Levi Garraway, Roche CMO (Source: Genentech)

FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US reg­u­la­tors hand­ed Roche and Genen­tech a big win Fri­day af­ter­noon, one that has mar­ket-shap­ing po­ten­tial for its high-priced ri­vals from No­var­tis and Bio­gen.

The FDA has green-lit the com­pa­nies’ spinal mus­cu­lar at­ro­phy drug ris­diplam, which will be mar­ket­ed as Evrys­di in the US, for use in pa­tients two months and old­er. It’s the first SMA drug that can be tak­en oral­ly, as Bio­gen’s Spin­raza is in­ject­ed in­to the spine while No­var­tis’ Zol­gens­ma is a gene ther­a­py.

An­a­lysts have pen­ciled in a peak sales es­ti­mate of around $2 bil­lion for this drug.

“Evrys­di, with its at-home de­liv­ery made pos­si­ble by oral dos­ing, rep­re­sents an im­por­tant and time­ly ad­vance­ment for peo­ple with SMA,” said Genen­tech CMO Levi Gar­raway in a state­ment emailed to End­points News. “Dur­ing these chal­leng­ing times, we are ex­treme­ly proud to bring this ground­break­ing med­i­cine to those liv­ing with this high­ly dis­abling, rare neu­ro­log­i­cal con­di­tion.”

Ris­diplam will be mar­ket­ed in the US by Genen­tech and glob­al­ly by Roche should it at­tain fur­ther ap­provals. The biotech de­vel­oped ris­diplam with the SMA Foun­da­tion and part­ner PTC Ther­a­peu­tics.

Fri­day’s an­nounce­ment came ahead of sched­ule, as the PDU­FA date on ris­diplam had been slat­ed for Au­gust 24.

Bill An­der­son

As for the cost, Evrys­di is priced sig­nif­i­cant­ly low­er than its SMA com­peti­tors. The price max­es out at $340,000 an­nu­al­ly, a Genen­tech spokesper­son told End­points News, and is scaled on a pa­tient’s weight. The cost for an in­fant weigh­ing 15 pounds who is less than two years old would be un­der $100,000, the spokesper­son added.

The pric­ing scale caps when the child reach­es 44 pounds, which puts the av­er­age at about $7727.27 per pound per year for every pa­tient.

Roche CEO Bill An­der­son said ear­ly this year that the plan was to “un­der­whelm with our pric­ing” and they seem to have done so, and more. Un­der­whelm­ing didn’t prove very chal­leng­ing — Zol­gens­ma has the du­bi­ous des­ig­na­tion of most ex­pen­sive drug in the world at $2.1 mil­lion per pa­tient, while Spin­raza costs $750,000 for the first year and then $375,000 every sub­se­quent year.

The rea­son­ing be­hind the weight-based scale is due to how in­fants me­tab­o­lize drugs dif­fer­ent­ly than old­er in­di­vid­u­als, PTC CEO Stu­art Peltz told End­points, adding “the strat­e­gy is there’s a large num­ber of pa­tients who are un­treat­ed, and I think Roche’s ex­pec­ta­tion is to get it to the pa­tients as rapid­ly as pos­si­ble.”

Stu­art Peltz

Evrys­di was ap­proved based on two clin­i­cal tri­als that test­ed ef­fi­ca­cy in var­i­ous ages and sever­i­ties, and the FDA hand­ed the drug quite the broad la­bel. Pre­vi­ous­ly on­ly Spin­raza had been ap­proved to treat SMA in adults and Peltz said the de­vel­op­ment teams fo­cused on a broad tri­al pop­u­la­tion in or­der to serve pa­tients that had pre­vi­ous­ly been un­der­rep­re­sent­ed. No ap­proved treat­ments for SMA ex­ist­ed be­fore 2016.

The Covid-19 pan­dem­ic has al­so thrown a wrin­kle in­to Evrys­di’s launch giv­en the drug’s ease of use at home. While the oth­er ther­a­pies in­volve go­ing out to re­ceive in­jec­tions, Evrys­di is a liq­uid in­gest­ed once a day oral­ly and Peltz said he be­lieves this ac­ces­si­bil­i­ty will make the drug more prefer­able for those still too ner­vous to ven­ture out­doors fre­quent­ly.

Baird an­a­lyst Bri­an Sko­r­ney large­ly agreed with that as­sess­ment, writ­ing to in­vestors, “With the fear of COVID on every­one’s mind, we think it puts Evrys­di in a mar­ketable ad­van­tage over Spin­raza.” That, com­bined with Evrys­di’s sig­nif­i­cant­ly low­er cost, re­sults in the drug “putting sig­nif­i­cant com­pet­i­tive pres­sure” on Spin­raza, which gar­nered $2.1 bil­lion in 2019.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's OCE makes the case for ac­cel­er­at­ed ap­proval rid­er in user fee reau­tho­riza­tion

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.

As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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