Acoramidis

The drug and biotech sector has outperformed the broader market, driven by new drug launches, pipeline successes, and increased M&A activity. Eli Lilly's obesity drugs have been particularly successful. Despite headwinds like pipeline setbacks and economic uncertainty, the sector has seen a surge in investments. Five large drugmakers—Pfizer, Eli Lilly, AstraZeneca, Bayer, and Sanofi—are recommended for portfolio growth, with strong financials and robust pipelines.

BridgeBio Pharma's infigratinib receives FDA Breakthrough Therapy Designation for treating achondroplasia, with PROPEL 2 trial showing significant height velocity increase. Ongoing PROPEL 3 study aims for completion by year-end. BridgeBio's diverse pipeline includes other genetic diseases, and recent developments include expedited review for BBP-812 gene therapy and discontinuation of BBP-631 program. Analysts maintain positive ratings, highlighting anticipated sales growth despite stock volatility.

BridgeBio, founded in 2015, has two products on the market and another awaiting FDA decision by November. The company is spinning out assets into new entities, discontinuing a gene therapy program, and focusing on late-stage programs in transthyretin amyloid cardiomyopathy and achondroplasia. BridgeBio aims to have six approved products by its tenth birthday, with a focus on commercializing its later-stage pipeline. The company’s success hinges on the approval and market performance of acoramidis for ATTR-CM and infigratinib for achondroplasia.

In 2023, FDA's CDER approved 55 new drugs, a five-year high. Key 2024 drug launches include KarXT for schizophrenia, Donanemab for Alzheimer's, Resmetirom for NASH, Sotatercept for PAH, Datopotamab deruxtecan for lung & breast cancer, Acoramidis for ATTR-CM, mRNA-1345 for RSV, Anktiva for NMIBC, Ensifentrine for COPD, and Imetelstat for MDS.

Acoramidis (Attruby) treats cardiomyopathy by stabilizing transthyretin, mimicking the T119M mutation's protective effects. Approved in the US in November 2024, it reduces cardiovascular death and hospitalization in ATTR-CM patients. Common side effects include diarrhea and abdominal pain. Demonstrated efficacy in clinical trials, showing significant benefits over placebo.