Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed ATTRV122I amyloidosis 3 milestones | |||||
| Filed | EU (EMA) | — | ATTRV122I amyloidosis | 2024-04-07 | “The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.” bridgebio.com ↗ “a Marketing Authorization Application (MAA) for acoramidis has been accepted by the European Medicines Agency (EMA)” bridgebio.com ↗ “Marketing authorization application (MAA) with the European Medicines Agency (EMA) was filed in January 2024” bayer.com ↗ “The EMA accepted the Company’s MAA for acoramidis for review, with an expected approval in 2025” bridgebio.com ↗ |
| Filed | US (FDA) | — | ATTRV122I amyloidosis | 2024-04-07 | “Based on the positive results from ATTRibute-CM, BridgeBio submitted an NDA to the U.S.” bridgebio.com ↗ |
| Filed | US (FDA) | — | ATTRV122I amyloidosis | 2023-12-05 | “today announced that it has submitted an NDA for acoramidis to the U.S. FDA for the treatment of ATTR-CM” bridgebio.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Beyonttra | — | 2025-02-10 | europa.eu ↗ |
| Approved | US (FDA) | ATTRUBY | — | 2024-11-22 | fda.gov ↗ |
Trials 11
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT04958135 | May → Aug 2021 | — | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04769479 | Mar → Aug 2021 | amyloidosis | Eidos Therapeutics, a BridgeBio company | Completed | No outcome recorded |
| Phase 36 trials · 1 met primary | ||||||
| Phase 3 | NCT07116473 | Nov 2025 → Nov 2037 expected | cardiomyopathy, hereditary amyloidosis, polyneuropathy | Eidos Therapeutics, a BridgeBio company | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT06563895 | May 2025 → Oct 2031 expected | cardiomyopathy, hereditary amyloidosis, polyneuropathy | Eidos Therapeutics, a BridgeBio company | Recruiting | No outcome recorded |
| Phase 3 | NCT04988386 | Oct 2021 → Apr 2028 expected | — | Eidos Therapeutics, a BridgeBio company | Active not recruiting | Met primary |
| Phase 3 | NCT04882735 | Sep 2021 → Sep 2026 | familial amyloid neuropathy | Eidos Therapeutics, a BridgeBio company | Withdrawn | No outcome recorded |
| Phase 3 | NCT04622046 | Nov 2020 → Nov 2023 | amyloidosis | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT03860935 | Mar 2019 → May 2023 | cardiomyopathy, hereditary amyloidosis | Eidos Therapeutics, a BridgeBio company | Completed | No outcome recorded |
| Phase 43 trials | ||||||
| Phase 4 | NCT07695701 | Jul 2026 → Aug 2030 expected | amyloidosis | Eidos Therapeutics, a BridgeBio company | Not yet recruiting | No outcome recorded |
| Phase 4 | NCT07298044 | Feb 2026 → Jul 2027 expected | amyloidosis | Bayer | Recruiting | No outcome recorded |
| Phase 4 | NCT07306949 | Oct 2025 → Mar 2028 expected | — | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 46
| Date | Issuer | Release |
|---|---|---|
| 2026-08-30 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Demonstrates Reversal of Cardiac Structural Disease Progression and Functional Decline and Significantly Increases Days Alive and Free from Hospitalization in ATTR-CM bridgebio.com ↗
Acoramidis demonstrated long-term efficacy and safety through 54 months across variant ATTR-CM subgroups, including p.Val142Ile and non-p.Val142Ile. |
| 2026-07-02 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Associated with First-Ever Early and Sustained Direct Kidney-Protective Effects in ATTR-CM bridgebio.com ↗
Acoramidis initiation was associated with rapid kidney-protective activity as exhibited in a hemodynamically mediated, reversible eGFR dip and a placebo-corrected 15.5% reduction in UACR by Day 28 (P<0.05), with no kidney-related adverse events observed in these post-hoc analyses |
| 2026-05-11 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Continues to Demonstrate Disease-Modifying Effects in ATTR-CM, Reducing sTTR Variability and Outpatient Worsening Heart Failure bridgebio.com ↗
Acoramidis increased sTTR early and significantly reduced intra-individual sTTR variability versus placebo (p<0.001), which is associated with reduced ACM, further reinforcing its differentiated profile in ATTR-CM |
| 2026-05-06 | Eidos Therapeutics, a BridgeBio company | Regulatory BEYONTTRA™ (acoramidis), the First Near-Complete TTR Stabilizer (≥90%), Approved by ANVISA to Treat ATTR-CM in Brazil bridgebio.com ↗
The approval of Beyonttra in Brazil is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date, including: |
| 2026-03-30 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Significantly Reduces the Risk of All-Cause and Cardiovascular Mortality in Patients with ATTR-CM through Month 54 bridgebio.com ↗
today presented long-term efficacy and safety data from the ATTRibute-CM open-label extension (OLE) trial, demonstrating sustained clinical benefit of acoramidis through Month 54 in patients with ATTR-CM. |
| 2025-11-08 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Significantly Reduces All-cause Mortality in the Overall ATTR-CM Variant and V142I (V122I) Populations bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, presented data from the ATTRibute-CM study, showing that acoramidis significantly reduces all-cause mortality (ACM) through Month 42 in the overall variant ATTR-CM population, and specifically in the p.Val142Ile (V142I, V122I) subpopulation. |
| 2025-11-03 | Eidos Therapeutics, a BridgeBio company | Results BridgeBio to Present Data from the Overall ATTR-CM Variant and V142I (V122I) Populations from ATTRibute-CM at the AHA Scientific Sessions 2025 bridgebio.com ↗
Acoramidis Reduces All-Cause Mortality and First Cardiovascular Hospitalization in Patients with Variant Transthyretin Amyloid Cardiomyopathy: Results from the ATTRibute-CM Study |
| 2025-09-28 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Begins to Reduce Cumulative Cardiovascular Outcomes Within the First Month of Treatment in Patients with ATTR-CM bridgebio.com ↗ |
| 2025-09-22 | Eidos Therapeutics, a BridgeBio company | Results BridgeBio to Present Additional Cardiovascular Outcomes Data from ATTRibute-CM at the HFSA Annual Scientific Meeting 2025 bridgebio.com ↗
Continuous Acoramidis Treatment Significantly Reduced Risk of All-cause Mortality and Cardiovascular-related Hospitalization at Month 42, in Patients with Wild-type and Variant Transthyretin Amyloidosis Cardiomyopathy |
| 2025-08-30 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Demonstrates Statistically Significant Reduction in Cardiovascular Mortality (CVM) through Month 42 of the ATTRibute-CM Open Label Extension bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, presented data from the ATTRibute-CM open label extension (OLE) through Month 42, which demonstrated a statistically significant reduction in CVM in the overall transthyretin amyloid cardiomyopathy (ATTR-CM) population. |
| 2025-05-20 | Eidos Therapeutics, a BridgeBio company | Results Acoramidis Reduced Incidence of Atrial Fibrillation Events in Patients with ATTR-CM bridgebio.com ↗
The reduction in new-onset atrial fibrillation and AF-related hospitalizations represents an important finding for the ATTR-CM community. |
| 2025-05-19 | Eidos Therapeutics, a BridgeBio company | Results Early and Sustained Increase in Serum TTR Levels by Acoramidis Independently Predicted Improved Survival in the ATTRibute-CM Study bridgebio.com ↗
The open-label extension data for all ATTRibute-CM participants at Month 42 showed that rapid, sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in ACM and CVH (including urgent outpatient treatment for heart failure exacerbations) |
| 2025-04-28 | Eidos Therapeutics, a BridgeBio company | Regulatory BEYONTTRA® (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved by the UK Medicines and Healthcare Products Regulatory Agency to Treat ATTR-CM bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced the Medicines and Healthcare products Regulatory Agency has granted marketing authorization in the United Kingdom (UK) for acoramidis, under the brand name BEYONTTRA®, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). |
| 2025-03-31 | Eidos Therapeutics, a BridgeBio company | Results acoramidis shows statistically significant improvements in cardiovascular outcomes in patients with variant attr-cm bridgebio.com ↗
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 analysis |
| 2025-03-27 | Eidos Therapeutics, a BridgeBio company | Regulatory beyonttra™ (acoramidis), the first near-complete ttr stabilizer (≥90%), approved in japan to treat attr-cm bridgebio.com ↗
The approval was based on a Japanese Phase 3 study and the global ATTRibute-CM Phase 3 trial, which demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date. |
| 2025-03-24 | Eidos Therapeutics, a BridgeBio company | Results bridgebio to present cardiovascular outcomes data in patients with variant and wild-type transthyretin amyloid cardiomyopathy (attr-cm) from the attribute-cm study at the acc annual scientific sessions bridgebio.com ↗
Acoramidis Improves NYHA Class at Month 30 Versus Placebo in Patients with ATTR-CM: Results from the ATTRibute-CM Study |
| 2025-02-11 | Bayer | Regulatory Heart drug Beyonttra™ (acoramidis) approved in EU for treatment of transthyretin amyloidosis in adults with cardiomyopathy bayer.com ↗
The European Commission has granted marketing authorization in the European Union (EU) for acoramidis, under the brand name Beyonttra™, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). |
| 2025-02-11 | Eidos Therapeutics, a BridgeBio company | Regulatory beyonttra™ (acoramidis), the first near complete ttr stabilizer (≥90%), approved by the european commission to treat attr-cm bridgebio.com ↗ |
| 2025-01-14 | Bayer | Regulatory Bayer’s Pharma Growth Strategy Progressing Well as Pipeline Advances bayer.com ↗
On the occasion of the 43rd J.P. Morgan Healthcare Conference in San Francisco, Bayer AG announces further progress in its pharmaceutical growth strategy, with multiple filing submissions underway for key growth drivers darolutamide and finerenone, acoramidis and elinzanetant. |
| 2024-12-13 | Bayer | Regulatory Acoramidis receives positive CHMP opinion for treatment of transthyretin amyloid cardiomyopathy bayer.com ↗
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended acoramidis, a selective small molecule, orally administered transthyretin (TTR) stabilizer, for marketing authorization in the European Union (EU). |
| 2024-12-13 | Eidos Therapeutics, a BridgeBio company | Regulatory acoramidis receives positive chmp opinion for treatment of transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗
The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of acoramidis in the EU based on positive results from the Phase 3 ATTRibute-CM study; |
| 2024-11-22 | Eidos Therapeutics, a BridgeBio company | Regulatory attruby™ (acoramidis), a near complete ttr stabilizer (≥90%), approved by fda to reduce cardiovascular death and cardiovascular-related hospitalization in attr-cm patients bridgebio.com ↗ |
| 2024-11-18 | Eidos Therapeutics, a BridgeBio company | Results open-label extension data confirms sustained benefit of acoramidis on cardiovascular outcomes, including statistically significant reduction in acm within 36 months bridgebio.com ↗
Acoramidis demonstrated the earliest known time to separation in cardiovascular outcomes in the ATTRibute-CM study (3 months), with statistically significant risk reduction of 36% on All-Cause Mortality (ACM) alone at Month 36 within the Open Label Extension |
| 2024-09-27 | Eidos Therapeutics, a BridgeBio company | Results bridgebio shares recurrent event analysis of attribute-cm, demonstrating a 42% reduction by acoramidis on the composite endpoint of all-cause mortality and recurrent cardiovascular-related hospitalization events bridgebio.com ↗
Acoramidis treatment led to a highly significant reduction in all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH) at Month 30 compared with placebo |
| 2024-08-30 | Eidos Therapeutics, a BridgeBio company | Results bridgebio shares data on serum ttr increase when switching participants from placebo and tafamidis to acoramidis in attribute-cm and its open-label extension bridgebio.com ↗
Increased serum TTR at Day 28 of ATTRibute-CM was correlated with reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in transthyretin amyloid cardiomyopathy (ATTR-CM) |
| 2024-05-29 | Eidos Therapeutics, a BridgeBio company | Results additional data showing acoramidis increases serum transthyretin which is associated with improved cardiovascular outcomes presented at isa from bridgebio pharma’s phase 3 attribute-cm study in transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced positive results of five new analyses of clinical endpoint events from its Phase 3 ATTRibute-CM study of acoramidis in ATTR-CM. |
| 2024-05-13 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents additional data and analyses from its phase 3 attribute-cm study in transthyretin amyloid cardiomyopathy (attr-cm) at esc-hf, including that acoramidis treatment significantly reduced all-cause mortality in a pre-specified sensitivity analysis of the entire study population bridgebio.com ↗
In a pre-specified Cochran-Mantel-Haenszel sensitivity analysis applied to the entire intention-to-treat (ITT) population of the study (N=632), acoramidis significantly reduced all-cause mortality (p=0.04), with no safety signals of potential clinical concern |
| 2024-04-07 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents cardiac magnetic resonance (cmr) imaging evidence consistent with clinical improvement observed in the attribute-cm phase 3 study in patients with transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗ |
| 2024-02-05 | Eidos Therapeutics, a BridgeBio company | Regulatory bridgebio pharma announces u.s. food and drug administration (fda) acceptance of new drug application (nda) for acoramidis for the treatment of patients with transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗
The FDA has set an action date of November 29, 2024 under the PDUFA. |
| 2024-02-02 | AstraZeneca | Results Positive high-level results from Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) showed consistency with global ATTRibute-CM Phase III trial astrazeneca.com ↗
These results offer further evidence that TTR stabilisation with acoramidis may improve survival and reduce disease severity for patients by preventing further breakdown of these proteins. |
| 2024-02-02 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma shares positive results of single-arm phase 3 study of acoramidis in japanese patients with transthyretin amyloid cardiomyopathy (attr-cm) including no mortality reported in the trial at 30 months bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today shared positive results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), conducted by Alexion, AstraZeneca Rare Disease. |
| 2024-01-10 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma announces publication of positive results from phase 3 attribute-cm study of acoramidis for patients with transthyretin amyloid cardiomyopathy (attr-cm) in the new england journal of medicine bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced that positive results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM were published in the New England Journal of Medicine (NEJM). |
| 2023-12-05 | Eidos Therapeutics, a BridgeBio company | Regulatory bridgebio pharma announces submission of new drug application (nda) to u.s. food and drug administration (fda) for acoramidis for the treatment of patients with transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that it has submitted an NDA for acoramidis to the U.S. FDA for the treatment of ATTR-CM. |
| 2023-11-12 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents additional clinical outcomes data from the phase 3 attribute-cm study of acoramidis in patients with transthyretin amyloidosis cardiomyopathy (attr-cm) bridgebio.com ↗
Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified |
| 2023-08-27 | Eidos Therapeutics, a BridgeBio company | Results bridgebio presents detailed positive results from phase 3 attribute-cm study of acoramidis for patients with transthyretin amyloid cardiomyopathy (attr-cm) at european society of cardiology congress 2023 bridgebio.com ↗ |
| 2023-07-17 | Eidos Therapeutics, a BridgeBio company | Results bridgebio announces consistently positive results from phase 3 attribute-cm study of acoramidis for patients with transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗
Highly statistically significant result observed on primary endpoint with a Win Ratio of 1.8 (p<0.0001) |
| 2022-05-05 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma reports first quarter 2022 financial results and business update bridgebio.com ↗
Reported data from ongoing Phase 2 open-label extension (OLE) study of acoramidis (AG10) in patients with symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) which demonstrated acoramidis continued to be well-tolerated |
| 2022-04-03 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents updated results from phase 2 open-label extension study of acoramidis in transthyretin amyloid cardiomyopathy (attr-cm) bridgebio.com ↗ |
| 2021-12-27 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma reports month 12 topline results from phase 3 attribute-cm study bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) today announced topline results from Month 12 (Part A) of ATTRibute-CM, an ongoing global Phase 3 study investigating acoramidis for the treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). |
| 2019-11-16 | Eidos Therapeutics, a BridgeBio company | Results bridgebio and eidos present data from phase 2 open label extension suggesting long-term tolerability of ag10 and stabilization of transthyretin amyloid cardiomyopathy disease measures bridgebio.com ↗
AG10 treatment was generally well-tolerated and resulted in near-complete TTR stabilization as measured using established ex vivo assays. |
| 2018-11-10 | Eidos Therapeutics, a BridgeBio company | Results eidos therapeutics announces positive phase 2 data for ag10 in symptomatic patients with mutant or wild-type ttr amyloid cardiomyopathy bridgebio.com ↗
Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced positive results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM). |
| 2018-10-26 | Eidos Therapeutics, a BridgeBio company | Regulatory eidos therapeutics receives positive opinion for orphan designation from the european medicines agency for ag10, a potent oral stabilizer for the treatment of transthyretin amyloidosis bridgebio.com ↗
Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), announced today that the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) adopted a positive opinion for the designation of Eidos’ product candidate, AG10, as an orphan medicinal product for the treatment of transthyretin (TTR) amyloidosis (ATTR). |
| 2018-10-03 | Eidos Therapeutics, a BridgeBio company | Regulatory fda grants orphan drug designation to eidos therapeutics’ product candidate, ag10, for treatment of transthyretin amyloidosis bridgebio.com ↗ |
| 2018-09-17 | Eidos Therapeutics, a BridgeBio company | Results eidos therapeutics presents data from its phase 1 clinical trial of ag10 at the 22nd annual scientific meeting of the heart failure society of america bridgebio.com ↗
Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX) announced the presentation of results from its Phase 1 clinical trial of AG10 during a poster session at the 22nd Annual Scientific Meeting of the Heart Failure Society of America (HFSA). |
| 2018-08-23 | Eidos Therapeutics, a BridgeBio company | Results eidos therapeutics announces publication of ag10 molecular design in journal of medicinal chemistry bridgebio.com ↗
The published paper demonstrates that AG10’s binding to TTR is driven by molecular interactions that mimic the structure of the T119M TTR variant that is known to protect carriers from developing ATTR and leads to prolonged lifespan in otherwise healthy individuals. |
| 2017-11-15 | Eidos Therapeutics, a BridgeBio company | Results preclinical data demonstrates potential of eidos therapeutics’ lead compound for the treatment for transythyretin amyloidosis bridgebio.com ↗
Eidos Therapeutics, a subsidiary of BridgeBio Pharma, today announced that recently presented preclinical data highlight the potential of AG10 to treat TTR amyloid cardiomyopathy (ATTR-CM). |
All press releases naming this drug 60 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 12 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Acoramidis | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04882735 ↗ |
| Known as | AG10 | “investigational drug acoramidis (AG10) hydrochloride (HCl) 800mg administered orally twice a day” NCT04882735 ↗ |
| Known as | ALXN2060 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04622046 ↗ |
| Route | Oral | “oral treatment with ALXN2060” NCT04622046 ↗ |
| Target | TTR | “Acoramidis is a high-affinity TTR stabilizer that acts to inhibit dissociation of tetrameric TTR” PMID 38197816 ↗ Jan 2024 |