A PHASE 1, OPEN-LABEL, RANDOMIZED, CROSSOVER, SINGLE DOSE, PIVOTAL BIOEQUIVALENCE STUDY TO COMPARE TAFAMIDIS FREE ACID TABLET AND COMMERCIAL TAFAMIDIS FREE ACID CAPSULE ADMINISTERED UNDER FASTED CONDITIONS IN HEALTHY ADULT PARTICIPANTS

The purpose of this clinical trial is to compare the amount of tafamidis in the blood of healthy adult participants after taking two different forms of tafamidis by mouth.

Start Date20 Dec 2024

Sponsor / Collaborator

Pfizer Inc.

NCT06393465

/ Active, not recruitingNot Applicable

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records

Start Date15 Jun 2024

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Tafamidis

100 Translational Medicine associated with Tafamidis

100 Patents (Medical) associated with Tafamidis

633

Literatures (Medical) associated with Tafamidis

01 May 2025·AMERICAN JOURNAL OF CARDIOLOGY

Impact of Sodium-Glucose Cotransporter-2 Inhibitors on Cardiovascular Outcomes in Transthyretin Amyloid Cardiomyopathy

Article

Author: Fradley, Michael G ; Dominic, Paari ; Byer, Stefano H ; Grewal, Udhayvir S ; Sivamurugan, Aravinthasamy

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive form of heart failure characterized by restrictive hemodynamics and high morbidity. Tafamidis remains the only approved treatment, but its limited availability underscores the need for alternative therapies. Sodium-glucose co-transporter 2 inhibitors (SGLT2i), shown to improve outcomes in heart failure with preserved ejection fraction (HFpEF), may offer therapeutic benefits in ATTR-CM due to shared pathophysiological mechanisms. A retrospective cohort analysis was conducted using data from the TriNetX Global Research Network. Patients with wild-type transthyretin amyloid cardiomyopathy (wtATTR-CM) were divided into 2 groups: those receiving SGLT2i therapy and those not treated with SGLT2i. Propensity score matching balanced 19 baseline characteristics. Clinical outcomes, including heart failure exacerbations, all-cause hospitalizations, acute kidney injury (AKI), and all-cause mortality, were assessed over 5 years. The study included 623 matched patients in each cohort. SGLT2i therapy was associated with significant reductions in heart failure exacerbations (hazard ratio [HR] 0.64, 95% confidence interval [CI]: 0.48 to 0.86, p <0.01), all-cause hospitalizations (HR 0.72, 95% CI: 0.58 to 0.91, p <0.01), and AKI (HR 0.53, 95% CI: 0.35 to 0.79, p <0.01). A trend toward reduced all-cause mortality (HR 0.83, 95% CI: 0.63 to 1.08, p = 0.165) was observed, though not statistically significant. In conclusions, SGLT2 inhibitors demonstrate significant potential to reduce morbidity and healthcare utilization in wt-ATTR-CM patients, with promising trends toward improved survival. These findings highlight SGLT2i as a viable adjunct to existing therapies like tafamidis and warrant further investigation through prospective randomized trials.

21 Apr 2025·Expert Review of Pharmacoeconomics & Outcomes Research

A retrospective observational analysis of the real-world care pathway of people with hereditary transthyretin amyloidosis with polyneuropathy in Italy

Article

Author: D’Amelio, Marco ; Martini, Nello ; Ronconi, Giulia ; Dondi, Letizia ; Piccinni, Carlo ; Esposito, Immacolata ; Addesi, Alice ; Gnesi, Marco ; Dondi, Leonardo ; Calabria, Silvia ; Cosentino, Nicola ; Dell’Anno, Irene ; Maggioni, Aldo Pietro

BACKGROUND:

This retrospective observational study described the epidemiology and the burden on the Italian healthcare service (SSN) of patients with polyneuropathy (PN) associated with hereditary transthyretin amyloidosis (ATTRv).

RESEARCH DESIGN AND METHODS:

From the Fondazione ReS (Ricerca e Salute) administrative healthcare database (~5.5 million inhabitants in 2021), patients were identified as having ATTRv-PN in 2021 if they had received treatments for ATTRv-PN under SSN reimbursement (i.e. tafamidis, patisiran, or inotersen) from 1 January 2014 to 31 December 2021 (index date in 2021). Demographics and comorbidities at the baseline, healthcare resource consumption, and related direct costs reimbursed by the SSN throughout the one-year follow-up were described.

RESULTS:

In 2021, 36 patients with ATTRv-PN (prevalence: 7.4/1,000,000) were identified (males were 83.3%; patients with ≥2 comorbidities were 61.1%; the mean age was 73 ± 8 years). During follow-up, of patients, 91.7% received drugs for ATTRv-PN; >50% received antiepileptics and acid suppressants; 22.2% were admitted to overnight hospitalizations; 30.6% accessed the emergency department; 97.2% received local outpatient specialist care. The per patient mean annual cost was € 122,017; drugs for ATTRv-PN accounted for 94.7% of the total expenditure.

CONCLUSIONS:

This study of real-world patients with ATTRv-PN showed a high rate of comorbidities, and substantial direct healthcare and economic burdens on the SSN.

01 Apr 2025·ESC Heart Failure

Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study

Article

Author: Kindermann, Ingrid ; Zimmer, Angela ; Papathanasiou, Maria ; Yilmaz, Ali ; Cejka, Vladimir ; Luedike, Peter ; Voss, Fabian ; Ney, Svenja ; Unsöld, Bernhard ; Pfister, Roman ; Bewarder, Yvonne ; Chamling, Bishwas ; Nies, Richard J. ; Michel, Lars ; Rassaf, Tienush ; Kelm, Malte ; Störk, Stefan ; Hahn, Katrin ; Meindl, Christine ; Polzin, Amin ; Spethmann, Sebastian ; Knebel, Fabian ; Paulus, Michael ; Morbach, Caroline

Abstract:

Aims:

Data on the clinical profiles of patients with transthyretin amyloidosis cardiomyopathy (ATTR‐CM) in the post‐approval era of tafamidis 61 mg are lacking. Study aims were characterization of contemporary ATTR‐CM patients, analysis of potential eligibility for the ‘Transthyretin Amyloidosis Cardiomyopathy Clinical Trial’ (ATTR‐ACT) and identification of factors associated with the decision on tafamidis 61 mg treatment.

Methods and results:

This retrospective study analysed ATTR‐CM patients seen at eight University Hospitals in the first year after approval of tafamidis 61 mg for ATTR‐CM in Germany (April 2020 to March 2021). The cohort comprised 366 patients (median age 79 [74; 82] years, 84% male), with 47% and 45% of the cohort being in National Amyloidosis Centre ATTR stage ≥ II and NYHA class ≥ III, respectively. Sixty‐four per cent of patients met key eligibility criteria of the pivotal ATTR‐ACT. In recently diagnosed patients (58% with diagnosis ≤6 months), the rate of variant ATTR was significantly lower than in patients diagnosed more than 6 months ago (9.3% vs. 19.7%). Of the 293 patients without prior ATTR specific treatment, tafamidis 61 mg was newly initiated in 77%. Patients with tafamidis 61 mg treatment were significantly younger, were more often eligible for ATTR‐ACT, had lower NYHA class and higher serum albumin levels. These variables explained 16% of the variance of treatment decision. Unadjusted survival was higher in patients with than those without treatment (1‐year survival 98.6% vs. 87.3%, P < 0.001).

Conclusions:

Wild‐type ATTR was the primary aetiology amongst contemporary ATTR‐CM patients and almost two‐thirds of patients were in an advanced disease stage. Clinical profiles of 64% of patients in routine care matched those of the ATTR‐ACT. Further effort is needed to detect patients at an earlier disease stage and to validate criteria justifying treatment initiation.

132

News (Medical) associated with Tafamidis

31 Mar 2025

·www.globenewswire.com

Acoramidis Shows Statistically Significant Improvements in Cardiovascular Outcomes in Patients with Variant ATTR-CM

Acoramidis’s hazard ratio of .41 for time to ACM or first CVH versus placebo in the ATTRibute-CM study subgroup of ATTRv-CM patients achieved statistical significance in a pre-specified analysisThis profound treatment effect, due to the near-complete stabilization (≥90%) and binding of TTR, represents the greatest observed benefit to date for ATTRv-CM patients, an ATTR-CM population with poor prognosis In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRw-CM patients: In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placeboA 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA™ by the European Commission and Japanese Pharmaceuticals and Medical Devices Agency PALO ALTO, Calif., March 31, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today presented results showing statistically significant improvements in clinical outcomes as compared to placebo for time to all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH) in both variant (ATTRv) and wild-type (ATTRwt) transthyretin amyloid cardiomyopathy (ATTR-CM) patients from a pre-specified subgroup analysis of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a poster presentation by Margot Davis, M.D. of Vancouver General Hospital, Canada. Acoramidis is a selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. “Variant ATTR-CM patients’ condition often presents at an earlier age and progresses more rapidly than patients with wild-type disease, which translates into a worse prognosis in many such patients. We know that pathogenic TTR variant tetramers are less stable than the wild-type tetramer and this property is directly responsible for the more aggressive disease trajectory for these patients. The findings from the ATTRibute-CM trial clearly demonstrate that the rapid and sustained increases in serum TTR levels upon initiation of acoramidis treatment in variant ATTR-CM patients were similar if not greater than those observed in wild-type ATTR-CM patients,” said Jonathan Fox, M.D., Ph.D., president and chief medical officer of BridgeBio Cardiorenal. “This provides evidence that acoramidis is the only disease-modifying therapy that provides near-complete stabilization of TTR, improving clinical outcomes in both variant and wild-type ATTR-CM patients to an extent that is independently statistically significant in both subgroups.” “Given the significant unmet need for patients with ATTRv-CM, we are highly encouraged by the magnitude of efficacy seen with acoramidis. A pre-specified analysis in the ATTRv-CM population has shown a statistically significant 59% hazard reduction for the composite of ACM and CVH at Month 30, demonstrating that acoramidis can make a profound impact on patients’ lives,” said Kevin Alexander, M.D. of Stanford University School of Medicine. Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature.1 The serum TTR level increase with acoramidis was accompanied by a significant reduction in the risk of ACM or first CVH versus placebo in both the ATTRv-CM (59.1% risk reduction, .41 hazard ratio) and ATTRwt-CM (31.2% risk reduction, .69 hazard ratio) subgroups. Acoramidis treatment also led to a greater proportional increase in serum TTR in ATTRv-CM patients and achieved similar absolute serum TTR levels in both ATTRv- and ATTRwt-CM patients, which is an in vivo reflection of acoramidis’ near-complete (≥90%) TTR stabilization. Additional acoramidis poster presentations and moderated posters at the ACC Annual Scientific Sessions & Expo included: Acoramidis Improves NYHA Class at Month 30 Versus Placebo in Patients with ATTR-CM: Results from the ATTRibute-CM Study shared by Kevin Alexander, M.D. of Stanford University School of Medicine In the ATTRibute-CM study, acoramidis treatment resulted in a greater proportion of patients whose New York Heart Association (NYHA) Class was stable or improved at Month 30 vs placebo, indicating better stabilization in their heart failure symptoms and functional status. The NYHA classification system categorizes heart failure patients into four classes based on a clinical assessment of their physical activity limitations and associated symptoms due to their condition In Participants Treated with Acoramidis, Addition of Concomitant Tafamidis Did Not Further Increase Serum TTR Levels shared by Mathew Maurer, M.D. of Columbia University Irving Medical Center The poster showed that in patients with ATTR-CM, treatment with acoramidis significantly increased serum TTR levels whether compared to placebo alone or in those who received placebo as well as tafamidis. Conversely, the addition of tafamidis to acoramidis did not demonstrate any further increase in serum TTR levels, reflecting the lack of any additional stabilization benefit of serum TTR from tafamidis in vivo, as previously demonstrated in vitro. The safety profile in this limited dataset of concomitant acoramidis and tafamidis was similar to the overall safety profile of acoramidis alone Primary Endpoint Efficacy Results in the ATTRibute-CM Study: Pre-specified Sensitivity Analyses Addressed Tafamidis Use shared by Daniel P. Judge, M.D. of Medical University of South Carolina Pre-specified analyses showed consistent results with the primary analysis, demonstrating that the concomitant use of tafamidis did not alter the statistical significance of the primary efficacy endpoint Acoramidis-mediated Early Increase in Serum Transthyretin Level Reduces Cardiovascular-related Hospitalizations and Mortality: Insights from the ATTRibute-CM Study shared by Nitasha Sarswat, M.D. of UChicago Medicine In this post-hoc analysis of ATTRibute-CM, incremental increases in serum TTR levels on Day 28, achieved with acoramidis, may independently predict greater reduction in risks of cardiovascular mortality and of first CVH in patients with ATTR-CM Robustness of Primary Endpoint Efficacy Results with Acoramidis in ATTR-CM in the ATTRibute-CM Study: Pre-specified NT-proBNP Sensitivity Analyses shared by Jan Griffin, M.D. of Medical University of South Carolina This poster shows that pre-specified sensitivity analyses using higher N-terminal pro-type natriuretic peptide (NT-proBNP) thresholds for declaring a difference in the changes in NT-proBNP levels between the treatment arms showed consistent efficacy favoring acoramidis in patients with ATTR-CM. This confirms the robustness of the acoramidis treatment effect regardless of NT-proBNP progression thresholds Geographic Healthcare Disparities and Diagnostic Trends Among Patients with Transthyretin Amyloid Cardiomyopathy shared by Joshua Mitchell, M.D. of Washington University School of Medicine in St. Louis Findings presented show that the diagnosed prevalence of amyloid has significantly increased since 2017 in the setting of available treatment, improved awareness and less invasive diagnostics. However, there remain geographic disparities and racial differences in ATTR-CM prevalence Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission and Japanese Pharmaceuticals and Medical Devices Agency with all labels specifying near-complete stabilization of TTR. More data on the benefit of Attruby for ATTRv-CM patients is planned for future medical meetings. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. About Attruby™ (acoramidis)Attruby is the first near-complete (≥90%) stabilizer of Transthyretin (TTR) approved in the U.S. for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Attruby was generally well-tolerated. The most common side effects were mild and included diarrhea and abdominal pain that were resolved without drug discontinuation. BridgeBio offers an extensive suite of programs to help patients access our medicines. About BridgeBioBridgeBio Pharma (BridgeBio; NASDAQ:BBIO) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter and Facebook. BridgeBio Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “continue,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic potential of our programs and product candidates, including our clinical development program for acoramidis for patients with transthyretin amyloid cardiomyopathy and the statements regarding the potential clinical benefits or of potential benefits for ATTR-CM patients in the quotes of Dr. Alexander, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA and other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, our ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in our share price, the impacts of current macroeconomic and geopolitical events, including changing conditions from, hostilities in Ukraine, and in Israel and the Gaza Strip, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@bridgebio.com (650)-789-8220

Clinical ResultDrug ApprovalPhase 3AHA

26 Mar 2025

·www.biospace.com

Trump’s Pharma Tariffs, Monarez for CDC, Novo’s New Obesity Play, More

President Donald Trump continues to warn of tariffs on the pharmaceutical industry; Susan Monarez replaces Dave Weldon as CDC director nominee; Novo Nordisk joins the triple-G race; Alnylam wins approval for Amvuttra in ATTR-CM; and Cassava Sciences ends development of simufilam in Alzheimer’s. > Listen on Spotify > Listen on Apple Products > Listen on Amazon Music > Listen on iHeart President Donald Trump doubled down on tariff threats targeting pharma, saying additional levies on pharmaceuticals will come “at some point,” per CNBC. Meanwhile, Johnson & Johnson became the latest big pharma to respond to Trump’s warning of potential tariffs if companies don’t reshore their manufacturing, announcing a massive $55 billion U.S. manufacturing and R&D investment. Not all companies are on board, however: AstraZeneca is looking eastward, pumping $2.5 billion into a new research facility in Beijing. Also on the policy front, Trump nominated acting CDC director Susan Monarez for the top job after pulling his first nominee, Dave Weldon , days before his senate hearing was expected to begin. If confirmed, Monarez would be the first CDC director since 1953 to not have a medical degree; she holds a Ph.D. in microbiology and immunology from the University of Wisconsin. In weight loss news, Novo Nordisk is paying China-based United Laboratories $200 million upfront to license a triple agonist of the GLP-1, GIP and glucagon receptors that could one day compete with Eli Lilly’s retatrutide. And BioSpace examines the next great challenge for GLP-1s: oral formulation manufacturing. Two more therapeutic spaces in focus last week are Duchenne muscular dystrophy and spinal muscular atrophy , where companies including Dyne Therapeutics, REGENXBIO and Novartis presented new data on their respective candidates. And the Duchenne community continued to react to news of the death of a patient taking Sarepta’s approved gene therapy Elevidys . In cardiovascular news, Alnylam won a much-anticipated approval for Amvuttra as the first RNAi silencer for transthyretin amyloid cardiomyopathy, setting up a three-way race with Pfizer’s tafamidis—marketed as Vyndaqel and Vyndama—and BridgeBio’s Attruby. Next up is Milestone Therapeutics’ CARDAMYST in paroxysmal supraventricular tachycardia, which has a PDUFA date of March 27. Finally, the saga of Cassava Sciences’ Alzheimer’s hopeful simufilam is over, as the company announced it has ended development of the controversial candidate.

Executive ChangeGene TherapyIPODrug Approval

21 Mar 2025

·endpts.com

Alnylam hopes its broad ATTR-CM label can give Amvuttra an edge in heart disease

Alnylam will likely face challenges in turning a profit this year, despite executives promising to do so. But the label for its newly-approved Amvuttra indication may give it an advantage over its rivals Pfizer and BridgeBio. That’s because Amvuttra’s label for transthyretin amyloid cardiomyopathy, or ATTR-CM, is broader than its competitors, saying it reduces the likelihood of hospitalization, death and urgent heart failure visits. Pfizer’s Vyndaqel/Vyndamax franchise, approved in 2019, and BridgeBio’s recently-approved Attruby are only indicated to reduce hospitalization and death. “The inclusion of urgent heart failure visits is unique and recognizes the effects that we’re also seeing on this important consequence of ATTR-cardiomyopathy,” Alnylam chief medical officer Pushkal Garg said on an investor call Thursday evening. Garg touted the broader label as key to getting Amvuttra in as many first-line patients as possible. Wall Street appeared to like what it saw as well after the FDA approved the drug for the rare heart disease. After a trading halt Thursday afternoon, Alnylam’s stock $ALNY was up more than 10% in Friday’s session. Most analysts expect getting Amvuttra into first-line patients will determine whether Alnylam is profitable this year, and they don’t think it will be easy. Both Vyndaqel/Vyndamax and Attruby are oral stabilizers, a class that’s been on the market for several years that works by stabilizing the TTR protein and preventing misfolding. Amvuttra, meanwhile, is the first therapy approved in its class for ATTR-CM, reducing the expression of the TTR gene through RNA interference. There’s also a wrinkle when it comes to second-line patients who could switch from the Pfizer and BridgeBio drugs to Amvuttra: There is no formal definition for ATTR-CM disease progression. The decisions that patients and their doctors make about which therapy to take — one of the Pfizer/BridgeBio stabilizers, Alnylam’s silencer or both mechanisms in combination — will be made on a case-by-case basis. In an interview before Thursday’s approval, CEO Yvonne Greenstreet told Endpoints News the company can support a successful launch in ATTR-CM by leveraging its existing physician network for doctors who prescribe Amvuttra in its other indication, hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. She also said she believes Alnylam’s clinical trial data will make patients’ choices easier, regardless of whether they’ve taken a stabilizer before. “The simpler it is for physicians, the easier it is for them to come to a determination,” Greenstreet told Endpoints. “But I have to say that, even without specific, clear guidelines with, let’s say, one biomarker to assess, physicians are able to interpret whether their patients are progressing or not.” The company is forecasting revenues between $1.6 billion and $1.725 billion for its ATTR medicines (Amvuttra and Onpattro) this year after they made about $1.2 billion in 2024, giving them what Greenstreet has called a “path to profitability.” Alnylam lost $278 million last year. Alnylam is maintaining the same price of about $476,000 per year for Amvuttra in ATTR-CM as it had for hATTR. It’s nearly double what Pfizer and BridgeBio charge for their drugs, at around $250,000 and $244,500, respectively. Despite that, analysts largely saw the label as a win for Alnylam in notes out to investors Thursday evening and Friday morning. TD Cowen’s Ritu Baral wrote the inclusion of urgent heart failure visits will help in payer talks, “especially for front line use,” as ATTR-CM care has moved into the outpatient setting. With the pricing information confirmed, Baral estimates $254 million in ATTR-CM sales and peak sales of $2.3 billion by 2030. Stifel analyst Paul Matteis, meanwhile, wrote that he believes investor fixation on label language was overblown, questioning whether it would have an impact on patient uptake. But the fact that Alnylam was able to deliver on a differentiated label is still a success. “How much this will matter remains open for debate, but on the margin it could help with the marketing message and it also may be helpful with [Alnylam’s] ability to maintain premium pricing,” Matteis wrote. That high price may make some payers balk in insurance discussions, Leerink analyst Mani Foroohar noted. Most of the patient population is covered under Medicare Part B, however, giving doctors different prescribing incentives. Greenstreet said that about 70% of patients taking Amvuttra for hATTR pay no out-of-pocket costs, and she expects Alnylam to eventually get to about that level in ATTR-CM as well. That dynamic, when combined with the data and label, are all the pieces Alnylam needs to put together.

Drug Approval

100 Deals associated with Tafamidis

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Transthyretin Amyloid Cardiomyopathy	United States	FoldRx Pharmaceuticals LLC	03 May 2019

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Amyloid Neuropathies, Familial	Phase 3	Japan	Pfizer Inc.	01 Nov 2011
Cutaneous Mastocytosis	Phase 3	United States	Pfizer Inc.	30 Sep 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	United States	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	Argentina	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	Brazil	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	France	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	Germany	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	Italy	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	Portugal	Pfizer Inc.	05 Aug 2009
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	Sweden	Pfizer Inc.	05 Aug 2009

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02791230 (ATTR-ACT, CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy	1,733	Tafamidis (Cohort A: Tafamidis (Parent Study) and Tafamidis (Extension Study))	xxaygsvkpp(vuchqolhxk) = hbqwmymptc gjnfflqwqo (uxmnxnkrbq, qcutqzikqq - nhnujvyzyc) View more	-	03 Feb 2025
				Tafamidis+Placebo (Cohort A: Placebo (Parent Study) and Tafamidis (Extension Study))	xxaygsvkpp(vuchqolhxk) = gfusrakoux gjnfflqwqo (uxmnxnkrbq, pjxytqdflt - evmswfrmxy) View more
NCT04814186 (CTgov)	Phase 4	Transthyretin Amyloid Cardiomyopathy	53	tafamidis	uttajakyvy = aopbufyhkq jnhmgtphmi (fjzbwvnvkq, uzifzjihap - rtvgcnezjp) View more	-	18 Dec 2024
ESC2024	Not Applicable	Transthyretin Amyloid Cardiomyopathy	-	Tafamidis	kgwainmgko(xytqkvsuzi) = qsaybnksmr okvwhcksmo (wmwlzwrame )	-	01 Sep 2024
NCT01994889 \| NCT02791230 (ATTR-ACT, ESC2024)	Phase 3	Transthyretin Amyloid Cardiomyopathy NT-proBNP \| eGFR	350	Tafamidis 80 mg	hlqwbbkvgk(dhbenreauy) = yxlxdxpvhw rmlpwrpnvw (hamlaqghqe ) View more	Positive	01 Sep 2024
				Placebo	hlqwbbkvgk(dhbenreauy) = bguweetetd rmlpwrpnvw (hamlaqghqe ) View more
ESC2024	Not Applicable	Senile cardiac amyloidosis	-	Tafamidis	wiytztinfq(pxcfxywpkj) = aopiifmalk cypymfbxuu (yautfvbiwi ) View more	-	31 Aug 2024
				Placebo	wiytztinfq(pxcfxywpkj) = vlawxqkfuh cypymfbxuu (yautfvbiwi ) View more
ESC2024	Not Applicable	Transthyretin cardiac amyloidosis	56	Improved renal function after tafamidis administration	hidobrmffc(bspdebswkl) = E’: 0.8 ± 1.7 cm/s vs. 0.2 ± 1.1 cm/s, p=0.127, LAVI: -8.7 ± 12.0 mL/m2 vs. 0.2 ± 16.8 mL/m2, p=0.093 wkdznwhizv (wttnkbshpu )	Positive	31 Aug 2024
				Deteriorated renal function after tafamidis administration
ESC2024	Not Applicable	Senile cardiac amyloidosis	710	Tafamidis treatment	hulqohrxzp(ekqljcnosk) = dajrpnscby xqafeochot (ymmyulkpdn )	Positive	30 Aug 2024
				No Tafamidis treatment	hulqohrxzp(ekqljcnosk) = ryxmiyarzx xqafeochot (ymmyulkpdn )
NCT02791230 (ATTR-ACT, ESC2024)	Phase 3	Transthyretin Amyloid Cardiomyopathy	1,481	Tafamidis free acid 61 mg	jixgqlrvfa(qogaowqbma) = xjrpwvlpvv tvhbkrqfor (dickxbsfjn, 0.6) View more	Positive	30 Aug 2024
NCT01994889 \| NCT02791230 (ATTR-ACT, Pubmed \| Eur J Heart Fail)	Phase 3	Transthyretin Amyloid Cardiomyopathy	-	Tafamidis 80 mg meglumine or 61 mg free acid	pnazwbxjvx(astvyekpie): P-Value = < 0.001	Positive	26 Jun 2024
				Placebo
ESC2024	Not Applicable	Senile cardiac amyloidosis	-	Tafamidis	havmjqgght(vpzbaxhzjq) = xznejgiytg ovgmiecxdb (behoybmfqj ) View more	-	12 May 2024

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis