Novartis sickle-cell drug gets U.S. FDA approval

By John Miller and Carl O'Donnell (Reuters) - Novartis AG on Friday won U.S. approval for its experimental sickle cell disease drug, Adakveo, making it the first of several proposed new therapies designed to offer lasting relief for patients with the debilitating blood disease to get U.S. regulatory clearance

Reuters November 16, 2019 03:06:52 IST
Novartis sickle-cell drug gets U.S. FDA approval

Novartis sicklecell drug gets US FDA approval

By John Miller and Carl O'Donnell

(Reuters) - Novartis AG on Friday won U.S. approval for its experimental sickle cell disease drug, Adakveo, making it the first of several proposed new therapies designed to offer lasting relief for patients with the debilitating blood disease to get U.S. regulatory clearance.

The biologic drug, also known as crizanlizumab, was shown in trials in its high-dose formula to cut sickle cell pain crises nearly by half to 1.63 incidents annually from 2.98 in those getting a placebo. The Food and Drug Administration's approval came nearly two months ahead of its due date for a decision in January.

Novartis has forecast that the drug's annual sales will top $1 billion (£781.49 million), much of which will likely come from U.S. government payers such as Medicare and Medicaid.

The drug is the first to offer patients with sickle cell disease targetted relief from pain crises, a common symptom of sickle cell disease, which often also causes swelling, infections, and vision problems. Current treatments, which include blood transfusions, drugs that increase haemoglobin, and pain medicines, only alleviate symptoms as they arise.

Novartis' drug may soon face competition from Voxelotor, a once daily pill developed by Global Blood Therapeutics Inc that is awaiting a decision by the FDA due in February. Voxelotor aims to treat the underlying cause of sickle cell disease by preventing sickle cells from deforming.

Other potential cures for the disease that would alter genetic mutations responsible for blood cell sickling are also undergoing clinical trials run by drugmakers including Bluebird Bio Inc and Pfizer Inc .

About 100,000 people in the United States, most of African descent, suffer from the disease in which their red blood cells are sickle-shaped, leading to episodes of intense pain, infections and deadly strokes.

(Reporting by John Miller in Zurich, Carl O'Donnell in New York and Trisha Roy in Bengaluru; Editing by Arun Koyyur and Matthew Lewis)

This story has not been edited by Firstpost staff and is generated by auto-feed.

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