A banner year for pharma

As 2018 draws to a close, the pharmaceutical industry is celebrating a prosperous year of new investments and therapeutic breakthroughs. These successes were driven by cutting-edge science and progress in finally translating long-standing technology into actual products, according to a cover story in Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society.

The biotech industry benefited from a wave of investment in 2018 from venture capital firms, writes Senior Correspondent Lisa M. Jarvis. In addition, biotech initial public offerings hit a new record. Promising developments during the year include the approval of the first RNA interference drug, advancements for the gene-editing tool CRISPR and the first crop of cannabis-based therapeutics.

In August, the U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals' Onpattro, the first RNA interference therapeutic to reach the market. In development for 16 years, Onpattro treats a rare genetic disorder called hereditary transthyretin-mediated amyloidosis. Interest in other RNA therapeutics, such as antisense oligonucleotides and mRNA vaccines, is also flourishing. Meanwhile, although CRISPR has suffered recent setbacks related to possible safety issues, companies are forging ahead with clinical trials planned in 2019 for sickle cell disease, a rare genetic form of blindness and liver disorders. 2018 also witnessed the first FDA approval of a cannabis-based drug: Epidiolex, made by GW Pharmaceuticals, treats rare seizure disorders in children and young adults. Other companies are following suit with clinical development programs targeting cannabinoid receptors.