LIVING WITH A RARE DISEASE OR DEVASTATING CONDITION
VICTOR, LIVING WITH PNH
Achieved with Rady Children’s Hospital for fastest genetic-testing diagnosis with a treatment option in less than 14 hours
On IDEA Pharma’s 2020 Pharmaceutical Innovation Index for adding value to our pipeline
PRIX GALIEN AWARD
For outstanding drug innovation in the Specialist Care category
Pioneering Breakthroughs for the Rare CommunityBeyond developing medicines, Alexion is advancing the field of diagnostics, how we engage patients for the input into our development programs and the way in which we share clinical trial results. Click on the “+” icon on each image to learn more about a few examples of this.
As the pandemic set in, Alexion worked quickly to protect the integrity and continuity of our clinical trials. We also built our own model tracking COVID-19 spread that provided greater detail than commercially available models.
Alexion has started a Biobank initiative that’s dedicated to streamlining our biospecimen procurement and archiving process and creating an accessible library that’s unique to rare disease.
Our work with Caelum Biosciences on a Phase 3 clinical program to evaluate CAEL-101 continued in 2020. This first-in-class therapy, in combination with standard-of-care therapy, aims to address impaired organ function due to a rare condition known as AL amyloidosis.
Accurate and early diagnoses can help save lives and prevent years of unnecessary suffering. People with rare diseases often wait years to receive a proper diagnosis and many never receive one at all. Expecting physicians to correctly diagnose something they may have never encountered before is incredibly difficult.
In response, we’ve developed RARE ANSWERS™ in collaboration with leading children’s hospitals and technology and data-science companies. RARE ANSWERS is a system of innovative and sustainable tools designed to help shorten the diagnostic journey and time to initiating treatment of children with a rare disease. These tools aim to deliver actionable data to physicians by analyzing phenotypic and genomic data and incorporating precision software, medical information and clinical insights. Alexion has acted as a steward of this program for five years as part of our commitment to the needs of all people with rare diseases.
Creating and Maintaining
One of the ways in which Alexion works to better understand patient needs is through the patient registries we have established to gather patient input and perspectives. Patient registries are conducted over the long term and are a key source of real-world data that allow us to better understand the disease, what patients experience in their daily lives, and how the disease affects their functional capacity and quality of life. They help us gain real insights from physicians and patients that enable us to improve patient outcomes and help shape the medical practice to ensure that patients’ needs are met.
In 2020, Alexion achieved the following:
Acquired Portola Pharmaceuticals. In this strategic transaction, we added the medicine, ANDEXXA® (coagulation factor Xa (recombinant), inactivated-zhzo) (marketed as ONDEXXYA® in Europe), which rapidly reverses factor Xa (FXa) inhibitor-related uncontrolled or life-threatening bleeds.
Completed Our Acquisition of Achillion Pharmaceuticals. This added two clinical-stage Factor D inhibitors to our growing pipeline.
Grew Our Development Pipeline. We have in place more than 20 programs and the potential for 10 launches by 2023, including treatments for both Wilson disease and ALS.