As­traZeneca's Farx­i­ga gets rough FDA re­view date for ground­break­ing move in­to chron­ic kid­ney dis­ease

As­traZeneca’s Farx­i­ga broke ground last year with an FDA nod for heart fail­ure pa­tients with or with­out type 2 di­a­betes — a first-in-class ap­proval. But Farx­i­ga isn’t done in­no­vat­ing and is now look­ing at a ap­proval in the com­ing months in an­oth­er po­ten­tial block­buster in­di­ca­tion.

The FDA will re­view As­traZeneca’s ap­pli­ca­tion to take SGLT2 in­hibitor Farx­i­ga in­to chron­ic kid­ney dis­ease some­time in the sec­ond quar­ter af­ter tag­ging the drug for pri­or­i­ty re­view, the British drug­mak­er said Wednes­day. CKD is be­lieved to cur­rent­ly af­fect rough­ly 37 mil­lion US pa­tients, As­traZeneca said.

Three months af­ter des­ig­nat­ing Farx­i­ga as a break­through ther­a­py in CKD pa­tients with or with­out type 2 di­a­betes, the FDA will now for­mal­ly speed its ap­pli­ca­tion in that in­di­ca­tion for what would be a first-in-class ap­proval. While John­son & John­son’s SGLT2 ri­val In­vokana sports an FDA ap­proval in di­a­bet­ic kid­ney dis­ease, a po­ten­tial nod for Farx­i­ga would make it the first SGLT2 on the mar­ket for kid­ney pa­tients with­out di­a­betes.

The FDA gave Farx­i­ga the break­through tag af­ter out­comes da­ta un­veiled in Au­gust at the Eu­ro­pean So­ci­ety of Car­di­ol­o­gy an­nu­al meet­ing showed Farx­i­ga cut the com­bined risk of wors­en­ing kid­ney func­tion, end-stage kid­ney dis­ease on­set, and kid­ney dis­ease- or car­dio­vas­cu­lar-re­lat­ed death by 39% over place­bo in pa­tients with CKD with or with­out type 2 di­a­betes.

In what As­traZeneca called “par­a­digm-shift­ing” find­ings, Farx­i­ga’s Da­pa-CKD out­comes tri­al showed the drug al­so sliced the risk of all-cause mor­tal­i­ty by 31% over place­bo, a key sec­ondary end­point. Those re­sults were con­sis­tent across pa­tients re­gard­less of whether they had a di­a­betes di­ag­no­sis.

As­traZeneca re­leased its top-line find­ings in Ju­ly af­ter halt­ing the tri­al ear­ly in March, when in­ter­im da­ta “showed Farx­i­ga’s ben­e­fits ear­li­er than orig­i­nal­ly an­tic­i­pat­ed,” the drug­mak­er said at the time.

If Farx­i­ga does cross the fin­ish line in CKD, it would rep­re­sent a sec­ond ap­proval once thought un­like­ly for a drug class de­vel­oped for di­a­betes.

In May, the FDA ap­proved Farx­i­ga to re­duce the risk of car­dio­vas­cu­lar death or hos­pi­tal­iza­tion in heart fail­ure pa­tients with a re­duced ejec­tion frac­tion (HFrEF) and with or with­out type 2 di­a­betes, an­oth­er first in the SGLT2 class.

Farx­i­ga got the OK to treat rough­ly six mil­lion US HFrEF pa­tients each year, ex­pand­ing on its ear­li­er FDA ap­provals to treat heart fail­ure pa­tients with type 2 di­a­betes and to im­prove glycemic con­trol in type 2 di­a­betes pa­tients.

That ap­proval fol­lowed an FDA pri­or­i­ty re­view grant­ed in Jan­u­ary 2020 and a fast-track des­ig­na­tion in Sep­tem­ber 2019. The drug al­so sports a fast-track re­view in heart fail­ure with a pre­served ejec­tion frac­tion (HF­pEF) — an in­di­ca­tion where No­var­tis’ En­tresto, a com­bi­na­tion of sacu­bi­tril and val­sar­tan, earned the FDA’s nod in De­cem­ber as a first ap­proved ther­a­py.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,200+ biopharma pros reading Endpoints daily — and it's free.

An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,200+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,200+ biopharma pros reading Endpoints daily — and it's free.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Covid-19 roundup: Italy won­ders aloud if it can sue Pfiz­er for vac­cine short­falls; Flood, dead­ly fire threat­en As­traZeneca vac­cine plants

As reports crop up that deliveries of Pfizer and BioNTech’s Covid-19 vaccine are being unexpectedly cut, Italy wonders if it can take the vaccine developers to court, according to the Wall Street Journal. 

After its shipment for this week was cut by 29%, the Italian government consulted its attorney general about taking legal action, the WSJ reported. Pfizer and BioNTech had warned the EU and Canada last week that their allocations would be reduced as Pfizer upgrades its Belgium factory. What Italy says it doesn’t appreciate, though, is the short notice.

Mike Grey, Plexium chairman (Horizon Therapeutics)

Plex­i­um adds in­dus­try vet Mike Grey to the brain trust with new in­vestor cash fund­ing its pro­tein degra­da­tion play

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.