Fulcrum Therapeutics considers sale after FDA blocks sickle cell drug development

Fulcrum Therapeutics announced it is undertaking a strategic review that could lead to a sale after its lead sickle cell candidate, pociredir, was effectively blocked by the FDA.

The agency cited safety concerns, noting higher-than-expected rates of secondary blood cancers seen with a similar drug, Ipsen’s Tazverik, and said the risk profile was unacceptable, leaving no viable regulatory pathway.

Fulcrum said it will discontinue development of pociredir, calling the decision very difficult, and will explore options for the company’s future, including a possible acquisition.

The setback joins recent challenges for other sickle cell drug developers such as Pfizer, Novartis and Intellia Therapeutics, underscoring the regulatory hurdles in this therapeutic area.