Blue­bird’s new gene ther­a­py among three new EMA OKs ahead of FDA

Ahead of a usu­al­ly speedy FDA, the Eu­ro­pean Med­i­cines Agency on Fri­day rec­om­mend­ed mar­ket­ing au­tho­riza­tions for blue­bird bio’s new gene ther­a­py for chil­dren with a rare, in­her­it­ed neu­ro­log­i­cal dis­ease, Glax­o­SmithK­line and Vir’s mon­o­clon­al an­ti­body treat­ment for Covid-19, and a new treat­ment for a rare liv­er dis­ease.

In the case of blue­bird’s Skysona (eli­v­al­do­gene au­totem­cel), the gene ther­a­py is for those younger than 18 who have what’s known as ear­ly Cere­bral Adrenoleukody­s­tro­phy (CALD). If un­treat­ed, near­ly half of those with CALD die with­in 5 years of symp­tom on­set, EMA said.

EMA’s rec­om­men­da­tion is based on a sin­gle-arm tri­al in 32 boys with CALD (the dis­or­der is al­most ex­clu­sive­ly seen in males) who re­ceived the one-time treat­ment, com­pared to 59 boys who had a stem cell trans­plan­ta­tion.

“An analy­sis con­duct­ed af­ter 24 months from the in­fu­sion on 30 sub­jects en­rolled in the study con­clud­ed that for 27 of them (90%) treat­ment with Skysona pre­served mo­tor func­tion and com­mu­ni­ca­tion abil­i­ty and im­proved sur­vival when com­pared to un­treat­ed pa­tients at an ear­ly stage of cere­bral dis­ease,” the EMA said.

Richard Colvin

Blue­bird bio said it is cur­rent­ly on track to sub­mit the Bi­o­log­ics Li­cense Ap­pli­ca­tion to the FDA by mid-2021.

Richard Colvin, in­ter­im CMO at blue­bird, said in a state­ment, “This pos­i­tive opin­ion from the CHMP marks the first reg­u­la­to­ry ap­proval rec­om­men­da­tion for any gene ther­a­py for CALD, bring­ing us clos­er to a one-time, durable treat­ment op­tion that sta­bi­lizes neu­ro­log­i­cal dis­ease while re­duc­ing the risk of the se­ri­ous im­mune com­pli­ca­tions as­so­ci­at­ed with al­lo­gene­ic stem cell trans­plan­ta­tion (al­lo-HSCT), which is the on­ly ther­a­peu­tic op­tion for chil­dren with this dev­as­tat­ing dis­ease.”

GSK and Vir mAb

The EMA on Fri­day al­so con­clud­ed that GSK’s and Vir’s mAb Covid-19 treat­ment, known as sotro­vimab, can be used to treat con­firmed Covid-19 in adults and ado­les­cents who don’t re­quire sup­ple­men­tal oxy­gen ther­a­py and are at risk of pro­gress­ing to se­vere Covid-19.

EMA said an in­ter­im analy­sis of the in­fu­sion in­di­cat­ed that sotro­vimab re­duced the risk of hos­pi­tal­iza­tion for more than 24 hours or death by 85% com­pared with place­bo. Hos­pi­tal­iza­tion for more than 24 hours or death oc­curred in 1% (3 out of 291) of pa­tients who re­ceived sotro­vimab and 7% (21 out of 292) of those who re­ceived place­bo.

EU mem­ber states can use the pos­i­tive sci­en­tif­ic opin­ion when mak­ing na­tion­al de­ci­sions about the ear­ly use of sotro­vimab pri­or to EMA mar­ket­ing au­tho­riza­tion. A rolling re­view of the treat­ment is on­go­ing, as of May 7, and will be the ba­sis for a mar­ket­ing au­tho­riza­tion, EMA said.

The com­pa­nies said they al­so sub­mit­ted an emer­gency use au­tho­riza­tion ap­pli­ca­tion for the mAb to the FDA, and Health Cana­da is al­so re­view­ing it.

Al­bireo’s rare liv­er dis­ease drug

The EMA al­so rec­om­mend­ed grant­i­ng mar­ket­ing au­tho­riza­tion to Al­bireo’s Byl­vay (ode­vix­i­bat) for the treat­ment of Pro­gres­sive Fa­mil­ial In­tra­hep­at­ic Cholesta­sis (PF­IC) in pa­tients aged 6 months or old­er.

PF­IC is a rare, life-threat­en­ing liv­er dis­ease and on­ly about half of the chil­dren af­fect­ed by the dis­ease sur­vive be­yond the age of 10 years.

Re­sults from a Phase III tri­al showed a sig­nif­i­cant re­duc­tion in serum bile acids ac­com­pa­nied by a sig­nif­i­cant re­duc­tion in pru­ri­tus in those treat­ed with ode­vix­i­bat.

“These re­sults were main­tained in an on­go­ing, long-term open-la­bel fol­low-up study. He­pat­ic pa­ra­me­ters and fi­bro­sis scores were im­prov­ing or were sta­ble for the du­ra­tion of the study (max. 72 weeks). How­ev­er, more da­ta are need­ed to de­ter­mine if ode­vix­i­bat can de­lay dis­ease pro­gres­sion and the need for liv­er trans­plan­ta­tion. The CHMP there­fore re­quest­ed a reg­istry-based ef­fi­ca­cy study as a fol­low-up,” EMA said.

The FDA has un­til Ju­ly 20 to de­cide on whether to ap­prove the drug, and the com­pa­ny said an ad­vi­so­ry com­mit­tee meet­ing is not ex­pect­ed. If ap­proved, Al­bireo al­so would win a lu­cra­tive rare pe­di­atric dis­ease pri­or­i­ty re­view vouch­er.

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

Fred Upton and Diana DeGette

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House Energy & Commerce leaders Fred Upton (R-MI) and Diana DeGette (D-CO) on Tuesday released new draft legislation with wide-ranging implications for public health, the FDA, NIH, and that would create a new, $6.5 billion federal advanced research agency under NIH, with an aim to cure cancer, Alzheimer’s and other difficult diseases.

Similar to DARPA, the new NIH division to be known as ARPA-H, would be run by a small group of program managers with more latitude to pursue high-risk, high-reward projects that other government agencies would likely shy away from.

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On the hunt for the next Mod­er­na, in­vestors have pumped 'plat­form plays' with cash. Can any­thing slow the run­away train?

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Sen. Ron Wyden (D-OR) with reporters in the Senate Subway (Graeme Sloan/Sipa via AP Images)

Top Wyden pri­or­i­ty for drug price re­forms: Medicare ne­go­ti­a­tions

As the Biden administration tries to wrangle the details of its infrastructure bill, Senate Finance Committee Chair Ron Wyden (D-OR) took a concrete step forward on drug pricing reforms on Tuesday and unveiled five principles for such reforms, including providing Medicare with the ability to negotiate prices.

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Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

Mer­ck­'s Keytru­da blazes a path in first-line cer­vi­cal can­cer, mak­ing good on drug­mak­er's push for ear­li­er pa­tients

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Top an­a­lyst chimes in on bub­bling de­bate about Glax­o­SmithK­line's fu­ture with a clear mes­sage: Let your £40B vac­cines group go

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Bio­gen's new Alzheimer's drug serves as a ral­ly­ing cry for drug pric­ing re­forms

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End­points News is now 5 years old. Here's how you can sup­port us for the next phase of growth

Endpoints News turned five years old over the weekend. I wanted to mark the happy occasion by extending our deepest gratitude to Endpoints’ premium subscribers while outlining several other ways to support us as we go broader and get bigger this year and beyond.

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