Alnylam Launches ‘Play by Play’ Initiative to Raise Awareness of ATTR-CM and Support Earlier Diagnosis
Alnylam Pharmaceuticals, Inc., a company focused on RNA interference (RNAi) therapeutics, has launched “AMVUTTRA® ‘Play by Play,’” a new U.S. educational initiative designed to raise awareness of transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM). The initiative features former professional athletes living with ATTR-CM, their caregivers and healthcare professionals who have been involved in their care.
Through firsthand experiences and clinical perspectives, the program is intended to help people recognize potential signs of ATTR-CM and encourage conversations with healthcare professionals. The initiative focuses on the challenges that can arise when symptoms are attributed to more common cardiovascular conditions, aging or other explanations, potentially delaying further investigation.
“Play by Play” begins with the story of Pete Shaw, a former professional football player who is living with ATTR-CM. Shaw shares his experience alongside his wife and caregiver, Kari, and one of his cardiologists, Rajeev Mohan, M.D. Their accounts describe the progression from initially experiencing symptoms that were not recognized as signs of ATTR-CM to pursuing additional testing, receiving a diagnosis and beginning treatment with AMVUTTRA.
The initiative is designed around the idea that managing ATTR-CM involves a team, with patients, caregivers and healthcare professionals each playing important roles throughout the diagnostic and treatment journey.
A Former Athlete Shares His ATTR-CM Experience
Shaw’s story serves as the first patient experience featured in the “Play by Play” initiative. As a former professional football player, he was accustomed to dealing with physical discomfort and continuing through demanding circumstances.
According to Shaw, this mindset contributed to difficulty recognizing that changes in his health could represent a serious underlying condition. Symptoms that might otherwise prompt medical evaluation were initially associated with his history of playing football or with getting older.
His wife, Kari, encouraged him to seek additional medical attention. That decision ultimately contributed to further evaluation and testing, leading to a diagnosis of ATTR-CM.
Shaw now receives AMVUTTRA under the supervision of his physician four times a year through subcutaneous injection. His experience is being shared as part of Alnylam’s educational effort to encourage people to pay attention to changes in their health and discuss concerning symptoms with healthcare professionals.
“I spent my career learning how to read the field and react to what was happening around me. But when it came to my own health, I didn’t recognize that the symptoms I was experiencing could be something serious and progressive,” Shaw said.
“That’s why I’m sharing my story. If it helps even one person pay closer attention to changes in their health, ask questions, or start a conversation with their doctor, then sharing my experience is worth it,” he added.
Understanding ATTR-CM
ATTR-CM is a serious and progressive form of transthyretin-mediated amyloidosis in which misfolded transthyretin, or TTR, proteins accumulate in the heart.
TTR is a protein produced primarily in the liver. Alnylam In transthyretin amyloidosis, abnormal TTR proteins can form amyloid deposits that accumulate in tissues and organs. When these deposits affect the heart, they can interfere with cardiac structure and function.
Recognizing ATTR-CM can be challenging because symptoms may overlap with those of more common cardiovascular conditions. Potential symptoms include shortness of breath, fatigue, swelling in the lower legs and irregular heart rhythms.
The hereditary form of the disease can also be associated with genetic variants. Family medical history may therefore provide an additional clue when physicians are evaluating a patient with a pattern of symptoms that could be consistent with ATTR-CM.
In Shaw’s case, the gene variant he carries is more common among people of African descent. This aspect of his story highlights the importance of considering family history and relevant clinical information when assessing patients for potential ATTR-CM.
The “Play by Play” initiative aims to use patient experiences such as Shaw’s to encourage greater awareness of symptoms and support discussions between patients and healthcare professionals.
Importance of Recognizing Potential Warning Signs
One of the central themes of the initiative is the challenge of identifying ATTR-CM before disease progression becomes more advanced.
Symptoms such as breathlessness, fatigue or swelling can have many possible causes. Patients may therefore not immediately associate them with a rare amyloidosis condition. Likewise, physicians may need to consider multiple possible explanations before determining whether additional testing for ATTR-CM is appropriate.
The educational campaign is intended to help address this awareness gap by presenting the experiences of people who have gone through the diagnostic process.
Rajeev Mohan, M.D., Director of the Advanced Heart Failure Program at Scripps Clinic and a clinical cardiologist and heart failure specialist, is featured alongside Shaw in the initiative.
Mohan said early diagnosis is an important consideration in managing ATTR-CM because identifying the disease can allow patients and physicians to discuss treatment options and establish an appropriate management strategy.
“Early diagnosis is one of the most important factors in the management of this disease,” Mohan said. “Pete’s journey underscores why awareness matters. An earlier diagnosis allows patients to have timely discussions about available treatment options and develop a management plan tailored to their needs.”
AMVUTTRA and RNAi-Based TTR Silencing
AMVUTTRA, also known as vutrisiran, is an RNAi therapeutic designed to reduce the production of transthyretin.
RNA interference is a biological mechanism that can be used to silence specific genes involved in disease processes. AMVUTTRA is designed to target TTR messenger RNA, reducing the production of TTR at its source.
According to Alnylam, AMVUTTRA provides rapid knockdown of TTR through RNAi-mediated gene silencing.
The Alnylam treatment is administered by a healthcare professional as a subcutaneous injection four times per year. This dosing schedule is part of the treatment experience described by Shaw in the “Play by Play” initiative.
Alnylam said AMVUTTRA is the only U.S. FDA-approved treatment indicated for both ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis, or hATTR-PN, in adults.
The Alnylam company is using the “Play by Play” campaign to explain the role of TTR silencing while also emphasizing the importance of conversations between patients and their care teams.
Tolga Tanguler, Chief Commercialization Officer at Alnylam, connected the campaign’s football theme with the company’s RNAi approach.
“The power of football is built play by play, and strength in silencing with AMVUTTRA is built on the same principle: making precise interventions where they matter,” Tanguler said.
He added that Alnylam views precise targeting of disease drivers as an important element of its RNAi approach.
Bringing Patients, Caregivers and Physicians Together
The “Play by Play” campaign is structured around the idea that ATTR-CM management involves multiple participants. Patients experience the symptoms and make decisions about seeking medical attention, caregivers can help identify changes and encourage further evaluation, and healthcare professionals provide diagnostic expertise and treatment guidance.
Shaw’s experience illustrates how these roles can intersect. His wife, Kari, encouraged him to pursue additional medical attention after noticing concerns about his health. His cardiology team then helped investigate the symptoms and connect the clinical findings with the possibility of ATTR-CM.
Mohan continues to play a role in Shaw’s disease management and is included in the campaign to provide a healthcare professional’s perspective on diagnosis and treatment.
By combining these viewpoints, Alnylam intends for the initiative to provide a broader picture of what navigating ATTR-CM can involve.
The campaign also seeks to encourage patients to be active participants in conversations about unexplained or persistent symptoms. People experiencing possible symptoms are encouraged to discuss them with a healthcare professional rather than attempting to diagnose themselves.
FDA-Approved Indications for AMVUTTRA
AMVUTTRA is approved by the U.S. Food and Drug Administration for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
It is also approved for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
The approval reflects AMVUTTRA’s role as a TTR-lowering therapy for two manifestations of transthyretin-mediated amyloidosis.
However, as with any prescription medicine, treatment decisions are made by healthcare professionals based on the individual patient’s condition and medical circumstances.
Important Safety Information
Treatment with AMVUTTRA causes a decrease in serum vitamin A levels. Patients receiving the treatment are advised to take vitamin A supplementation at the recommended daily allowance.
The prescribing information cautions that higher-than-recommended doses should not be used in an attempt to normalize serum vitamin A levels during treatment because serum vitamin A measurements do not necessarily reflect the body’s total vitamin A stores.
Patients who develop ocular symptoms that could indicate vitamin A deficiency, including night blindness, should be referred to an ophthalmologist.
In clinical studies of patients with hATTR-PN, the most common adverse reactions reported among patients receiving AMVUTTRA included pain in an extremity, arthralgia, dyspnea and decreased vitamin A. Reported rates were 15%, 11%, 7% and 7%, respectively.
In a study involving patients with ATTR-CM, Alnylam reported that no new safety issues were identified.
Patients and healthcare professionals should consult the full U.S. prescribing information for additional information about AMVUTTRA, including its approved uses, warnings and safety information.
Educational Focus of the Initiative
The “Play by Play” program is intended as an educational resource rather than a substitute for medical evaluation. Its central message is that recognizing possible symptoms and discussing them with a healthcare professional can be an important step in the diagnostic process.
Through Shaw’s experience, the campaign illustrates how symptoms that appear unrelated or attributable to other causes may warrant further discussion, particularly when they persist or occur alongside other potential signs of cardiovascular disease.
Alnylam plans to use the initiative to provide personal stories and expert perspectives that can help people learn more about ATTR-CM and the diagnostic journey.
The Alnylam company also provides additional educational information about ATTR-CM and AMVUTTRA through the campaign’s dedicated online resources.
Shaw and Mohan have been compensated for their participation in the initiative.
As ATTR-CM remains a complex condition that can be difficult to recognize, the campaign places emphasis on awareness, communication and collaboration between patients, caregivers and healthcare professionals. Through these perspectives, Alnylam is seeking to highlight the importance of understanding potential disease signs and pursuing appropriate medical conversations.
AMVUTTRA remains a prescription treatment, and individuals experiencing symptoms that may be associated with ATTR-CM should consult a qualified healthcare professional for evaluation and guidance. The campaign’s patient stories represent individual experiences and do not necessarily reflect the experiences or outcomes of all people receiving treatment.
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) demonstrates strength in RNAi-powered transthyretin (TTR) silencing, delivering rapid knockdown of TTR at the source of disease to address the underlying cause of transthyretin amyloidosis (ATTR). In the HELIOS-B Phase 3 study, AMVUTTRA reduced the risk of all-cause mortality and recurrent CV events compared to placebo in the overall and monotherapy populations by 28.2% and 32.8%, respectively, through 36 months.
It is the only TTR silencer approved for both the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) and cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in countries globally. AMVUTTRA is administered once quarterly via subcutaneous injection.
About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating, and fatal disease caused by pathogenic transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant. It is estimated that more than 500,000 people worldwide live with ATTR, with ~80% remaining undiagnosed.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines.
Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Alnylam Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health.