FDA gives sped-up review to Novartis’ sickle cell disease drug

The FDA granted priority review to crizanlizumab as a treatment for preventing painful, life-threatening events in sickle cell disease patients.

The Food and Drug Administration has given priority review to an approval application from Novartis for a drug to treat sickle cell disease.

The Swiss drugmaker said Tuesday that the FDA had accepted its filing for crizanlizumab as a treatment to prevent vaso-occlusive crises in patients with the disease. VOCs, also known as sickle-cell pain crises, are unpredictable and extremely painful events that can be life-threatening and are the most common cause of emergency room visits and hospital stays for patients with the disease.

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A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.

The submission is based on data from the Phase II SUSTAIN study, which showed that treatment with crizanlizumab reduced the median annual rate of VOCs leading to healthcare visits by 45.3 percent compared with placebo. Data presented at the American Society of Hematology’s annual meeting last year included 132 patients in the intent-to-treat (ITT) population – meaning all of those who were randomized – and 81 in the per-protocol (PP) population, meaning patients who were able to follow the standard treatment regimen. Overall, 35.8 percent and 37.5 percent of patients receiving crizanlizumab in the respective ITT and PP populations did not experience any VOCs while on treatment, compared with 16.9 percent and 12.2 percent of those on placebo.

Novartis is also running two additional Phase II studies of crizanlizumab – one in children and one in patients aged 16 and older to assess things like pharmacological properties, safety, dosing and efficacy. A Phase III study, STAND, is investigating the drug’s safety and efficacy in patients aged 12 and older.

Novartis took control of crizanlizumab through its November 2016 acquisition of Selexys Pharmaceuticals Corp., which had originally sponsored the SUSTAIN trial under a different company name. The deal was worth up to $665 million in upfront, acquisition and milestone payments.

In a note to investors that gave an overview of therapies in development for sickle cell disease last month, based on an interview with a physician who treats sickle cell disease, Cowen analyst Yaron Weber wrote that crizanlizumab has compelling data that are likely to lead to the drug’s approval in adults. The note also included the physician’s views on bluebird bio’s gene therapy LentiGlobin, Global Blood Therapeutics’ voxelotor and Pfizer and Glycomimetics’ rivipansel.

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The European Medicines Agency approved LentiGlobin, under the brand name Zynteglo, for beta-thalassemia in June. Meanwhile, Global Blood Therapeutics plans to complete a rolling submission to the FDA of data for voxelotor in the second half of this year, though the Cowen note cited the physician as saying the drug needs more meaningful data to demonstrate efficacy and safety in the real world, outside of clinical trials.

Photo: MilosJokic, Getty Images