Novartis' crizanlizumab wins speedy FDA review, putting it closer to blockbuster sickle cell launch

Novartis’ plan to launch over 10 potential blockbuster drugs or new indications by 2021 is quickly unfolding.

The FDA has accepted a biologics application for the pharma giant's sickle cell disease candidate crizanlizumab (SEG101) and put the drug under priority review, which slashes four months off its evaluation time to just six months in total, the company said Tuesday.

If approved, it could sit alongside multiple sclerosis drug Mayzent, spinal muscular atrophy gene therapy Zolgensma and age-related macular degeneration candidate brolucizumab—which also under priority review—among the Swiss drugmaker's most important launches these days. But several competitors are not far behind.

Crizanlizumab, a drug Novartis got via its acquisition of Selexys Pharmaceuticals in 2016, is designed to prevent vaso-occlusive crises (VOCs), the painful events resulting from blood vessel blockage in sickle cell disease patients. The drug reduces clusters of cells that block blood flow by targeting the P-selectin protein.

In a phase 2 study, crizanlizumab, given once monthly, cut the median rate of VOCs that required healthcare visits by 45.3% in a year versus placebo. What’s more, more than double the number of crizanlizumab patients didn’t experience any VOCs at all. The reductions were seen in patients with or without the use of the old standard treatment hydroxyurea, according to Novartis.

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Novartis has a couple of small players to beat, though. First, there’s Emmaus Life Sciences’ (previously Emmaus Medical) Endari, which two years ago became the first FDA-approved treatment for sickle cell disease in about two decades. The drug is an oral powder formulation of amino acid L-glutamate that’s given orally twice daily to reduce the acute complications of sickle cell disease. For 2018, the small California biotech registered Endari net sales of $14.6 million.

Closely trailing Novartis is Global Blood Therapeutics with its voxelotor, which works for stabilizing hemoglobin. According to recently unveiled phase 3 data from a study of 274 patients, 60% of patients in the higher-dose arm had a greater than 1 g/dL increase in hemoglobin, compared with 9% in the placebo arm.

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Besides the small size of GBT, voxelotor also looks like a minor problem for Novartis in that the drug didn’t show a strong reduction in VOCs despite a numerical trend in favor of it over placebo. Still, the FDA has agreed to an accelerated approval pathway using hemoglobin as the primary endpoint, the company said recently, adding that it plans to file a new drug application in the second half of this year.

Pfizer and partner Glycomimetics have rivipansel in phase 3, but it’s for treating patients already hospitalized with VOCs, rather than preventing the episodes—meaning it won’t compete directly with crizanlizumab.

However, there’s a potential threat to all these sickle cell drugs: Bluebird Bio’s gene therapy Zynteglo. An EU nod in hand, the drug could get an FDA go-ahead to treat beta-thalassemia soon. It’s in clinical trials for severe sickle cell disease, and early findings suggest it might provide long-lasting effects in producing normal red blood cells.