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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.

Tae Han Kim, Samsung Biologics CEO (SeongJoon Cho/Bloomberg via Getty Images)

Sam­sung Bi­o­log­ics nets $330M+ deal from As­traZeneca ahead of 'Su­per Plan­t' con­struc­tion

Just a few weeks after announcing plans to construct a $2 billion “Super Plant,” Samsung Biologics is keeping its foot on the gas.

The Korean CDMO has inked a $330.8 million manufacturing contract with AstraZeneca, the companies announced Monday evening, providing the British drugmaker the ability to expand production capabilities in the Asia market. Per terms of the deal, the partnership could be increased to $545.6 million.

Francesco De Rubertis

Medicx­i's David Grainger and Francesco De Ru­ber­tis pump €200M in­to six com­pa­nies and what they say is a first-of-its kind fund

In what they’re billing as a first for biotech, David Grainger, Francesco De Rubertis and their team at Medicxi have put down a €200 million to sweep up stakes in six companies from their predecessor VC and pump new money into them.

Medicxi didn’t disclose which companies it was investing in but the portfolio draws from Index Ventures Life VI, one of the last funds the Medicxi team launched while they were still part of the multinational, tech-focused VC firm Index Ventures. That team kept advising Index on their life sciences portfolio even after they spun out to form their own firm in the middle of 2016.

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Scripps reach­es $10M set­tle­ment with gov­ern­ment over al­le­ga­tions NIH grants weren't prop­er­ly ac­count­ed for

Scripps Research Institute has settled a case with the Justice Department alleging claims of misappropriated funds, the US attorney for the district of Maryland announced late last week.

Prosecutors said the institute improperly used NIH-funded research grants for non-grant related activities, including working on new grant applications, teaching activities and other administrative tasks. As part of the settlement, Scripps has agreed to pay $10 million.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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