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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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When will the FDA re­scind a break­through des­ig­na­tion? New draft guid­ance spells out the com­pli­cat­ed de­tails

Although it’s exceedingly rare for the FDA to rescind a breakthrough designation once it’s granted, there has been a recent uptick — as in 2020 and 2021 combined, the agency rescinded 17 BTDs, compared to just 18 rescinded from 2015 to 2019 combined.

Protagonist Therapeutics saw this reality up close and personal in April after a clinical hold lifted on its experimental blood cancer drug, as the company revealed that the FDA sought to revoke the BTD. The decision, Protagonist says, stems from “observed malignancies” related to the hold, initially imposed in September 2021.

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