ICER says it's fair for Bio­Marin and CSL to price in­com­ing he­mo­phil­ia gene ther­a­pies at $2M-$3M

Get ready for mil­lion-plus-dol­lar gene ther­a­pies to be­come the norm rather than the ex­cep­tion, and in some cas­es, the Boston-based drug pric­ing watch­dog ICER is com­ing on board.

The non­prof­it ICER (In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view) on Wednes­day un­veiled a new re­port find­ing that CSL Behring’s po­ten­tial gene ther­a­py etranaco­gene deza­parvovec for he­mo­phil­ia B, which is due for an FDA ap­proval de­ci­sion by the end of this month, could be fair­ly priced at around $3 mil­lion. ICER al­so up­dat­ed its pre­vi­ous as­sess­ment of Bio­Marin’s EU-au­tho­rized he­mo­phil­ia A gene ther­a­py Roc­ta­vian, which ICER said could be fair­ly priced at about $2 mil­lion.

David Rind

“The new gene ther­a­pies can re­sult in suc­cess­ful­ly-treat­ed pa­tients ap­pear­ing ‘cured’ for at least a pe­ri­od of time,” ICER chief med­ical of­fi­cer David Rind said in a state­ment. “Dur­ing this pe­ri­od these gene ther­a­pies will elim­i­nate the need for ex­pen­sive pro­phy­lac­tic treat­ment. How­ev­er, the du­ra­tion of this ‘cure’ and the safe­ty of ther­a­pies re­main im­por­tant un­cer­tain­ties.”

In the case of CSL’s he­mo­phil­ia B can­di­date, which us­es an AAV5 vec­tor to car­ry the Pad­ua gene vari­ant of Fac­tor IX (FIX-Pad­ua), gen­er­at­ing FIX pro­teins that are 5x-8x more ac­tive than nor­mal, ICER not­ed that treat­ed pa­tients saw an 80% re­duc­tion in treat­ed joint bleeds and sim­i­lar re­duc­tions in oth­er bleeds when com­pared with their bleed­ing rates pri­or to gene ther­a­py.

While no pa­tients suc­cess­ful­ly treat­ed with the gene ther­a­py re­turned to pri­or pro­phy­lac­tic treat­ment dur­ing the first 18 months on the he­mo­phil­ia B ther­a­py, ICER said it “is not yet clear that the ini­tial in­crease in fac­tor IX lev­els will be main­tained for decades, though the re­sults are en­cour­ag­ing. Fi­nal­ly, the re­duc­tion in bur­den of ther­a­py – no longer need­ing week­ly or more fre­quent IX fac­tor ther­a­py is a ma­jor ben­e­fit for pa­tients.”

About 30,000 Amer­i­cans have ei­ther he­mo­phil­ia A or B, which means they have an in­creased ten­den­cy to bleed due to in­her­it­ed de­fi­cien­cies of fac­tor VI­II and fac­tor IX.

In the case of Bio­Marin’s Roc­ta­vian, or val­rox, which has al­ready been through a round of re­jec­tion at FDA and faces a new PDU­FA date of March 31, 2023, ICER con­clud­ed that there is “low cer­tain­ty” about the net health ben­e­fit for Roc­ta­vian com­pared with Roche’s Hem­li­bra (emi­cizum­ab), which costs about $630,000 per year.

In the EU, how­ev­er, Roc­ta­vian has al­ready won a con­di­tion­al ap­proval and set its price at about $1.5 mil­lion.

But ICER says Bio­Marin could go even high­er with that price, to al­most $2 mil­lion. And at a cost of $2.5 mil­lion, if the dura­bil­i­ty of the ther­a­py holds up for four years, ICER notes that val­rox goes from not cost-ef­fec­tive to dom­i­nant.

“The gene ther­a­pies have large cost sav­ings as­so­ci­at­ed with them with very large life­time costs as­so­ci­at­ed with both the treat­ments and com­para­tors in both mod­els,” ICER con­clud­ed. “In ad­di­tion, the gene ther­a­pies are as­so­ci­at­ed with slight­ly high­er QALYs and low­er bleeds.”

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

Sanofi's new headquarters, La Maison Sanofi, in Paris (Credit: Luc Boegly)

Sanofi wel­comes 500 staffers to new Paris HQ af­ter €30M ren­o­va­tion

When Paul Hudson took the helm at Sanofi back in 2019, he promised to reinvent the pharma giant — including its Paris headquarters. This week, the company set up shop in new “state-of-the-art” digs.

La Maison Sanofi, as the new HQ is called, is officially open for business, Hudson announced on Monday. The 9,000-square-meter (just under 97,000-square-foot) space accommodates 500 employees across the company’s government and global support functions teams, including finance, HR, legal and corporate affairs — and it was built with environmental sustainability and hybrid work in mind.

Sta­da to place $50M+ in­vest­ment in a new fa­cil­i­ty in Ro­ma­nia

While Romania may conjure up images of vast mountain ranges and tales of medieval kings, one generic manufacturer has broken ground on a new facility there.

German pharma company Stada said Monday that it has placed a €50 million ($51.9 million) investment into a 100,000 square-meter (1.08 million square-foot) site in Turda, Romania, a city in the Southeast of the country. According to a Stada spokesperson in an email to Endpoints News, the company has developed only 281,500 square feet of the site so far.

Rachael Rollins (Charles Krupa/AP Images)

US seeks jail time for co-CEO of New Eng­land com­pound­ing cen­ter af­ter dead­ly 2012 fun­gal out­break

The US attorney for the district of Massachusetts late last week called on the state’s district court to sentence the former co-owner of the now-defunct New England Compounding Center to 18 months of jail time for his role in the center’s quality deviations that led to more than 100 people dead from a fungal meningitis outbreak.

Gregory Conigliaro was convicted of conspiring with more than a dozen others at NECC to deceive the FDA and misrepresent the fact that the center was only dispensing drugs pursuant to patient-specific prescriptions.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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