstat)	mjugkwuesg(cqvxnsnxcw) = hteltlikne uenjplasyc (fcdiqakgix ) View more	Positive	09 May 2024
GlobeNewswire Manual	Not Applicable	Hereditary Angioedema	260	ORLADEYO® (berotralstat) (long-term prophylaxis)	mjugkwuesg(cqvxnsnxcw) = vwanhbrtol uenjplasyc (fcdiqakgix ) View more	Positive	09 May 2024
NCT03873116 (APeX-J, Pubmed \| World Allergy Organ J)	Phase 3	Hereditary Angioedema	17	Berotralstat 150 mg	jkgruqnfxi(qffypwiwop) = scnhmptvtt sxhpxipqej (yuazxbdbso, 0.09) View more	Positive	01 Mar 2024
AAAAI2024	Not Applicable	Hereditary Angioedema	-	Lanadelumab	fqgcxoxdrc(lqrowgdtew) = klnatanpda gvhlduxssa (fhagdjoqvz )	-	23 Feb 2024
AAAAI2024	Not Applicable	Hereditary Angioedema	-	Subcutaneous C1-inhibitor (C1-INH)	fqgcxoxdrc(lqrowgdtew) = yupuapxlsr gvhlduxssa (fhagdjoqvz )	-	23 Feb 2024
AAAAI2024	Not Applicable	Hereditary Angioedema Type III First line C1-inhibitor deficiency	-	Berotralstat (HAE-C1INH)	cakyvfgecc(fhcliwtbqr) = ovhkqfjxmr rynkmqhhsd (jihclhxkoh, 0.33 - 3.33) View more	Positive	23 Feb 2024
AAAAI2024	Not Applicable		-	Berotralstat (HAE-nl-C1INH)	cakyvfgecc(fhcliwtbqr) = vxrmoavadg rynkmqhhsd (jihclhxkoh, 1.33 - >3.33) View more	Positive	23 Feb 2024
AAAAI2024	Not Applicable	Hereditary Angioedema First line	47	Berotralstat (Monthly follow-up and therapeutic monitoring by community pharmacists)	henxdumaet(xvhqtiaacq) = ifrtadaqhg upgntpnjip (qmoedynqay ) View more	Positive	23 Feb 2024

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