US approves new drug to manage sickle cell disease

U.S. reg­u­la­tors on Fri­day approved a new med­i­cine that can help reduce extreme­ly painful sick­le cell dis­ease flare-ups.

The Food and Drug Admin­is­tra­tion approved Novar­tis AG’s Adakveo for patients 16 and old­er. The month­ly infu­sion, which halves occur­rences of sick­le cell pain episodes, will car­ry a list price of rough­ly $85,000 to $113,000 per year, depend­ing on dos­ing. Insured patients gen­er­al­ly will pay less.

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Sick­le cell dis­ease is one of the most com­mon inher­it­ed blood dis­or­ders, about 100,000 Amer­i­cans, most of them , and about 300 mil­lion peo­ple world­wide.

Its hall­mark is peri­od­ic episodes in which red blood cells stick togeth­er, block­ing blood from reach­ing organs and small blood ves­sels. That caus­es intense pain and cumu­la­tive organ dam­age that short­ens the lives of peo­ple with the dis­ease.

“The dura­tion and sever­i­ty of these pain crises wors­ens with aging. Often patients die dur­ing one of these crises,” said Dr. Biree Ande­mari­am, chief med­ical offi­cer of the Sick­le Cell Dis­ease Asso­ci­a­tion of Amer­i­ca.

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Novar­tis’s Adakveo (Novar­tis via AP)

Ande­mari­am, a for­mer Novar­tis advi­so­ry , said the drug appears to work bet­ter the longer patients receive it.

The Swiss drug­mak­er is con­tin­u­ing patient test­ing to deter­mine whether Adakveo, also known as crizan­l­izum­ab, length­ens patients’ lives, said Ameet Mallik, the com­pa­ny’s of U.S. oncol­o­gy and blood dis­or­ders.

He said severe pain episodes send U.S. patients to emer­gency depart­ments about 200,000 times per year. About 85% are hos­pi­tal­ized for days to a , run­ning up big bills.

The debil­i­tat­ing con­di­tion also caus­es ane­mia, delayed growth, vision dam­age and painful swelling in hands and feet, it hard for some peo­ple to main­tain jobs or attend school.

Cur­rent treat­ments include a 21-year-old can­cer drug called hydrox­yurea and Endari, approved in 2017.

In patient stud­ies, Endari reduced fre­quen­cy of pain episodes about 25% and hydrox­yurea reduced them by half. Hydrox­yurea can seri­ous effects and requires week­ly blood tests. Both drugs have com­pli­cat­ed dos­ing and don’t work — or stop work­ing — in some patients.

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In a one-year study of 198 patients, those get­ting the high­er of two Adakveo dos­es aver­aged 1.6 pain episodes over that year and 36% had none. A com­par­i­son group on place­bo aver­aged three pain episodes that year and 17% had none. Adakveo’s side effects includ­ed influen­za and high fever.

Danielle Jami­son, of Island­ton, South Car­oli­na, has with sick­le cell pain episodes since short­ly after birth. The 35-year-old pre­vi­ous­ly had a half-dozen pain crises requir­ing hos­pi­tal trips each year. Those less­ened by about half when she began tak­ing hydrox­yurea nine years ago.

She has­n’t been in the hos­pi­tal since she start­ed tak­ing crizan­l­izum­ab two years ago as part of a patient study. She still has mild dai­ly pain, but she said she can now take care of her home and dri­ve her 9‑year-old daugh­ter to activ­i­ties.

“It’s made a huge dif­fer­ence in how much I’m able to do,” Jami­son said.

All three drugs work through dif­fer­ent mech­a­nisms, so doc­tors may switch patients to Adakveo or to add it to their cur­rent treat­ment, said Ande­mari­am, head of Uni­ver­si­ty of Con­necti­cut’s sick­le cell treat­ment and research pro­gram.

Mean­while, numer­ous drugs to treat sick­le cell dis­ease and gene ther­a­pies to pos­si­bly cure it are being test­ed.

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