US clears Novartis' anti-P-selectin antibody Adakveo to reduce rate of sickle cell-related pain crises

Novartis announced Friday that the FDA has approved its filing for Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs) in adult and paediatric patients aged 16 years and older with sickle cell disease (SCD). The decision makes Adakveo, previously known as SEG101, the first targeted therapy approved for SCD, specifically inhibiting the P-selectin cell adhesion protein that plays a key role in VOCs, according to the FDA.

The drug is expected to be available in the coming weeks, with Susanne Schaffert, president of Novartis Oncology, saying Adakveo "marks a new era in the treatment of SCD." The price is set between $84,852 and $113,136 per year for most patients, who will typically infuse themselves with three to four vials each month, the company said. Novartis has forecast that the drug's annual sales could top $1 billion, much of which will likely come from Medicare and Medicaid.

The filing was supported by data from the randomised Phase II SUSTAIN trial, which involved 198 patients with any genotype of SCD and a history of two to 10 VOCs in the previous 12 months. Adakveo was administered over a period of 30 minutes by intravenous infusion at baseline, week two, and then every four weeks thereafter, for a treatment duration of 52 weeks. The primary efficacy outcome was the annual rate of VOCs leading to a healthcare visit.

According to the study results, Adakveo significantly lowered the median annual rate of VOCs to 1.63, compared to 2.98 for placebo, translating to a reduction of 45%. The data also showed a 42% decrease in the median annual rate of days hospitalised from 6.87 days to 4 days. Meanwhile, a post-hoc analysis has demonstrated that 36% of Adakveo-treated patients did not experience a VOC, compared to 17% for placebo, while the median time to first VOC was 4.1 months and 1.4 months, respectively.

Global Blood Therapeutics' SCD drug candidate voxelotor is currently under priority review with the FDA, with a decision expected in February. The company has said that if approved, the once-daily oral therapy would be the first that targets haemoglobin polymerisation, the root cause of SCD damage.

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