Hereditary angioedema (HAE) new drug! Takhzyro (rana lu monoclonal antib) received priority review by the US FDA: treatment of pediatric patients aged 2-12 years!

Takhzyro is the first monoclonal antibody drug for the treatment of HAE, which specifically binds to and inhibits the activity
of plasma kallikrein enzyme.
In a Phase 3 clinical trial, Takhzyro treated pediatric patients aged 2-12 years, reducing HAE episodes by 94.
8%.

BIOON, Oct.
20, 2022 /BioValley/ --Takeda Pharmaceuticals (Takeda) today announced that the U.
S.
Food and Drug Administration (FDA) has accepted a supplemental biologics license application (sBLA)
for Takhzyro (lanadelumab, ranaluzumab injection).
The sBLA seeks to potentially expand the use of Takhzyro: for the prevention of hereditary angioedema (HAE) attacks
in pediatric patients aged 2 to under 12 years of age.

FDA has granted sBLA priority review and expects to make a review decision
in the first half of 2023.
Currently, there is no approved preventive treatment plan
for children under 6 years of age with HAE.
If approved, Takhzyro would be the first preventive treatment for
this population (children under 6 years of age).

The sBLA is based on data
from the Phase 3 SHP643-301 study (SPRING, NCT04070326).
The study is the first and only open-label Phase 3 trial
in patients with HAE under 12 years of age.
The positive results of the trial were presented
at the Congress of the European Society of Allergy and Clinical Immunology (EAACI) in 2022.
These results are consistent
with earlier studies conducted in adults and adolescents with HAE.

SHP643-301 (SPRING) is a multicenter, open-label Phase 3 study conducted in pediatric patients with HAE aged 2 to 12 years to evaluate the safety, pharmacokinetics (PK), pharmacodynamics, and clinical activity/outcome
of Takhzyro for the prevention of acute exacerbations of HAE.
During the study's 52-week treatment period, children aged 2 to under 6 years received a dose of 150 mg Takhzyro every 4 weeks (Q4W), and children aged 6 to 12 years received a dose of 150 mg every 2 weeks (Q2W
).

The study has been completed and the target
has been met.
The data showed that Takhzyro preventive treatment reduced the incidence of HAE episodes in children by an average of 94.
8%, from 1.
84 per month to 0.
08 during treatment
, compared to baseline.
During the 52-week treatment period, most patients (76.
2%) were seizure-free, with an average of 99.
5% of the days without seizures
.

No deaths or serious treatment-period adverse events (TEAEs) were reported during the study period, and no patients withdrew from the study
due to TEAE.
The most common TEAE is pain at the injection site, and most TEAE are mild or moderate in severity
.
These results are consistent
with the favorable efficacy and safety profile of Takhzyro observed in earlier studies conducted in adults and adolescents with HAE.

Takhzyro-lanadelumab mechanism of action (Image source: pharmacodia.
com)

Hereditary angioedema (HAE) is a rare genetic disorder that causes recurrent edema-swelling
in various parts of the body, including the abdomen, face, feet, genitals, hands, and throat.
Swelling can be debilitating and painful
.
Episodes of airway obstruction can lead to choking and can be life-threatening
.
With an estimated 1 in 50,000 people worldwide having HAE, the disease is often underestimated, underdiagnosed and undertreated
.

Takhzyro is the first monoclonal antibody approved for the treatment of HAE, approved for marketing in August 2018, for the routine prevention of HAE episodes
in patients aged 12 years and older.
Takhzyro is not indicated for the acute treatment
of HAE episodes.
Takhzyro is a fully human monoclonal antibody that specifically binds to and inhibits the activity
of plasma kallikrein enzyme.
The drug is produced
using recombinant DNA technology in Chinese hamster ovary cells (CHO).

Takhzyro is administered by subcutaneous injection with a half-life of 14 days in HAE patients, and after training by medical professionals, administered by the patient himself or by a caregiver, the injection
can be completed in 1 minute or less.
In terms of medication, the recommended starting dose of Takhzyro is 300 mg
every 2 weeks.
In patients receiving treatment with well-controlled disease without seizures, a dose of 300 mg every 4 weeks can be considered, especially in patients
with low body weight.
Depending on regional authorization, Takhzyro is available
as a vial of 300 mg dose or as a prefilled syringe.
(Bio Valley Bioon.
com)

U.
S.
Food and Drug Administration Accepts Takeda's Supplemental Biologics License Application for Use of TAKHZYRO® (lanadelumab-flyo) to Prevent Hereditary Angioedema (HAE) Attacks in Children 2 Years of Age and Older