# Shire plc CHMP Recommends EU Marketing Authorisation of lanadelumab

- Link: https://www.directorstalkinterviews.com/shire-plc-chmp-recommends-eu-marketing-authorisation-of-lanadelumab/412760250
- Published: 2018-10-19T13:32:25+01:00
- Author: Charlotte Edwards

**Shire plc** (LON: SHP, NASDAQ: SHPG), the leading global biotechnology company
focused on rare diseases, today announced that the European Medicines Agency’s (
EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion
recommending the granting of marketing authorisation of lanadelumab injection for
routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients
aged 12 years and older. If approved, lanadelumab will be a first-of-its-kind, fully
human monoclonal antibody (mAb) available in the EU that inhibits the activity of
plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to help prevent
attacks.1

HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000
people worldwide.2,3 The condition results in recurring attacks of oedema (swelling)
in various parts of the body, including the abdomen, face, feet, genitals, hands
and throat, that can be debilitating and painful. Laryngeal attacks that obstruct
the airways are potentially life-threatening due to the risk of asphyxiation.2,4,5

“This positive opinion marks an important step towards providing adults and adolescents
living with HAE in Europe a first-of-its-kind monoclonal antibody treatment option
to help prevent attacks,” said Andreas Busch, Ph.D., Executive Vice President, Head
of Research and Development at Shire. “We are excited about the future potential
of lanadelumab in helping to address the needs of those living with this chronic
and unpredictable disease.”

The positive opinion is supported by data from the Phase III HELP (Hereditary Angioedema
Long-term Prophylaxis) Study™, the largest randomised controlled prevention study
conducted to date in HAE, which evaluated the efficacy and safety of subcutaneously
administered lanadelumab versus placebo over 26 weeks in 125 patients 12 years of
age or older with HAE.6

Lanadelumab was previously granted accelerated assessment by the EMA, reducing the
number of evaluation days required from 210 to 150. The CHMP’s positive opinion 
will be reviewed by the European Commission, which has the authority to grant marketing
authorisation in the EU.

Lanadelumab received approval for the prevention of HAE attacks in patient 12 years
and older in the U.S. on 23 August 2018 and Canada on 19 September 2018, under the
brand name TAKHZYRO™.1,7

**Supportive Data**
 The CHMP submission was based on outcomes of the HELP Study
™, a multicentre, randomised, double-blind, placebo-controlled parallel group trial
that evaluated the efficacy and safety of subcutaneously administered lanadelumab
versus placebo over 26 weeks in 125 patients 12 years of age or older with HAE.6

The primary endpoint of the HELP Study™ was the number of investigator-confirmed
HAE attacks over the entire 26-week study duration. Lanadelumab demonstrated that
subcutaneous injections every two or four weeks reduced the mean monthly number 
of attacks across all three lanadelumab treatment arms studied: 300 mg every two
weeks, 300 mg every four weeks, and 150 mg of lanadelumab every four weeks. At 300
mg every two weeks, lanadelumab reduced the number of mean monthly HAE attacks by
87% vs. placebo (adjusted P<0.001).6

Overall, each lanadelumab treatment arm demonstrated statistically significant attack
rate reductions compared with placebo for all secondary efficacy endpoints (adjusted
P<0.001 for all comparisons). Patients taking lanadelumab 300 mg every 2 weeks had
83% fewer moderate or severe attacks and 87% fewer attacks that needed on-demand
treatment. A pre-specified, exploratory analysis showed that 44% of patients (n=
27) receiving lanadelumab 300 mg every two weeks had zero attacks compared to placebo(
2%, n=41) for the 26-week treatment period.1 Additionally, in a post hoc sensitivity
analysis of the steady state period from Day 70 to Day 182, 77% of patients (n=26)
treated with lanadelumab in the same dosage arm of the trial were attack-free compared
to placebo (3%, n=37).8

Relevant Safety Information from the HELP Study™
 No serious Treatment Emerging 
Adverse Events (TEAEs) or deaths were reported. The most common TEAE was HAE attack.
The most commonly reported adverse events, excluding events related to HAE attacks,
were injection site pain (42.9%), followed by upper respiratory infection (23.8%),
headache (20.2%) and injection site erythema (9.5%). The majority of TEAEs were 
mild to moderate in severity. TEAEs resulted in discontinuation for 1 patient from
the 300 mg every four weeks lanadelumab arm (ALT/AST elevation) and 2 from the placebo
arm (tension headache, HAE attack).

**About lanadelumab**
 Lanadelumab, a fully human monoclonal antibody that specifically
binds and decreases plasma kallikrein activity, is currently being evaluated by 
the EMA for routine prevention of recurrent attacks of hereditary angioedema (HAE)
in patients aged 12 years and older. Lanadelumab is formulated for subcutaneous 
administration and has a half-life of approximately two weeks in patients with HAE.
6 Lanadelumab is intended for self-administration or administration by a caregiver,
only after training by a healthcare professional.

For full U.S. and Canada Prescribing Information, including the approved indications
and important safety information, please visit US Prescribing Information and the
Canadian Product Monograph.

**Shire’s Commitment to Hereditary Angioedema**
 Shire is a dedicated, long-term
partner to the HAE community with a decade of experience supporting patients. We
are committed to serial innovation in HAE and our portfolio of products includes
a number of therapy options to help meet the individual needs of those living with
the disease. Beyond our focus on developing novel treatments, we provide specialised
services and support offerings tailored to the HAE community.

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