- British drugmaker GlaxoSmithKline plans to exit rare disease drug development as part of a major revamp of R&D under new CEO Emma Walmsley, putting the business up for sale a little over a year after winning approval for only the second commercial gene therapy for an inherited disease.
- In a quarterly update yesterday, GSK said it had conducted a strategic review of the unit and is actively considering options for "future ownership" of the assets.
- Walmsley did indicate GSK would put its cell and gene therapy technology platform to work supporting cancer research. Still, the move is a retreat from a field that appears close to notching significant advances after years of setbacks.
GSK's decision underscores the difficulty drugmakers can face in attempting to commercialize seeming "cures."
The company won approval in Europe last year for Strimvelis, an ex vivo stem cell therapy designed to treat a rare genetic condition called ADA-SCID but better known as the "bubble boy" disease.
Despite impressive clinical trial results, only two patients ever received Strimvelis, according to a report from Reuters. The limited number of patients affected by ADA-SCID each year in Europe meant Strimvelis' commercial ceiling was always limited. But for a therapy that notched a 100% survival rate three years post-treatment, the slow uptake stands out.
Strimvelis' roughly $665,000 upfront cost also makes it one of the more expensive treatments in the world, even with a money-back guarantee that GSK made to ease worries the treatment effect may not persist.
In addition to Strimvelis, GSK's rare disease business includes several other rare disease therapies currently in clinical development. The company has four compounds in late-stages including treatments in development for transthyretin-mediated amyloidosis — an abnormal buildup of protein, metachromatic leukodystrophy — a lysosomal storage disease, and Wiscott-Aldrich syndrome — a recessive genetic disease.
It is easy to see, though, why GSK would look to offload the unit amid a broader trimming of its pipeline. Respiratory and HIV are mainstays, and the company is also targeting oncology and inflammation as priority areas of investment. Focusing investment means cuts and rare disease hasn't been GSK's calling card.
"We're going to have fewer and more focused priorities for GSK," Walmsley said on the July 26 earnings call, noting the company hasn't consistently translated R&D output into commercial successes.
Strimvelis is still a premiere gene therapy asset, though, and other buyers in biotech could see value in adding GSK's business — depending on the price.