Efficacy Data - C1 Esterase Inhibitor| Ruconest® (C1 esterase inhibitor [recombinant])

97% or attacks needed just 1 dose of RUCONEST. 50 U/kg (max 4200 U) [open-label extension phase, n=44; 170 attacks] — 97% of attacks needed
JUST 1 DOES OF RUCONEST ^{1
1} 50 U/kg (max 4200 U) [open-label
extension phase, n=44; 170 attacks] ^{1
1}

~ 9 of 10 patients achieved symptom relief with just one dose of RUCONEST at 50 U/kg (n=44) in the primary clinical study ^{1
1}
In the primary clinical study, patients saw symptom relief in 90 minutes vs 152 minutes with placebo ^{1
1}
In the extension study, symptom relief began in 75 minutes ^{1
1}

RUCONEST stopped 93% of attacks for at least 3 days. As shown in a review of studies that included 68 people (280 attacks) — RUCONEST STOPPED 93% OF
ATTACKS FOR AT LEAST 3 DAYS* As shown in a review of studies that
included 68 people (280 attacks)^{3
3}

Most patients had no HAE symptom relapse for at least 3 days*^{3
3}

*Based on a post hoc analysis of pooled data from the randomized controlled study and open-label extension phases of 2 studies involving 127 patients aged ≥13 years who were treated with RUCONEST 50 U/kg (max 4200 U) for acute attacks of HAE. Data for 72 hours were available for 68 of 127 patients.

An acute therapy for your HAE patients

Michael Manning, MD
Diplomate American Board of Internal Medicine,
Diplomate American Board of Allergy and Immunology

RUCONEST normalized C1-INH levels in 94% of patients

RUCONEST used the optimal dose to normalize C1-INH levels in 94% of study participants^{4
4}

Maximum functional C1-INH concentrations after one dose of RUCONEST^{1
1}

Observed maximum functional C1-INH (U/kg) greater than or equal to 94% at 50 U/kg dose.

In a dose ranging study of RUCONEST, 50 U/kg was identified as the optimal dose^{5
5}
RUCONEST provides 100% bioavailability; the C1-INH gets to the site of action immediately^{5
5}

Corey a patient speaker states "I reach for my Ruconest first over other acute medicines because it works for me."

Patient permission was granted for this quote

In clinical trials, the most common adverse events were

In clinical trials, the most common adverse events were headache 9%, nausea 2%, and diarrhea 2%.

Adverse reactions in ≥2% of patients in RCT and OLE studies (N=205)
In clinical trials, the most serious adverse reaction was anaphylaxis

RUCONEST is contraindicated in patients with a history of allergy to rabbits or rabbit-derived products, and patients with a history of life-threatening immediate hypersensitivity reactions to C1 esterase inhibitor preparations, including anaphylaxis.

Rabbit allergy testing is not required.

*Included doses up to 100 U/kg.
RCT=randomized controlled trial; OLE=open-label extension.

Approximately 85% of over 600 patients were confident in self-injecting Ruconest. Injection is done over 5 minutes by a small 23-gauge butterfly needle — ~85% of over 600 patients were
confident in self injecting RUCONEST^{5
5_*} Injection is done over 5 minutes by a small
23-gauge butterfly needle

RUCONEST Patient Advocates (RPAs) are Clinical Educators who do not provide nursing services, medical advice, or treatment advice. RPAs do not sell, market, or promote RUCONEST.

*Data from 601 patients using RUCONEST show that ~85% of patients trained feel somewhat confident, mostly confident, or extremely confident on a Likert scale from 1-5 in terms of their ability to self-infuse.

Introduce your patients to Vein Finder, a tool to help them self-inject.

It is important that your patients treat HAE at the first sign of an attack. RUCONEST SOLUTIONS provides access to a variety of IV injection tools that can help support long-term self-administration of RUCONEST.

Relatively painless with proper technique

Vein Finders are available on loan for eligible RUCONEST patients:

Designed to help patients find veins and improve first-stick success
Allow patients to rotate injection sites to help keep veins healthy

picture of vein finder with stand

RUCONEST allows appropriately trained patients and their caregivers to treat attacks at home or on the go

RUCONEST is injected over
5 minutes with a small, 23-
gauge butterfly needle ^{1
1}

Prior to reconstitution,
RUCONEST does not
need to be refrigerated ^{1
1}

Prior to reconstitution,
RUCONEST has a 4-year shelf
life when stored at room
temperature (36º-77ºF) ^{1
1}

According to a National Survey of Family Growth, ~50% of pregnancies in the United States are unplanned ^{6
6}

There are no adequate and well-controlled studies for any HAE treatment in pregnant women. RUCONEST is not indicated for prophylaxis.
*Data cannot definitively establish the absence of any risk because of the small sample size and nonrandomized study design.

In a retrospective case collection study, 14 pregnant women aged 17-37 years with HAE received RUCONEST at 50 U/kg IV (max 4200 U) for acute attacks or prophylaxis during pregnancy^{1
1}^*

All women delivered at full term without complications
RUCONEST is plasma-free and eliminates the risk of human bloodborne pathogen transmission
No adverse events related to RUCONEST were reported during the pregnancy period

BODY WEIGHT	RUCONEST dose for IV injection	Volume (mL) of reconstituted solution (150 U/mL) to be administered
<84 kg	50 U/kg	Body weight in kg divided by 3
>84 kg	4200 U (2 vials)	28 mL

Do not exceed 4200 U per dose
No more than 2 doses should be administered within a 24-hour period

RUCONEST raised functional C1-INH levels to normal in >94% of patients^{4
4}

RUCONEST allows appropriately trained participants and their caregivers to treat attacks at home or on the go

Intravenous injection that can be self-administered in just 5 minutes. ^{1
1}

Maximum dose of 4200 U (2 vials) for patients weighing ≥84 kg

Enter weight (lb)

Science of HAE

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HAE challenges

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WHAT IS RUCONEST?

RUCONEST® (C1 esterase inhibitor [recombinant]) is indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness in clinical studies was not established in HAE patients with laryngeal attacks.

IMPORTANT SAFETY INFORMATION

RUCONEST is contraindicated in patients with a history of allergy to rabbits or rabbit-derived products and for patients with a history of life-threatening immediate hypersensitivity reactions, including anaphylaxis, to C1 esterase inhibitor (C1-INH) preparations.

Monitor patients for early signs of allergic or hypersensitivity reactions (including hives, generalized urticaria, tightness of the chest, wheezing, hypotension, and/or anaphylaxis). If symptoms occur, discontinue RUCONEST and administer appropriate treatment.

Serious arterial and venous thromboembolic (TE) events have been reported with plasma-derived C1-INH products. Risk factors may include the presence of an indwelling venous catheter/access device, prior history of thrombosis, underlying atherosclerosis, use of oral contraceptives or certain androgens, morbid obesity, and immobility. Monitor patients with known risk factors for TE events during and after RUCONEST administration.

Appropriately trained patients may self-administer RUCONEST upon recognition of an HAE attack. Advise patients to seek medical attention if progress of any attack makes them unable to properly prepare or administer a dose of RUCONEST. No more than 2 doses should be administered within a 24-hour period.

The serious adverse reaction reported in clinical trials was anaphylaxis. The most common adverse reactions (incidence ≥2%) were headache, nausea, and diarrhea.

Before prescribing RUCONEST, please read the full Prescribing Information including the Patient Product Information.