Attruby™: A New Milestone in Cardiovascular Treatment
As of February 17, 2025, a significant milestone has been reached in the medical community with 1,028 unique patient prescriptions for Attruby™, the first and only near-complete TTR stabilizer, having been written by 516 unique prescribers since its FDA approval. This groundbreaking drug, known chemically as acoramidis, was approved by the Food and Drug Administration (FDA) on November 22, 2024, for reducing cardiovascular death and cardiovascular-related hospitalization in ATTR-CM (atrioventricular transthyretin-related cardiomyopathy) patients.
European Approval and Commercial Success
The European Medicines Agency (EMA) approved acoramidis as BEYONTTRA™ on February 10, 2025. This approval marked a $75 million milestone payment for the company and ongoing royalties in a tiered structure beginning in the low-thirties percent on sales in the European Union. The success of Attruby™ in Europe is a testament to its efficacy in treating ATTR-CM patients, who often face an aggressive phenotype and poor prognosis.
Clinical Trials and Statistical Significance
In clinical trials, acoramidis demonstrated a 59% hazard reduction on the composite endpoint of all-cause mortality and first cardiovascular-related hospitalization in the variant ATTR-CM population by month 30. To the company’s knowledge, this benefit is the largest and the only statistically significant result in this patient population. This significant reduction in risk is expected to greatly improve the quality of life for those suffering from ATTR-CM.
Ongoing Clinical Trials
The company has fully enrolled three global registrational studies – FORTIFY (BBP-418 for LGMD2I/R9), CALIBRATE (encaleret for ADH1), and PROPEL 3 (infigratinib for achondroplasia) – with the last participant-last visit expected for each study before the end of 2025.
Financial Overview
The company ended the fourth quarter with $681 million in cash, cash equivalents, and short-term restricted cash. Furthermore, the company expects to receive $105 million in regulatory milestones in the first half of 2025 from acoramidis Europe and Japan approvals.
Impact on Individuals
For individuals diagnosed with ATTR-CM, the approval of Attruby™ brings new hope. This drug, which stabilizes the transthyretin protein, reduces the risk of cardiovascular death and hospitalization, significantly improving their quality of life. While the financial implications of this approval may not directly impact individuals, the potential for increased research and development in cardiovascular treatments could lead to better outcomes for all patients.
Impact on the World
The approval of Attruby™ is a significant step forward in the world of cardiovascular treatments. Its ability to reduce the risk of cardiovascular death and hospitalization in ATTR-CM patients has the potential to save countless lives and improve the quality of life for thousands. Furthermore, the success of Attruby™ could pave the way for further advancements in the understanding and treatment of cardiovascular diseases.
Conclusion
In summary, the approval of Attruby™ (acoramidis) marks a major milestone in the medical community, with 1,028 unique patient prescriptions written since its FDA approval. Its European approval has brought commercial success and ongoing royalties for the company. The drug’s 59% hazard reduction in clinical trials has demonstrated significant benefits for ATTR-CM patients, who often face an aggressive phenotype and poor prognosis. With ongoing clinical trials and a strong financial position, the future looks bright for this groundbreaking drug and the cardiovascular community as a whole.
- Attruby™ (acoramidis) approved by FDA in November 2024 for reducing cardiovascular death and hospitalization in ATTR-CM patients.
- European approval of acoramidis as BEYONTTRA™ in February 2025 brings commercial success and ongoing royalties.
- 59% hazard reduction in clinical trials for the composite endpoint of all-cause mortality and first cardiovascular-related hospitalization in variant ATTR-CM population.
- Three global registrational studies ongoing with last participant-last visit expected before the end of 2025.
- Company ended Q4 with $681 million in cash, cash equivalents, and short-term restricted cash.
- Expected $105 million in regulatory milestones in 1H 2025 from European and Japanese approvals.
- Impact on individuals: Improved quality of life for ATTR-CM patients.
- Impact on the world: Potential for significant advancements in cardiovascular treatments.
