EMA said its review was prompted by preliminary results from the drugmaker’s ongoing global phase-3 STAND study in patients with the disease. These indicated that after one year of treatment Adakveo did not reduce the number of painful crises leading to a healthcare visit, compared with a placebo.

In October 2020, the regulator approved the drug for sickle cell disease patients ages 16 and older. EMA has stressed that the review, which it requested, is part of the marketing authorization process.

The agency said it will look at the study’s findings in the context of all available data and assess their impact on the benefit-risk balance of Adakveo in its approved indication before deciding whether marketing authorization should be amended.

The Swiss drugmaker said the study’s results do not suggest any new safety concerns for the drug, and that the results are "inconsistent" with those of a prior study called SUSTAIN. This showed that Adakveo could lower the median annual rate of the crises by 45% compared with a placebo.

Administered monthly, the drug Novartis gained with its $665 million acquisition of Selexys Pharmaceuticals in 2016 is aimed at reducing pain crises associated with sickle cell disease by inhibiting the P-selectin protein.

Despite “high uncertainty” about its long-term effectiveness and cost-effectiveness, the UK’s National Institute for Heath and Care Excellence (NICE) in 2021 inked a deal with Novartis to sell Adakveo in England as the first new therapy for sickle cell disease in 20 years. The terms were said to include an unquantified discount.

Keeping the drug on the market could be crucial to the Basel-based pharma giant, analysts said, in particular as it now has a competitor in Pfizer, which recently bought Global Blood Therapeutic (GBT). That company developed a sickle cell disease-related medicine called Oxbryta that is approved in the EU to treat hemolytic anemia in sickle cell disease patients aged 12 and older.

Oxbryta, however, has cost issues of its own. In 2020, US drug pricing watchdog, Institute for Clinical and Economic Review (ICER), said the sickle cell disease drugs from GBT, Novartis and Emmaus Medical  were all too expensive to meet traditional cost-effectiveness measures, and the companies should “dramatically cut” their prices.

Author: Dede Williams, Freelace Journalist