Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfiz­er is launch­ing its sec­ond-ever re­bate pro­gram, this time for Panzy­ga, its treat­ment for a rare neu­ro­log­i­cal dis­ease of the pe­riph­er­al nerves.

The pro­gram be­gan last month, ac­cord­ing to STAT which first re­port­ed the news, and of­fers a re­fund of out-of-pock­et costs for pa­tients who must dis­con­tin­ue their course be­fore the fifth treat­ment for “clin­i­cal rea­sons.”

Panzy­ga was ap­proved back in 2018 to treat pri­ma­ry im­mun­od­e­fi­cien­cy (PI) in pa­tients two years and old­er and chron­ic im­mune throm­bo­cy­tope­nia (cITP) in adults. It has since picked up an in­di­ca­tion in chron­ic in­flam­ma­to­ry de­myeli­nat­ing polyneu­ropa­thy (CIDP), a con­di­tion that’s char­ac­ter­ized by weak­ness of the arms or legs, tin­gling or numb­ness, and a loss of deep ten­don re­flex­es, ac­cord­ing to the NIH.

At whole­sale cost, Panzy­ga can run up a tab from around $193 to $5,790 per vial, de­pend­ing on the dose amount, ac­cord­ing to in­for­ma­tion pro­vid­ed re­cent­ly to Col­orado pre­scribers. Panzy­ga is ad­min­is­tered in two load­ing dos­es, fol­lowed by main­te­nance dos­es every three weeks.

To take the edge off, Pfiz­er says it will re­fund CIPD pa­tients for their out-of-pock­et costs for the first four treat­ments if the drug doesn’t work out, up to a max­i­mum of $16,500 per treat­ment or $50,000 to­tal.

“If your com­mer­cial in­sur­ance and/or oth­er pay­ers (“Your Plan(s)”) paid for all or a por­tion of the cost of PANZY­GA, Pfiz­er will, on your be­half, re­fund Your Plan(s) the Av­er­age Sales Price as cal­cu­lat­ed by Pfiz­er up to the Max­i­mum, less doc­u­ment­ed out-of-pock­et pay­ments pro­vid­ed by you or on your be­half,” the com­pa­ny’s web­site states.

While Pfiz­er’s re­bate pro­gram for its can­cer drug Xalko­ri is avail­able to Medicare pa­tients, the Panzy­ga pro­gram is on­ly avail­able to com­mer­cial­ly-in­sured pa­tients or those who pay cash, ex­clud­ing pa­tients cov­ered by gov­ern­ment pro­grams such as Medicare and Med­ic­aid.

Un­der the Xalko­ri pro­gram, el­i­gi­ble pa­tients can get their out-of-pock­et costs cov­ered if they dis­con­tin­ue the drug be­fore the fourth 30-day sup­ply is ad­min­is­tered.

The Panzy­ga re­bate on­ly ap­plies if the drug was dis­con­tin­ued for “clin­i­cal rea­sons de­fined at the dis­cre­tion of the health­care provider,” ac­cord­ing to Pfiz­er. Pa­tients who stop tak­ing the drug by choice or be­cause of af­ford­abil­i­ty don’t qual­i­fy. Pa­tients are al­so dis­qual­i­fied if they’ve used an­oth­er im­munoglob­u­lin prod­uct in the past 12 months.

“Ac­cess to treat­ment for Chron­ic In­flam­ma­to­ry De­myeli­nat­ing Polyneu­ropa­thy may be a bar­ri­er for pa­tients across the U.S.,” a Pfiz­er spokesper­son said in an emailed state­ment. “As part of our com­mit­ment to pa­tients and be­lief in our med­i­cines, Pfiz­er is proud to of­fer the Pfiz­er Pledge War­ran­ty Pro­gram for PANZY­GA. PANZY­GA, ap­proved by the U.S. FDA for the treat­ment of CIDP, has an es­tab­lished clin­i­cal pro­file and HCPs are able to as­sess pa­tient re­sponse with­in the first few treat­ments ad­min­is­tered.”

The news comes as the Sen­ate re­cent­ly passed ma­jor drug pric­ing re­form, which would en­able Medicare ne­go­ti­a­tions and cap se­niors’ out-of-pock­et costs at $2,000 per year. While Pres­i­dent Joe Biden tout­ed the leg­is­la­tion as a “crit­i­cal step” that would “help Amer­i­cans save mon­ey on pre­scrip­tion drugs, health pre­mi­ums, and much more,” phar­ma ex­ecs have ar­gued that the bill will stymie in­no­va­tion.

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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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.

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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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.

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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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.