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

2020-02-25 04:40:48    阅读:038427

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t patients will be infused by a healthcare provider.) FILE PHOTO: Novartis' logo is seen at the Swiss drugmaker's plant in the north

ern town of Stein, Switzerland on October 23, 2017. REUTERS/Arnd WiegmannBy 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 las

ting relief for patients with the debilitating blood disease to get U.S. regulatory clearance. The drug will be priced between $84,852 and $113,

136 per year for most patients, who will be infused by a healthcare provider with between three and four vials each month, Novartis said. The biologic drug, also known as crizanli

zumab, 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 billi

on, 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 targeted 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 t

ransfusions, drugs that increase haemoglobin, and pain medicines, only alle

viate 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 fro

m 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. “Hope has never been higher for people l香港马会资料129期 iving with sickle cell disease and their families and supporters, with a pipeline of new treatments on the horizon, like the one being approved today,” said acting FDA commissioner Brett Giroir. About 100,000 people in the United States, most of African descent, su

ffer from the d

isease in which their red blood cells are sickle-shaped, leading to episodes of intense pain, infections and deadly strokes. Novartis is targeting patients who suffer two or more pain crises per year, around 60% of the total population with the disease. According to the Centers for Medicare & Medicaid Services, 55,349 people who were covered by Medicaid, the government insurance for poor Americans, were identified with sickle cell disease. Chief Executive Vas Narasimhan has said Novartis placed a “big focus” on access in the lead-up to approval, including discussions with Medicaid. In 2018, the company launched a campaign that includes enlisting African-American actor Jordin Sparks, the 2007 “American Idol” winner, to help increase awareness in communities where people are most likely to suffer from the disease. Sickle cell disease remains most prevalent in sub-Saharan African countries, where many of the estimated 300,000 children born annually with the condition may die before the age of 5. The disease is also more common in India. Novartis’ Narasimhan last year initiated a partnership with Ghana, seeking to boost early childhood diagnosis so kids c

an get antibiotics needed to keep them alive and to help create a paediatric formulation of a cheap, older drug, hydroxyurea, used for decades to help manage sickle cell disease’s symptoms.