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Press Release

The European Medicines Agency (EMA) Grants Accelerated Assessment for Shire’s Lanadelumab Being Evaluated for the Prevention of Attacks in Hereditary Angioedema (HAE) Patients Aged 12 Years and Older

Posted on: 28 Feb 18

-       EMA’s accelerated assessment allows Shire a shorter review period from a typical 210 to 150 days

-       Shire is on track to submit EU Marketing Authorization Application (MAA) in the coming weeks

-       HAE is a rare, genetic disorder that causes debilitating, painful and sometimes life-threatening swelling in the body1

-       Recent regulatory milestones for lanadelumab in the U.S. and E.U. reinforce Shire’s leadership in rare diseases with its innovative pipeline of 15 late-stage development programs


Cambridge, Mass. – February 27, 2018 – Shire plc (Shire) (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, announced today the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an accelerated assessment for lanadelumab (SHP643). Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with the rare, genetic disorder, hereditary angioedema (HAE).


“The EMA decision, coupled with the U.S. FDA’s recent Priority Review designation for lanadelumab, reinforces Shire’s dedication to advancing new treatment options for patients suffering from HAE,” said Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire. “As the first investigational monoclonal antibody being studied in HAE, lanadelumab utilizes a novel mechanism of action inhibiting plasma kallikrein for the prevention of HAE attacks. We look forward to further progressing lanadelumab through the regulatory review process, as we strive to bring new and innovative solutions to the patients who need them most.”


HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.[i], [ii] The condition results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat that can be can debilitating and painful.1,[iii], [iv] Attacks that obstruct the airways (asphyxiation) are potentially life-threatening.1,3,4,[v]


Shire is on track to submit its EU Marketing Authorization Application (MAA) in the coming weeks. Accelerated assessments by the CHMP of a marketing authorization filed under the centralized European procedure, reduces the amount of evaluation days required, from 210 to 150. The EMA will grant, upon request, accelerated assessment of an EU MAA if they deem the product to be of major interest for public health and therapeutic innovation.


The clinical development program for Shire's investigational HAE treatment includes data from four clinical trials, including HELP™, the pivotal Phase 3 efficacy and safety study, along with interim data from its extension study. HELP, the largest prevention study in HAE conducted to date, enrolled a total of 125 patients aged 12 years and over with type I/II HAE. The HELP study demonstrated that subcutaneous administration of 300 mg lanadelumab once every two weeks resulted in an 87% reduction in the mean frequency of HAE attacks. In addition, an exploratory endpoint, which will require further confirmatory studies, showed that during the steady state stage of the trial (day 70-182) a 91% attack reduction was achieved with 8 out of 10 patients reaching an attack free state. In this study, no treatment-related serious adverse events or deaths were reported. The most common adverse event was injection site pain (29.3% placebo vs. 42.9% combined lanadelumab arms). 


“I’m excited to see lanadelumab receive an accelerated assessment in Europe because we are one step closer to potentially having a new option to help prevent HAE attacks,” said Marcus Maurer, Prof. Dr. Med., Department of Dermatology and Allergy, Charité – Universitätsmedizin Berlin, Germany and clinical trial investigator. “As an investigator, I want to express my gratitude to the HAE patients and their families who participated in the clinical trial and the dedicated study site personnel, who have helped to advance science in a way that may transform the treatment of HAE.”


As the leading rare disease-focused biotech, Shire is deeply committed to developing treatments that help address the individual needs of rare disease patients around the world. The company has received Priority Review status from the U.S. Food and Drug Administration (FDA), Priority Review and Orphan Drug Designation from the Therapeutic Goods Administration in Australia and Priority Review from Health Canada for review of lanadelumab in HAE. In 2017, Shire received Breakthrough Therapy, Orphan Drug or Fast Track designations by the FDA for five rare disease therapies. We anticipate continued progress of key regulatory milestones for late-stage programs in Shire’s pipeline. Additional information can be found on


About Lanadelumab

Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein[vi] and is being studied as a treatment for the prevention of angioedema attacks in patients 12 years and older with HAE. Lanadelumab is formulated for subcutaneous administration, and has a half-life of approximately 14 days in patients with HAE.[vii]


Shire’s Commitment to Hereditary Angioedema
Shire is a dedicated, long-term partner to the HAE community with nearly a decade of experience supporting patients. We believe each patient deserves a right-fit approach to treatment and are committed to serial innovation. Our existing portfolio of products includes a number of therapy options to help meet the needs of appropriate patients with HAE. Beyond our focus on developing novel treatments, we provide specialized services and support offerings tailored to the HAE community. Learn more at


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Editor's Details

Mike Wood

Last updated on: 28/02/2018

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