Acoramidis

In 2024, the European Medicines Agency received a record number of marketing authorization applications for advanced therapy medicinal products. Notable filings include UCB’s orphan drug for thymidine kinase 2 deficiency and Novo Nordisk’s Alhemo for hemophilia. The FDA approved 50 novel agents and 11 biologics. EMA recommended several new products for EU approval, including treatments for hereditary angioedema and transthyretin amyloidosis. Sanofi’s rilzabrutinib and Alvotech/Advanz Pharma’s golimumab are under review. EMA also reconsidered Eisai/Biogen’s Alzheimer’s drug for approval. The UK approved Eli Lilly’s Alzheimer’s drug, despite NICE's reservations.

FDA approves BridgeBio Pharma's acoramidis (Attruby) for ATTR-CM, setting up a market competition with Pfizer's Vyndamax/Vyndaqel/Vynmac. Attruby, priced at $18,759 for a 28-day supply, aims to reduce cardiovascular death and hospitalization in ATTR-CM patients. BridgeBio plans to pursue regulatory approvals in Europe, Japan, and Brazil. Analysts predict potential peak sales of $2 billion or more for Attruby.

Alleviant Medical's atrial shunt treats heart failure without permanent implants, receiving FDA breakthrough designation. Emory University Hospital implanted the BrioVAD System, enhancing quality of life. Acoramidis, a new FDA-approved drug, treats transthyretin amyloid cardiomyopathy. Deepak Bhatt highlights AI-enhanced cardiac screening and coronary inflammation's growing importance.

FDA approved acoramidis for reducing cardiovascular death and hospitalization in ATTR-CM patients, based on positive Phase 3 study results showing a win ratio of 1.8. Dr. Martha Grogan highlighted its potential to make ATTR-CM a manageable condition.

The FDA approved acoramidis (Attruby), a drug developed at Stanford Medicine for treating ATTR-CM, a rare heart condition caused by misfolded proteins. Created by former Stanford researchers, it prevents protein misfolding, improving survival and quality of life. Its development highlights academic-industry collaboration's role in advancing rare disease treatments.

In 2024, the FDA approved 50 new drugs, focusing on cancer, with 15 approvals. Small molecules led with 32 approvals, followed by proteins and oligonucleotides. Notable approvals included a schizophrenia treatment and a NASH drug, marking significant advancements in therapeutic areas.

BridgeBio Pharma, with a $5.3B market cap, focuses on genetic diseases. Its FDA-approved Acoramidis could significantly boost revenue. Analysts rate it a 'Strong Buy', predicting a 150% stock increase to $70, despite a 31.5% YTD drop. The company faces competition and R&D challenges but has a promising pipeline.

The FDA's 2024 approvals include 48 novel drugs, targeting conditions like familial chylomicronemia syndrome and non-small cell lung cancer, alongside diagnostic tools, marking significant progress in oncology, cardiology, and rare diseases, enhancing patient care and addressing unmet medical needs.

RLY-2608, an oral, mutant-selective PI3Kα allosteric inhibitor, addresses off-target toxicities of current modulators, currently in Ph. I for HR+/HER2- breast cancer treatment.