- An experimental sickle cell disease drug developed by Novartis could win U.S. approval by early next year, after the Food and Drug Administration granted the Swiss pharma's application a speedy regulatory review.
- Rather than the standard 10 months, the FDA said it would examine Novartis' case of approval of crizanlizumab in six months, Novartis announced Tuesday, setting up a January 2020 decision date.
- If approved on schedule, crizanlizumab could reach the U.S. market before Global Blood Therapeutics' experimental treatment for sickle cell disease, which the biotech expects to file later this year for an accelerated approval.
Sickle cell disease is one of the most common genetic blood disorders, but treatment options have been mostly limited to blood transfusions and hydroxyurea, a decades-old myelosuppressive drug.
While the FDA approved a pharmaceutical-grade version of l-glutamine for the condition in 2017, the need for more effective, targeted therapies remains high.
Novartis, along with Global Blood, Pfizer and Bluebird bio, aim to change that with their experimental medicines, and crizanlizumab could become the first to secure a U.S. OK.
Crizanlizumab, a monoclonal antibody that targets a molecule found on blood platelets, is designed to prevent the characteristic pain crises which often affect individuals with sickle cell disease.
In such events, sickled red blood cells stick together, creating clumps that reduce or block blood flow and, in the process, trigger vaso-occlusive crises.
Novartis' case for approval rests on results from a Phase 2 study of 111 patients which showed treatment with crizanlizumab reduced the median annual crisis rate by about one event. More than twice as many patients on Novartis' drug didn't experience any vaso-occlusive crises than those given placebo.
Importantly, no increase in infections was observed among patients in the trial. Infections are a significant contributor to both morbidity and mortality among sickle cell patients, due in part to organ damage to the spleen.
While Novartis is now on a fast track toward an approval decision, other companies in the space are advancing as well. Following the release of full results from a Phase 3 study, Global Blood now plans to submit a high dose of its drug voxelotor for accelerated U.S. approval by the end of this year.
Results show the drug increased hemoglobin levels over placebo, but data comparing the rate of vaso-occlusive crises between study groups wasn't significant.
"We don't view the drugs as direct competitors, but rather complementary treatments, given both drugs show beneficial results in different measures, and work through different mechanism of actions," wrote Cantor Fitzgerald analyst Elemer Piros in a Tuesday note to investors.
Pfizer and partner GlycoMimetics, meanwhile, are awaiting Phase 3 data this year on their drug rivipansel, also for vaso-occlusive crises.
Further out is Bluebird bio's gene therapy LentiGlobin, which in June won European approval to treat another blood disorder, beta-thalassemia. The treatment aims to deliver functional copies of a modified beta-globin gene, in theory allowing patients to produce functional, rather than sickled, red blood cells.
A Phase 1/2 study of LentiGlobin in sickle cell disease is ongoing and the biotech is planning a two-year Phase 3 trial.