The week in pharma: action, reaction and insight – week to December 15, 2023

By Barbara Obstoj-Cardwell. Editor

Regulatory news last week saw the US Food and Drug Administration (FDA) make a land-mark decision to approve the first gene therapies for sickle cell disease (SCD) – Vertex Pharmaceuticals and CRISPR’s Casgevy and bluebird bio’s Lyfgenia. Also of note, the FDA announced the creation of a new advisory committee for genetic metabolic diseases. M&A and deal-making news saw the UK’s AstraZeneca reveal a $1.1 billion takeover bid for Icosavax, aiming to strengthen its late-stage vaccine pipeline. Additionally, USA-based C4 Therapeutics announced a license and research collaboration with pharma giant Merck & Co on degrader antibody conjugates. On the research front, Vertex reported compelling Phase II data on VX-548, its non-opioid treatment for diabetic peripheral neuropathy (DPN).

Two approvals for SCD, but black box and no PRV put bluebird in the hot seat